Neurofibromatosis Type 1NCT01058330
Effects of Physical Training on Bone and Muscle Quality, Muscle Strength, and Motor Coordination in Children With NF1
A study testing Plyometric training program in people with Neurofibromatosis Type 1, run by Shriners Hospitals for Children at 2 sites in United States.
Trial summary
A physical training program will improve quality of life, participation in physical activity, motor coordination, muscle strength, and bone and muscle strength in children with neurofibromatosis type 1. Read more about this study
- Who is it for?
- Ages 4 to 19 years
- Conditions the study is for
- Neurofibromatosis Type 1
- What is being tested?
- Plyometric training program
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 2 sitesUnited States
- Study dates
- Feb 2010to Feb 2014
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 4 to 19 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Fulfill NIH clinical diagnostic criteria
Who cannot join
- Visual impairment
- Participation in a simultaneous medical intervention trial
- Orthopedic procedure within the last 6 months.
- Pregnancy
- Home location greater than 3-4 hours drive time from Shriners Hospital
- Tibial pseudarthrosis
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Shriners Hospitals for Children, Salt Lake City
Salt Lake City, Utah, 84103
University of Utah
Salt Lake City, Utah, 84132
About this study
Official title: Effects of Physical Training on Bone and Muscle Quality, Muscle Strength, and Motor Coordination in Children With Neurofibromatosis Type 1
A physical training program will improve quality of life, participation in physical activity, motor coordination, muscle strength, and bone and muscle strength in children with neurofibromatosis type 1.
Read the detailed description
Disorders of the Ras pathway have significant phenotypic overlap and include Noonan syndrome, Cardiofaciocutaneous syndrome (CFC syndrome), Legius syndrome, Costello syndrome and neurofibromatosis type 1 (NF1). NF1 is one of the most common genetic disorders presenting in childhood with an incidence of 1/3000. NF1 is associated with skeletal abnormalities such as short stature, scoliosis, and long bone fracture with non-union. We recently reported that children with NF1 have abnormalities of bone and muscle architecture as evidenced by decreased bone mineral density, decreased bone strength, and low muscle mass, all of which may predispose them to fractures and scoliosis (Stevenson et al., 2005, 2007, 2009). Our preliminary data show that children with NF1 have poor motor coordination and muscle strength, potentially secondary to abnormal neuromotor learning. We hypothesize that poor motor coordination and decreased muscle strength contribute to the osteopenia in NF1. Our objective is to identify effective and non-invasive strategies to improve motor coordination, muscle strength, and bone and muscle architecture in children with disorders of the Ras pathway, in hopes of decreasing fractures and improving physical activity levels. Plyometric physical training consists of quick, high-intensity, weight-bearing movements, and is an encouraging intervention for use in these children.
- Conditions
- Neurofibromatosis Type 1
- Being tested
- Plyometric training program
- Keywords
- Neurofibromatosis type 1, NF1
- Medical subject headings
- Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Plyometric training programOther
The intervention is a year long individualized plyometric exercise training program. Examples of plyometric activities include jumping, hopping, running, and throwing. The number of plyometric exercises will increase gradually to prevent over training to a total of five lower extremity exercises and five upper extremity exercises
| Study group | Role | What they receive |
|---|---|---|
| Plyometric physical training | Active comparator | Individualized plyometric training program to increase strength, coordination, and bone density. Receives: Other: Plyometric training program |
| Control Group | No intervention | This group will have no intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bone & muscle quality, DXA, pQCT, & bone ultrasound.
Measured: 1 year
Secondary outcomes (1)
Motor proficiency BOT-2. Muscle strength force plate & dynamometer. Quality of life questionnaires.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2010 (estimated)
- Study startFeb 2010
- Primary completionFeb 2014
- Study completionFeb 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- 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
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shriners Hospitals for Children
- Sponsor type
- University, hospital or other
- Collaborators
- Thrasher Research Fund, University of Utah
- Responsible party
- Principal investigator, David Stevenson, MD, Shriners Hospitals for Children
- Investigators
- David Stevenson, MD, Principal investigator, Shriners Hospitals for Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01058330
- Sponsor's protocol ID
- SLC00038711
- Status last verified
- Mar 2021
- First submitted
- 26 Jan 2010
- Last updated
- 23 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Physical Training on Bone and Muscle Quality, Muscle Strength, and Motor Coordination in Children With Neurofibromatosis Type 1
- ClinicalTrials.gov ID
- NCT01058330
- Acronym / sponsor ID
- SLC00038711
- Conditions
- Neurofibromatosis Type 1
- Keywords
- Neurofibromatosis type 1, NF1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Shriners Hospitals for ChildrenUniversity, hospital or other
- Collaborators
- Thrasher Research Fund, University of Utah
- Study start
- Feb 2010
- Primary completion
- Feb 2014 (actual)
- Study completion
- Feb 2014 (actual)
- First posted
- 28 Jan 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A physical training program will improve quality of life, participation in physical activity, motor coordination, muscle strength, and bone and muscle strength in children with neurofibromatosis type 1.
Detailed description
Disorders of the Ras pathway have significant phenotypic overlap and include Noonan syndrome, Cardiofaciocutaneous syndrome (CFC syndrome), Legius syndrome, Costello syndrome and neurofibromatosis type 1 (NF1). NF1 is one of the most common genetic disorders presenting in childhood with an incidence of 1/3000. NF1 is associated with skeletal abnormalities such as short stature, scoliosis, and long bone fracture with non-union. We recently reported that children with NF1 have abnormalities of bone and muscle architecture as evidenced by decreased bone mineral density, decreased bone strength, and low muscle mass, all of which may predispose them to fractures and scoliosis (Stevenson et al., 2005, 2007, 2009). Our preliminary data show that children with NF1 have poor motor coordination and muscle strength, potentially secondary to abnormal neuromotor learning. We hypothesize that poor motor coordination and decreased muscle strength contribute to the osteopenia in NF1. Our objective is to identify effective and non-invasive strategies to improve motor coordination, muscle strength, and bone and muscle architecture in children with disorders of the Ras pathway, in hopes of decreasing fractures and improving physical activity levels. Plyometric physical training consists of quick, high-intensity, weight-bearing movements, and is an encouraging intervention for use in these children.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Plyometric physical training | Active comparator | Other: Plyometric training programIndividualized plyometric training program to increase strength, coordination, and bone density. |
| Control Group | No intervention | –This group will have no intervention |
Who may qualify
- Ages
- 4 to 19 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Fulfill NIH clinical diagnostic criteria
Key exclusion criteria (6 listed)
- Visual impairment
- Participation in a simultaneous medical intervention trial
- Orthopedic procedure within the last 6 months.
- Pregnancy
- Home location greater than 3-4 hours drive time from Shriners Hospital
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 |
|---|---|---|
| Plyometric training programThe intervention is a year long individualized plyometric exercise training program. Examples of plyometric activities include jumping, hopping, running, and throwing. The number of plyometric exercises will increase gradually to prevent over training to a total of five lower extremity exercises and five upper extremity exercises | Other | Plyometric physical training |
Primary outcome
Bone & muscle quality, DXA, pQCT, & bone ultrasound.
Time frame: 1 year
1 secondary outcome
Motor proficiency BOT-2. Muscle strength force plate & dynamometer. Quality of life questionnaires.
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2021.
- 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.
David Stevenson, MD principal investigator
Shriners Hospitals for Children
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Plyometric training program in people with Neurofibromatosis Type 1, run by Shriners Hospitals for Children at 2 sites in United States.
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 1 inclusion and 6 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: finished, as of the sponsor's last update on 23 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Bone & muscle quality, DXA, pQCT, & bone ultrasound. (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Mar 2021.