Spinal Muscular Atrophy Type 3NCT03056144
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy
A study testing whole body vibration therapy in people with Spinal Muscular Atrophy Type 3, run by The Hong Kong Polytechnic University at 1 site in Hong Kong.
Trial summary
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers. Osteopenia is common for this population group due to poor bone growth and muscle disuse. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Spinal Muscular Atrophy Type 3
- What is being tested?
- whole body vibration therapy
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteHong Kong
- Study dates
- 1 Aug 2017to 12 Jul 2018
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of type III spinal muscular atrophy
- Be able to stand on the vibration platform with or without support
- Be able to undertake clinical examination and DXA evaluation
- Informed consent by the participant's parent/ guardian
Who cannot join
- There is a history of fracture within 8 weeks of enrolment of the present study and acute thrombosis, muscle or tendon inflammation, renal stones, discopathy or arthritis as reported by their parent/ guardian.
- There is a history of using any of the following medications, regardless of dose, for at least 1 month, within 3 months of enrolment into the present study: anabolic agents, or growth hormone.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Effect of Whole Body Vibration Therapy on Muscle Function, Gross Motor Function and Bone Mineral Density in Children With Spinal Muscular Atrophy - a Feasibility Study
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers. Osteopenia is common for this population group due to poor bone growth and muscle disuse. As a result, non-traumatic related fractures and bone pain are common. Recently, whole body vibration therapy (WBVT) has been proven to improve bone health and muscle function in healthy adults and post-menopausal women. Among the limited studies on the WBVT for children with muscular dystrophies, promising results have been shown on gross motor function, balance, and muscle strength and the WBVT appears to be safe for children with SMA.
The present pilot study is designed to investigate if WBVT is safe and feasible for individuals with SMA and if WBVT can improve muscle function, functional abilities, postural control and bone mineral density in children with SMA. Convenience samples of 10 individuals with SMA type III will be recruited. The participants will receive the WBVT of 25 Hertz and a peak-to-peak amplitude of 4mm for a session of about 18 minutes, 3 days per week for 4 weeks. Assessment will be performed at the baseline and the completion of the intervention to examine the muscle function, functional abilities, postural control and bone mineral density of the participants.
It is anticipated that the outcomes of this pilot study for SMA may show if this intervention is safe, feasible and beneficial for children with SMA type III regarding to muscle function, functional abilities, postural control and bone mineral content and if there may be any related practical issues of this intervention to this population group. The outcomes also provide research evidence to clinicians if this intervention should be recommended to individuals of similar problems.
Read the detailed description
Spinal muscular atrophy (SMA) is an X-chromosome-linked disorder, in which there is a loss of motor neurons from the anterior horn of the spinal cord due to a deletion of the SMN1 gene. SMA is usually classified under 4 categories, based on the onset time and severity of the conditions. Type I SMA is the most severe category, when the boy is diagnosed before 6 months old and has severe muscle weakness, causing them to have poor head control and unable to sit independently. Boys with type II SMA are diagnosed between 6 to 18 months of age and able to sit independently but cannot stand or walk without any assistance. SMA type III is diagnosed between 18 months to 30 years of age and the boys can stand and walk independently but still with variable degrees of muscle weakness. Some would lose ambulation in their early adulthood and require wheelchair mobility. Type IV SMA is the mildest form with an adult onset, normal mobility and longevity. However, they also experience mild muscle weakness throughout their life. This muscle weakness would lead to early loss of ambulation, reduced pulmonary function and complications due to immobilisation such as osteoporosis. Early fatality is not uncommon.
Osteopenia due to disuse is, in fact, common in children with physical disabilities. In a study of 69 children with moderate to severe cerebral palsy (CP), it was shown that the distal femur and lumbar spine areal bone mineral density (BMD) z-scores appeared to worsen with time, which may reflect the possibility of poor bone growth velocity in individuals with CP. Fracture and bone pain are the major complications of osteopenia in CP and the majority of non-traumatic fractures occur in the femur and humerus. Other factors that may contribute to osteopenia in physical disabilities include pubertal delay, vitamin D deficiency, dietary calcium deficiency, under-nutrition and low body weight, corticosteroids or anticonvulsants. Despite of minimising these factors, osteopenia appears to persist.
Limited studies have been done to examine the bone health in children with SMA but more in children with Duchene muscular dystrophy (DMD), which have similar clinical presentations although with different pathologies. A study on 41 boys with DMD, bone density in the proximal femur was significantly decreased even in the ambulatory boys (mean z-score -1.6) and progressing rapidly to a level of 4 standard deviations below the norm when compared with normal boys. Forty-four percent of the boys had an episode of fracture, mostly in the lower limbs.
Recently whole body vibration therapy (WBVT) has been preliminarily shown as a simple and effective technique to increase bone mass, muscle mass and strength. In general, the user stands in a static position such as standing or performs some dynamic movements on a device providing vibrations from a few Hz to 50 Hz (Hertz, Hz represents the number of complete up and down movement cycle per second). It has been hypothesised that the vibrations stimulate the muscle spindles and alpha-motor neurons, eliciting a muscle contraction. The latter would increase the muscle mass and in turn, increase the bone mass. It has also been postulated that direct effect by mechanical deformation of bones and increased fluid flow in the canalicular spaces and stimulation of the osteocytes may contribute increase in bone mass with the vibration therapy. Increase in oxygen consumption, body temperature and skin blood flow (erythema) have also been demonstrated. As WBVT does not elicit a significant cardiovascular response, it appears to be safe to be used in children with various medical conditions.
In a systematic review on 22 studies (including 7 studies on CP and 2 on DMD) for the effect of WBVT on body composition and physical fitness in children and adolescents with disabilities, the authors concluded that WBVT appeared to improve bone mass and muscle strength in this population group. However, heterogeneity of the studies was noticed, including great variations in the treatment protocols and lacking of a control group and hence, no recommended minimal dosage of WBVT can be concluded. Since this review, two more randomised controlled trials (RCT) were published on children with CP. In one recent study, 30 children with spastic diplegia CP of GMFCS levels I to II were randomised into a treatment group (WBVT with traditional physiotherapy) and a control group (physiotherapy only). The treatment group received 3 lots of 3 minutes on and 3 minutes off vibration (12 to 18 Hz), 2 to 5 times per week for 3 months. Significant improvement in knee extensor strength and standing stability was reported in the treatment group. In another study in 2013, 27 children with spastic diplegia or hemiplegia CP of GMFCS levels I to III were randomised to a treatment group or control group and then crossed over after 4 weeks. The treatment group performed specific trunk exercise on the vibration platform (35 Hz), 5 to 10 minute per session, 2 to 4 sessions per week for 4 weeks. Significant improvement was found in gait speed, muscle thickness of the abdominal muscle and number of sit-ups done 1 minute. A visual improvement was also shown in sitting and standing postures.
Although it has been shown that high frequency low amplitude vibration seemed to be a safe rehabilitation in mice with muscular dystrophy, intensive strengthening exercises, which may induce more damage to the muscle fibres for children with DMD or SMA as clinically indicated with a raised serum creatine kinase (SCK) level, remain as a concern. Hence current studies on this population group targeted to examine the safety of this intervention. Three studies on children with DMD and 1 on DMD and SMA using WBVT were found. In general, it appears that WBVT seems to be safe for children with DMD or SMA. Although there might be a raised SCK level, the level would gradually reduce to the baseline level, or if not, there was no clinical sign or symptom for muscle damage. A promising result was also shown in improving bone mineral density in children with DMD. However, due to the overall small number of studies and sample sizes, there is no definite conclusion if WBVT is effective in improving the bone density and muscle strength for this population group yet.
Based on current research evidence, it has been suggested that 10 to 20 minutes per session, at least 3 times per week for minimum 26 weeks with frequency between 25 to 35 Hz and a peak to peak amplitude less than 4 mm may be an appropriate protocol targeting to improve bone mass and muscle strength of children and adolescents with disabilities. Studies of rigorous research designs and homogeneous participants are required to investigate if this recommended dosage of WBVT can improve children with disabling conditions.
Methodology This feasibility study aims to examine the safety of the WBVT on children with type III SMA. Children with type III SMA are targeted as they have adequate independence living in the community but still experience early fatigue during normal level of exercises due to the nature of their condition. They are at high risk of suffering from complications due to compromised mobility such as osteopenia, early loss of ambulation when compared with their healthy peers.
The WBVT will be performed on the GalileoTM Med L Plus (Novotech Medical GmbH) with the study participants standing with both knees flexed at least 20 degrees. The vibration frequency and duration will be increased over 5 days to the maximum of 3 minutes of 24 to 25 Hz with a peak to peak amplitude of 4mm. The participants will undergo the WBVT 1 session per day, 3 days per week for 4 weeks. The whole WBVT session will last 18 minutes with 9 minutes of vibration.
Participants:
10 children with type III SMA aged from 6 to 18 years will be recruited. The age range is extended aiming to increase the number of recruitment due to the rarity of the condition. All participants will continue their usual intervention regime, if any, during the study period.
Recruitment:
Children and families will be identified by their paediatrician at the neuromuscular clinic at the Duchess of Kent Children's Hospital in Hong Kong. Participants and/or their parents/guardians will be asked if they are interested to participate in this study and their contact details (name and contact telephone number) will be passed onto the PI. PI will contact the families by telephone.
Power analysis:
There is no previous study specifically conducted for this group of children and adolescents and hence no data is available for the power calculation. Most importantly, the aim of this study is to examine the safety and feasibility of the WBVT for this group of clients.
- Conditions
- Spinal Muscular Atrophy Type 3
- Being tested
- whole body vibration therapy
- Keywords
- spinal muscular atrophy, total body vibration, children
- Medical subject headings
- Spinal Muscular Atrophies of Childhood, Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
whole body vibration therapyDevice
The whole body vibration therapy regime is as follows:
Day Vibration 1 Rest 1 Vibration 2 Rest 2 Vibration 3 Rest 3
- st 1 min;12Hz 3 min 1 min;12Hz 3 min 1 min;15Hz 3 min
- nd 1 min;15Hz 3 min 1 min;15Hz 3 min 2 min;15Hz 3 min
- th 2 min;15Hz 3 min 3 min;15Hz 3 min 3 min;15Hz 3 min
- th 2 min;24-25Hz 3 min 2 min;24-25Hz 3 min 2 min;24-25Hz 3 min >5th 3 min;24-25Hz 3 min 3 min;24-25Hz 3 min 3 min;24-25Hz 3 min
The participants will perform mini-squats during Vibrations 1 and 3 and weight-shifting between right and left legs during Vibration 2 on the vibration platform under the supervision of a trained research assistant.
| Study group | Role | What they receive |
|---|---|---|
| Intervention group | Experimental | The participants will undergo the whole body vibration therapy 1 session per day, 3 days per week for 4 weeks. The whole total whole body therapy session will last 18 minutes with 9 minutes of vibration. Receives: Device: whole body vibration therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
North Star Ambulatory Assessment
examine the gross motor function of the participants. A summed score will be added from each test item.
Measured: 4 weeks
2-minute Walk Test
assess submaximal exercise capacity by measuring the distance covered in the 2 minutes in metres
Measured: 4 weeks
Segmental Assessment of Trunk Control-static
assess the segmental trunk control in sitting position with an ordinal score will be given in static trunk control. Assessment score represents as follows: 1= learning head control, 2= learning upper thoracic control, 3= learning mid-thoracic control, 4= learning lower thoracic control, 5= learning at upper lumber control, 6= learning lower lumbar control, 7= learning full trunk control and 8= achieved full trunk control.
Measured: 4 weeks
Pediatric Evaluation of Disability Inventory
assess functional capacities in the domains of self care, mobility and social function with a summary score in each domain. A dichotomous score will be given to each question in each domain: 0= unable and 1= able. In self care domain, there are 73 questions, i.e. maximal score is 73. In mobility domain, there are 59 questions i.e. maximal score is 59. In social function domain, there are 65 questions i.e. maximal score is 65.
Measured: 4 weeks
Body Height
measure height in cm
Measured: 4 weeks
Body Weight
measure weight in kilograms
Measured: 4 weeks
Body Mass Index
calculated based on body height and weight in terms of kg/m2
Measured: 4 weeks
Bone Mineral Content of Femur
Distal femur BMC will be measured in grams
Measured: 4 weeks
Bone Mineral Content of Whole Body (Excluding Head)
Whole body (excluding head) BMC will be measured in grams
Measured: 4 weeks
Areal Bone Mineral Density of Femur
Areal bone mineral density of femur will be measured in grams/cm2
Measured: 4 weeks
Areal Bone Mineral Density of Total Body (Excluding Head)
Areal bone mineral density of total body (excluding head) will be measured in grams/cm2
Measured: 4 weeks
Volumetric Bone Mineral Density of Lumbar Spine
Volumetric bone mineral density of lumbar spine (L2 to L4) in grams/cm3
Measured: 4 weeks
Range of Right Hip Flexion
measure hip flexion in supine using goniometer in degrees
Measured: 4 weeks
Range of Left Hip Flexion
measure hip flexion in supine using goniometer in degrees
Measured: 4 weeks
Range of Right Hip Extension
measure hip extension in prone using goniometer in degrees
Measured: 4 weeks
Range of Left Hip Extension
measure hip extension in prone using goniometer in degrees
Measured: 4 weeks
Range of Right Hip Abduction
measure hip abduction in supine using goniometer in degrees
Measured: 4 weeks
Range of Left Hip Abduction
measure hip abduction in supine using goniometer in degrees
Measured: 4 weeks
Range of Right Knee Flexion
measure knee flexion in prone using goniometer in degrees
Measured: 4 weeks
Range of Left Knee Flexion
measure knee flexion in prone using goniometer in degrees
Measured: 4 weeks
Range of Right Knee Extension
measure knee extension in sitting using goniometer in degrees
Measured: 4 weeks
Range of Left Knee Extension
measure knee extension in sitting using goniometer in degrees
Measured: 4 weeks
Range of Right Ankle Dorsiflexion
measure ankle dorsiflexion in sitting using goniometer in degrees
Measured: 4 weeks
Range of Left Ankle Dorsiflexion
measure ankle dorsiflexion in sitting using goniometer in degrees
Measured: 4 weeks
Range of Right Ankle Plantarflexion
measure ankle plantarflexion in sitting using goniometer in degrees
Measured: 4 weeks
Range of Left Ankle Plantarflexion
measure ankle plantarflexion in sitting using goniometer in degrees
Measured: 4 weeks
Muscle Strength of Right Hip Flexors
measure muscle strength of hip flexors in supine using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Left Hip Flexors
measure muscle strength of hip flexors in supine using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Right Hip Extensors
measure muscle strength of hip extensors in prone using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Left Hip Extensors
measure muscle strength of hip extensors in prone using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Right Knee Flexors
measure muscle strength of knee flexors in sitting using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Left Knee Flexors
measure muscle strength of knee flexors in sitting using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Right Knee Extensors
measure muscle strength of knee extensors in sitting using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Left Knee Extensors
measure muscle strength of knee extensors in sitting using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Right Hip Abductors
measure muscle strength of hip abductors in supine using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Left Hip Abductors
measure muscle strength of hip abductors in supine using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Right Ankle Dorsiflexors
measure muscle strength of ankle dorsiflexors in sitting using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Left Ankle Dorsiflexors
measure muscle strength of ankle dorsiflexors in sitting using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Right Ankle Plantarflexors
measure muscle strength of ankle plantarflexors in sitting using dynamometer in terms of Newton
Measured: 4 weeks
Muscle Strength of Left Ankle Plantarflexors
measure muscle strength of ankle plantarflexors in sitting using dynamometer in terms of Newton
Measured: 4 weeks
Segmental Assessment of Trunk Control_active
assess the segmental trunk control in sitting position with an ordinal score will be given in active trunk control. Assessment score represents as follows: 1= learning head control, 2= learning upper thoracic control, 3= learning mid-thoracic control, 4= learning lower thoracic control, 5= learning at upper lumber control, 6= learning lower lumbar control, 7= learning full trunk control and 8= achieved full trunk control.
Measured: 4 weeks
Segmental Assessment of Trunk Control-reactive
assess the segmental trunk control in sitting position with an ordinal score will be given in reactive trunk control. Assessment score represents as follows: 1= learning head control, 2= learning upper thoracic control, 3= learning mid-thoracic control, 4= learning lower thoracic control, 5= learning at upper lumber control, 6= learning lower lumbar control, 7= learning full trunk control and 8= achieved full trunk control.
Measured: 4 weeks
Secondary outcomes (2)
Percentage of Attendance of Participants
record the percentage of attendance and comments during the intervention
Measured: 4 weeks
Visual Analogue Scale
record discomfort during the intervention in a scale of 0 (no discomfort) to 10 (maximal discomfort).
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2017
- Study start1 Aug 2017
- Primary completion12 Jul 2018
- Study completion12 Jul 2018
Why it stopped: All potential participants opt to a simultaneous pharmacological clinical trial
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (20)Papers the sponsor has linked to this study.
- Other trials for Spinal Muscular Atrophy Type 3Studies of the same condition, open and under way.
- More trials run by The Hong Kong Polytechnic UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A pilot study with a before and after study design
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Hong Kong Polytechnic University
- Sponsor type
- University, hospital or other
- Collaborators
- Manchester Metropolitan University, The University of Hong Kong
- Responsible party
- Principal investigator, Dr Tamis Wai-mun PIN, The Hong Kong Polytechnic University
- Investigators
- Tamis W Pin, PhD, Principal investigator, The Hong Kong Polytechnic University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03056144
- Sponsor's protocol ID
- YBPA
- Status last verified
- Jul 2019
- First submitted
- 8 Nov 2016
- Last updated
- 26 Jul 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Vry J, Schubert IJ, Semler O, Haug V, Schonau E, Kirschner J. Whole-body vibration training in children with Duchenne muscular dystrophy and spinal muscular atrophy. Eur J Paediatr Neurol. 2014 Mar;18(2):140-9. doi: 10.1016/j.ejpn.2013.09.005. Epub 2013 Oct 11.
PubMed 24157400Henderson RC, Kairalla JA, Barrington JW, Abbas A, Stevenson RD. Longitudinal changes in bone density in children and adolescents with moderate to severe cerebral palsy. J Pediatr. 2005 Jun;146(6):769-75. doi: 10.1016/j.jpeds.2005.02.024.
PubMed 15973316Stevenson RD, Conaway M, Barrington JW, Cuthill SL, Worley G, Henderson RC. Fracture rate in children with cerebral palsy. Pediatr Rehabil. 2006 Oct-Dec;9(4):396-403. doi: 10.1080/13638490600668061.
PubMed 17111566Mergler S, Evenhuis HM, Boot AM, De Man SA, Bindels-De Heus KG, Huijbers WA, Penning C. Epidemiology of low bone mineral density and fractures in children with severe cerebral palsy: a systematic review. Dev Med Child Neurol. 2009 Oct;51(10):773-8. doi: 10.1111/j.1469-8749.2009.03384.x. Epub 2009 Jul 8.
PubMed 19614941Houlihan CM, Stevenson RD. Bone density in cerebral palsy. Phys Med Rehabil Clin N Am. 2009 Aug;20(3):493-508. doi: 10.1016/j.pmr.2009.04.004.
PubMed 19643349Henderson RC, Lark RK, Gurka MJ, Worley G, Fung EB, Conaway M, Stallings VA, Stevenson RD. Bone density and metabolism in children and adolescents with moderate to severe cerebral palsy. Pediatrics. 2002 Jul;110(1 Pt 1):e5. doi: 10.1542/peds.110.1.e5.
PubMed 12093986Larson CM, Henderson RC. Bone mineral density and fractures in boys with Duchenne muscular dystrophy. J Pediatr Orthop. 2000 Jan-Feb;20(1):71-4.
PubMed 10641693Rauch F. Vibration therapy. Dev Med Child Neurol. 2009 Oct;51 Suppl 4:166-8. doi: 10.1111/j.1469-8749.2009.03418.x.
PubMed 19740225Jordan MJ, Norris SR, Smith DJ, Herzog W. Vibration training: an overview of the area, training consequences, and future considerations. J Strength Cond Res. 2005 May;19(2):459-66. doi: 10.1519/13293.1.
PubMed 15903391Rehn B, Lidstrom J, Skoglund J, Lindstrom B. Effects on leg muscular performance from whole-body vibration exercise: a systematic review. Scand J Med Sci Sports. 2007 Feb;17(1):2-11. doi: 10.1111/j.1600-0838.2006.00578.x. Epub 2006 Aug 10.
PubMed 16903900Matute-Llorente A, Gonzalez-Aguero A, Gomez-Cabello A, Vicente-Rodriguez G, Casajus Mallen JA. Effect of whole-body vibration therapy on health-related physical fitness in children and adolescents with disabilities: a systematic review. J Adolesc Health. 2014 Apr;54(4):385-96. doi: 10.1016/j.jadohealth.2013.11.001. Epub 2014 Jan 1.
PubMed 24388109El-Shamy SM. Effect of whole-body vibration on muscle strength and balance in diplegic cerebral palsy: a randomized controlled trial. Am J Phys Med Rehabil. 2014 Feb;93(2):114-21. doi: 10.1097/PHM.0b013e3182a541a4.
PubMed 24434887Unger M, Jelsma J, Stark C. Effect of a trunk-targeted intervention using vibration on posture and gait in children with spastic type cerebral palsy: a randomized control trial. Dev Neurorehabil. 2013;16(2):79-88. doi: 10.3109/17518423.2012.715313.
PubMed 23477461Novotny SA, Mader TL, Greising AG, Lin AS, Guldberg RE, Warren GL, Lowe DA. Low intensity, high frequency vibration training to improve musculoskeletal function in a mouse model of Duchenne muscular dystrophy. PLoS One. 2014 Aug 14;9(8):e104339. doi: 10.1371/journal.pone.0104339. eCollection 2014.
PubMed 25121503Chelly J, Desguerre I. Progressive muscular dystrophies. Handb Clin Neurol. 2013;113:1343-66. doi: 10.1016/B978-0-444-59565-2.00006-X.
PubMed 23622359
Study at a glance
From the registry record, last updated by the sponsor on 26 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Whole Body Vibration Therapy on Muscle Function, Gross Motor Function and Bone Mineral Density in Children With Spinal Muscular Atrophy - a Feasibility Study
- ClinicalTrials.gov ID
- NCT03056144
- Acronym / sponsor ID
- YBPA
- Conditions
- Spinal Muscular Atrophy Type 3
- Keywords
- spinal muscular atrophy, total body vibration, children
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: All potential participants opt to a simultaneous pharmacological clinical trial
- Enrolment
- 1 participants (actual)
- Lead sponsor
- The Hong Kong Polytechnic UniversityUniversity, hospital or other
- Collaborators
- Manchester Metropolitan University, The University of Hong Kong
- Study start
- 1 Aug 2017 (actual)
- Primary completion
- 12 Jul 2018 (actual)
- Study completion
- 12 Jul 2018 (actual)
- First posted
- 17 Feb 2017 (actual)
- Results
- Posted 26 Jul 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers. Osteopenia is common for this population group due to poor bone growth and muscle disuse. As a result, non-traumatic related fractures and bone pain are common. Recently, whole body vibration therapy (WBVT) has been proven to improve bone health and muscle function in healthy adults and post-menopausal women. Among the limited studies on the WBVT for children with muscular dystrophies, promising results have been shown on gross motor function, balance, and muscle strength and the WBVT appears to be safe for children with SMA.
The present pilot study is designed to investigate if WBVT is safe and feasible for individuals with SMA and if WBVT can improve muscle function, functional abilities, postural control and bone mineral density in children with SMA. Convenience samples of 10 individuals with SMA type III will be recruited. The participants will receive the WBVT of 25 Hertz and a peak-to-peak amplitude of 4mm for a session of about 18 minutes, 3 days per week for 4 weeks. Assessment will be performed at the baseline and the completion of the intervention to examine the muscle function, functional abilities, postural control and bone mineral density of the participants.
It is anticipated that the outcomes of this pilot study for SMA may show if this intervention is safe, feasible and beneficial for children with SMA type III regarding to muscle function, functional abilities, postural control and bone mineral content and if there may be any related practical issues of this intervention to this population group. The outcomes also provide research evidence to clinicians if this intervention should be recommended to individuals of similar problems.
Detailed description
Spinal muscular atrophy (SMA) is an X-chromosome-linked disorder, in which there is a loss of motor neurons from the anterior horn of the spinal cord due to a deletion of the SMN1 gene. SMA is usually classified under 4 categories, based on the onset time and severity of the conditions. Type I SMA is the most severe category, when the boy is diagnosed before 6 months old and has severe muscle weakness, causing them to have poor head control and unable to sit independently. Boys with type II SMA are diagnosed between 6 to 18 months of age and able to sit independently but cannot stand or walk without any assistance. SMA type III is diagnosed between 18 months to 30 years of age and the boys can stand and walk independently but still with variable degrees of muscle weakness. Some would lose ambulation in their early adulthood and require wheelchair mobility. Type IV SMA is the mildest form with an adult onset, normal mobility and longevity. However, they also experience mild muscle weakness throughout their life. This muscle weakness would lead to early loss of ambulation, reduced pulmonary function and complications due to immobilisation such as osteoporosis. Early fatality is not uncommon.
Osteopenia due to disuse is, in fact, common in children with physical disabilities. In a study of 69 children with moderate to severe cerebral palsy (CP), it was shown that the distal femur and lumbar spine areal bone mineral density (BMD) z-scores appeared to worsen with time, which may reflect the possibility of poor bone growth velocity in individuals with CP. Fracture and bone pain are the major complications of osteopenia in CP and the majority of non-traumatic fractures occur in the femur and humerus. Other factors that may contribute to osteopenia in physical disabilities include pubertal delay, vitamin D deficiency, dietary calcium deficiency, under-nutrition and low body weight, corticosteroids or anticonvulsants. Despite of minimising these factors, osteopenia appears to persist.
Limited studies have been done to examine the bone health in children with SMA but more in children with Duchene muscular dystrophy (DMD), which have similar clinical presentations although with different pathologies. A study on 41 boys with DMD, bone density in the proximal femur was significantly decreased even in the ambulatory boys (mean z-score -1.6) and progressing rapidly to a level of 4 standard deviations below the norm when compared with normal boys. Forty-four percent of the boys had an episode of fracture, mostly in the lower limbs.
Recently whole body vibration therapy (WBVT) has been preliminarily shown as a simple and effective technique to increase bone mass, muscle mass and strength. In general, the user stands in a static position such as standing or performs some dynamic movements on a device providing vibrations from a few Hz to 50 Hz (Hertz, Hz represents the number of complete up and down movement cycle per second). It has been hypothesised that the vibrations stimulate the muscle spindles and alpha-motor neurons, eliciting a muscle contraction. The latter would increase the muscle mass and in turn, increase the bone mass. It has also been postulated that direct effect by mechanical deformation of bones and increased fluid flow in the canalicular spaces and stimulation of the osteocytes may contribute increase in bone mass with the vibration therapy. Increase in oxygen consumption, body temperature and skin blood flow (erythema) have also been demonstrated. As WBVT does not elicit a significant cardiovascular response, it appears to be safe to be used in children with various medical conditions.
In a systematic review on 22 studies (including 7 studies on CP and 2 on DMD) for the effect of WBVT on body composition and physical fitness in children and adolescents with disabilities, the authors concluded that WBVT appeared to improve bone mass and muscle strength in this population group. However, heterogeneity of the studies was noticed, including great variations in the treatment protocols and lacking of a control group and hence, no recommended minimal dosage of WBVT can be concluded. Since this review, two more randomised controlled trials (RCT) were published on children with CP. In one recent study, 30 children with spastic diplegia CP of GMFCS levels I to II were randomised into a treatment group (WBVT with traditional physiotherapy) and a control group (physiotherapy only). The treatment group received 3 lots of 3 minutes on and 3 minutes off vibration (12 to 18 Hz), 2 to 5 times per week for 3 months. Significant improvement in knee extensor strength and standing stability was reported in the treatment group. In another study in 2013, 27 children with spastic diplegia or hemiplegia CP of GMFCS levels I to III were randomised to a treatment group or control group and then crossed over after 4 weeks. The treatment group performed specific trunk exercise on the vibration platform (35 Hz), 5 to 10 minute per session, 2 to 4 sessions per week for 4 weeks. Significant improvement was found in gait speed, muscle thickness of the abdominal muscle and number of sit-ups done 1 minute. A visual improvement was also shown in sitting and standing postures.
Although it has been shown that high frequency low amplitude vibration seemed to be a safe rehabilitation in mice with muscular dystrophy, intensive strengthening exercises, which may induce more damage to the muscle fibres for children with DMD or SMA as clinically indicated with a raised serum creatine kinase (SCK) level, remain as a concern. Hence current studies on this population group targeted to examine the safety of this intervention. Three studies on children with DMD and 1 on DMD and SMA using WBVT were found. In general, it appears that WBVT seems to be safe for children with DMD or SMA. Although there might be a raised SCK level, the level would gradually reduce to the baseline level, or if not, there was no clinical sign or symptom for muscle damage. A promising result was also shown in improving bone mineral density in children with DMD. However, due to the overall small number of studies and sample sizes, there is no definite conclusion if WBVT is effective in improving the bone density and muscle strength for this population group yet.
Based on current research evidence, it has been suggested that 10 to 20 minutes per session, at least 3 times per week for minimum 26 weeks with frequency between 25 to 35 Hz and a peak to peak amplitude less than 4 mm may be an appropriate protocol targeting to improve bone mass and muscle strength of children and adolescents with disabilities. Studies of rigorous research designs and homogeneous participants are required to investigate if this recommended dosage of WBVT can improve children with disabling conditions.
Methodology This feasibility study aims to examine the safety of the WBVT on children with type III SMA. Children with type III SMA are targeted as they have adequate independence living in the community but still experience early fatigue during normal level of exercises due to the nature of their condition. They are at high risk of suffering from complications due to compromised mobility such as osteopenia, early loss of ambulation when compared with their healthy peers.
The WBVT will be performed on the GalileoTM Med L Plus (Novotech Medical GmbH) with the study participants standing with both knees flexed at least 20 degrees. The vibration frequency and duration will be increased over 5 days to the maximum of 3 minutes of 24 to 25 Hz with a peak to peak amplitude of 4mm. The participants will undergo the WBVT 1 session per day, 3 days per week for 4 weeks. The whole WBVT session will last 18 minutes with 9 minutes of vibration.
Participants:
10 children with type III SMA aged from 6 to 18 years will be recruited. The age range is extended aiming to increase the number of recruitment due to the rarity of the condition. All participants will continue their usual intervention regime, if any, during the study period.
Recruitment:
Children and families will be identified by their paediatrician at the neuromuscular clinic at the Duchess of Kent Children's Hospital in Hong Kong. Participants and/or their parents/guardians will be asked if they are interested to participate in this study and their contact details (name and contact telephone number) will be passed onto the PI. PI will contact the families by telephone.
Power analysis:
There is no previous study specifically conducted for this group of children and adolescents and hence no data is available for the power calculation. Most importantly, the aim of this study is to examine the safety and feasibility of the WBVT for this group of clients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A pilot study with a before and after study design
| Arm | Type | Receives |
|---|---|---|
| Intervention group | Experimental | Device: whole body vibration therapyThe participants will undergo the whole body vibration therapy 1 session per day, 3 days per week for 4 weeks. The whole total whole body therapy session will last 18 minutes with 9 minutes of vibration. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Diagnosis of type III spinal muscular atrophy
- Be able to stand on the vibration platform with or without support
- Be able to undertake clinical examination and DXA evaluation
- Informed consent by the participant's parent/ guardian
Key exclusion criteria (2 listed)
- There is a history of fracture within 8 weeks of enrolment of the present study and acute thrombosis, muscle or tendon inflammation, renal stones, discopathy or arthritis as reported by their parent/ guardian.
- There is a history of using any of the following medications, regardless of dose, for at least 1 month, within 3 months of enrolment into the present study: anabolic agents, or growth hormone.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| whole body vibration therapyThe whole body vibration therapy regime is as follows: Day Vibration 1 Rest 1 Vibration 2 Rest 2 Vibration 3 Rest 3 1. st 1 min;12Hz 3 min 1 min;12Hz 3 min 1 min;15Hz 3 min 2. nd 1 min;15Hz 3 min 1 min;15Hz 3 min 2 min;15Hz 3 min 3. th 2 min;15Hz 3 min 3 min;15Hz 3 min 3 min;15Hz 3 min 4. th 2 min;24-25Hz 3 min 2 min;24-25Hz 3 min 2 min;24-25Hz 3 min >5th 3 min;24-25Hz 3 min 3 min;24-25Hz 3 min 3 min;24-25Hz 3 min The participants will perform mini-squats during Vibrations 1 and 3 and weight-shifting between right and left legs during Vibration 2 on the vibration platform under the supervision of a trained research assistant. | Device | Intervention group |
Primary outcome
North Star Ambulatory Assessment
Time frame: 4 weeks
examine the gross motor function of the participants. A summed score will be added from each test item.
2-minute Walk Test
Time frame: 4 weeks
assess submaximal exercise capacity by measuring the distance covered in the 2 minutes in metres
Segmental Assessment of Trunk Control-static
Time frame: 4 weeks
assess the segmental trunk control in sitting position with an ordinal score will be given in static trunk control. Assessment score represents as follows: 1= learning head control, 2= learning upper thoracic control, 3= learning mid-thoracic control, 4= learning lower thoracic control, 5= learning at upper lumber control, 6= learning lower lumbar control, 7= learning full trunk control and 8= achieved full trunk control.
Pediatric Evaluation of Disability Inventory
Time frame: 4 weeks
assess functional capacities in the domains of self care, mobility and social function with a summary score in each domain. A dichotomous score will be given to each question in each domain: 0= unable and 1= able. In self care domain, there are 73 questions, i.e. maximal score is 73. In mobility domain, there are 59 questions i.e. maximal score is 59. In social function domain, there are 65 questions i.e. maximal score is 65.
Body Height
Time frame: 4 weeks
measure height in cm
Body Weight
Time frame: 4 weeks
measure weight in kilograms
Body Mass Index
Time frame: 4 weeks
calculated based on body height and weight in terms of kg/m2
Bone Mineral Content of Femur
Time frame: 4 weeks
Distal femur BMC will be measured in grams
Bone Mineral Content of Whole Body (Excluding Head)
Time frame: 4 weeks
Whole body (excluding head) BMC will be measured in grams
Areal Bone Mineral Density of Femur
Time frame: 4 weeks
Areal bone mineral density of femur will be measured in grams/cm2
Areal Bone Mineral Density of Total Body (Excluding Head)
Time frame: 4 weeks
Areal bone mineral density of total body (excluding head) will be measured in grams/cm2
Volumetric Bone Mineral Density of Lumbar Spine
Time frame: 4 weeks
Volumetric bone mineral density of lumbar spine (L2 to L4) in grams/cm3
Range of Right Hip Flexion
Time frame: 4 weeks
measure hip flexion in supine using goniometer in degrees
Range of Left Hip Flexion
Time frame: 4 weeks
measure hip flexion in supine using goniometer in degrees
Range of Right Hip Extension
Time frame: 4 weeks
measure hip extension in prone using goniometer in degrees
Range of Left Hip Extension
Time frame: 4 weeks
measure hip extension in prone using goniometer in degrees
Range of Right Hip Abduction
Time frame: 4 weeks
measure hip abduction in supine using goniometer in degrees
Range of Left Hip Abduction
Time frame: 4 weeks
measure hip abduction in supine using goniometer in degrees
Range of Right Knee Flexion
Time frame: 4 weeks
measure knee flexion in prone using goniometer in degrees
Range of Left Knee Flexion
Time frame: 4 weeks
measure knee flexion in prone using goniometer in degrees
Range of Right Knee Extension
Time frame: 4 weeks
measure knee extension in sitting using goniometer in degrees
Range of Left Knee Extension
Time frame: 4 weeks
measure knee extension in sitting using goniometer in degrees
Range of Right Ankle Dorsiflexion
Time frame: 4 weeks
measure ankle dorsiflexion in sitting using goniometer in degrees
Range of Left Ankle Dorsiflexion
Time frame: 4 weeks
measure ankle dorsiflexion in sitting using goniometer in degrees
Range of Right Ankle Plantarflexion
Time frame: 4 weeks
measure ankle plantarflexion in sitting using goniometer in degrees
Range of Left Ankle Plantarflexion
Time frame: 4 weeks
measure ankle plantarflexion in sitting using goniometer in degrees
Muscle Strength of Right Hip Flexors
Time frame: 4 weeks
measure muscle strength of hip flexors in supine using dynamometer in terms of Newton
Muscle Strength of Left Hip Flexors
Time frame: 4 weeks
measure muscle strength of hip flexors in supine using dynamometer in terms of Newton
Muscle Strength of Right Hip Extensors
Time frame: 4 weeks
measure muscle strength of hip extensors in prone using dynamometer in terms of Newton
Muscle Strength of Left Hip Extensors
Time frame: 4 weeks
measure muscle strength of hip extensors in prone using dynamometer in terms of Newton
Muscle Strength of Right Knee Flexors
Time frame: 4 weeks
measure muscle strength of knee flexors in sitting using dynamometer in terms of Newton
Muscle Strength of Left Knee Flexors
Time frame: 4 weeks
measure muscle strength of knee flexors in sitting using dynamometer in terms of Newton
Muscle Strength of Right Knee Extensors
Time frame: 4 weeks
measure muscle strength of knee extensors in sitting using dynamometer in terms of Newton
Muscle Strength of Left Knee Extensors
Time frame: 4 weeks
measure muscle strength of knee extensors in sitting using dynamometer in terms of Newton
Muscle Strength of Right Hip Abductors
Time frame: 4 weeks
measure muscle strength of hip abductors in supine using dynamometer in terms of Newton
Muscle Strength of Left Hip Abductors
Time frame: 4 weeks
measure muscle strength of hip abductors in supine using dynamometer in terms of Newton
Muscle Strength of Right Ankle Dorsiflexors
Time frame: 4 weeks
measure muscle strength of ankle dorsiflexors in sitting using dynamometer in terms of Newton
Muscle Strength of Left Ankle Dorsiflexors
Time frame: 4 weeks
measure muscle strength of ankle dorsiflexors in sitting using dynamometer in terms of Newton
Muscle Strength of Right Ankle Plantarflexors
Time frame: 4 weeks
measure muscle strength of ankle plantarflexors in sitting using dynamometer in terms of Newton
Muscle Strength of Left Ankle Plantarflexors
Time frame: 4 weeks
measure muscle strength of ankle plantarflexors in sitting using dynamometer in terms of Newton
Segmental Assessment of Trunk Control_active
Time frame: 4 weeks
assess the segmental trunk control in sitting position with an ordinal score will be given in active trunk control. Assessment score represents as follows: 1= learning head control, 2= learning upper thoracic control, 3= learning mid-thoracic control, 4= learning lower thoracic control, 5= learning at upper lumber control, 6= learning lower lumbar control, 7= learning full trunk control and 8= achieved full trunk control.
Segmental Assessment of Trunk Control-reactive
Time frame: 4 weeks
assess the segmental trunk control in sitting position with an ordinal score will be given in reactive trunk control. Assessment score represents as follows: 1= learning head control, 2= learning upper thoracic control, 3= learning mid-thoracic control, 4= learning lower thoracic control, 5= learning at upper lumber control, 6= learning lower lumbar control, 7= learning full trunk control and 8= achieved full trunk control.
2 secondary outcomes
Percentage of Attendance of Participants
Time frame: 4 weeks
Visual Analogue Scale
Time frame: 4 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jul 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 24157400Vry J, Schubert IJ, Semler O, Haug V, Schonau E, Kirschner J. Whole-body vibration training in children with Duchenne muscular dystrophy and spinal muscular atrophy. Eur J Paediatr Neurol. 2014 Mar;18(2):140-9. doi: 10.1016/j.ejpn.2013.09.005. Epub 2013 Oct 11.
- Background: PubMed 15973316Henderson RC, Kairalla JA, Barrington JW, Abbas A, Stevenson RD. Longitudinal changes in bone density in children and adolescents with moderate to severe cerebral palsy. J Pediatr. 2005 Jun;146(6):769-75. doi: 10.1016/j.jpeds.2005.02.024.
- Background: PubMed 17111566Stevenson RD, Conaway M, Barrington JW, Cuthill SL, Worley G, Henderson RC. Fracture rate in children with cerebral palsy. Pediatr Rehabil. 2006 Oct-Dec;9(4):396-403. doi: 10.1080/13638490600668061.
- Background: PubMed 19614941Mergler S, Evenhuis HM, Boot AM, De Man SA, Bindels-De Heus KG, Huijbers WA, Penning C. Epidemiology of low bone mineral density and fractures in children with severe cerebral palsy: a systematic review. Dev Med Child Neurol. 2009 Oct;51(10):773-8. doi: 10.1111/j.1469-8749.2009.03384.x. Epub 2009 Jul 8.
- Background: PubMed 19643349Houlihan CM, Stevenson RD. Bone density in cerebral palsy. Phys Med Rehabil Clin N Am. 2009 Aug;20(3):493-508. doi: 10.1016/j.pmr.2009.04.004.
- Background: PubMed 12093986Henderson RC, Lark RK, Gurka MJ, Worley G, Fung EB, Conaway M, Stallings VA, Stevenson RD. Bone density and metabolism in children and adolescents with moderate to severe cerebral palsy. Pediatrics. 2002 Jul;110(1 Pt 1):e5. doi: 10.1542/peds.110.1.e5.
- Background: PubMed 10641693Larson CM, Henderson RC. Bone mineral density and fractures in boys with Duchenne muscular dystrophy. J Pediatr Orthop. 2000 Jan-Feb;20(1):71-4.
- Background: PubMed 19740225Rauch F. Vibration therapy. Dev Med Child Neurol. 2009 Oct;51 Suppl 4:166-8. doi: 10.1111/j.1469-8749.2009.03418.x.
- Background: PubMed 15903391Jordan MJ, Norris SR, Smith DJ, Herzog W. Vibration training: an overview of the area, training consequences, and future considerations. J Strength Cond Res. 2005 May;19(2):459-66. doi: 10.1519/13293.1.
- Background: PubMed 16903900Rehn B, Lidstrom J, Skoglund J, Lindstrom B. Effects on leg muscular performance from whole-body vibration exercise: a systematic review. Scand J Med Sci Sports. 2007 Feb;17(1):2-11. doi: 10.1111/j.1600-0838.2006.00578.x. Epub 2006 Aug 10.
- Background: PubMed 24388109Matute-Llorente A, Gonzalez-Aguero A, Gomez-Cabello A, Vicente-Rodriguez G, Casajus Mallen JA. Effect of whole-body vibration therapy on health-related physical fitness in children and adolescents with disabilities: a systematic review. J Adolesc Health. 2014 Apr;54(4):385-96. doi: 10.1016/j.jadohealth.2013.11.001. Epub 2014 Jan 1.
- Background: PubMed 24434887El-Shamy SM. Effect of whole-body vibration on muscle strength and balance in diplegic cerebral palsy: a randomized controlled trial. Am J Phys Med Rehabil. 2014 Feb;93(2):114-21. doi: 10.1097/PHM.0b013e3182a541a4.
- Farooq FT, Holcik M, MacKenzie A. Spinal Muscular Atrophy: Classification, Diagnosis, Background, Molecular Mechanism and Development of Therapeutics 2013.Link provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Tamis W Pin, PhD principal investigator
The Hong Kong Polytechnic University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing whole body vibration therapy in people with Spinal Muscular Atrophy Type 3, run by The Hong Kong Polytechnic University at 1 site in Hong Kong.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 26 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Hong Kong. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Jul 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
North Star Ambulatory Assessment (time frame: 4 weeks). The study lists 42 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03056144, which also lists 20 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jul 2019.