Myotonic Dystrophy 1CABLAMYDNCT04712422
Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1
An observational study of Myotonic Dystrophy 1, run by Istituto Auxologico Italiano at 2 sites in Italy.
Trial summary
Impairment of balance and gait are frequent complaints in patients with myotonic dystrophy type 1 (DM1). In these persons, there is an increased risk for stumbles and falls when compared to normal subjects. An underestimated cause of falls might be the weakness of neck flexor muscles (due to cervical ataxia). Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Myotonic Dystrophy 1
- What is being tested?
- Healthy subjects and Pathologic group
- How many people
- 42 participants
- Where
- 2 sitesItaly
- Study dates
- 27 Oct 2020to 31 Dec 2022
- Phase
- Observational
- 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 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Study population: The participants will be recruited in the NeuroMuscolar Omniservice (NEMO) clinical centre, on an outpatient basis, from a physiatric referral, or from an inpatient rehabilitation unit.
Who may be able to join
- Diagnosis of DM1 since at least 5 years;
- Ability to stand erect with open eyes for at least 20 s;
- Rivermead Mobility Index (RMI) score ≥ 10/15;
- Visual acuity > 10/20, also with corrective lenses;
- Mini Mental State Examination (MMSE) score ≥ 26/30;
- ability to wittingly sign the informed consent form.
Who cannot join
- neurological or orthopedic pathologies with impact on balance;
- pregnancy;
- previous orthopedic surgical intervention;
- head or neck trauma in the 6 months preceding the study;
- other pathological conditions which could alter balance;
- drug therapy, underway for less than one month before the study, with impact on balance.
Trial locations and contacts
2 sites in 1 country.
Italy2 sites
Istituto Auxologico Italiano
Milan, MI, 20121
The NEuroMuscular Omnicentre (NEMO) Clinical Center
Milan, MI, 20162
About this study
Impairment of balance and gait are frequent complaints in patients with myotonic dystrophy type 1 (DM1). In these persons, there is an increased risk for stumbles and falls when compared to normal subjects. An underestimated cause of falls might be the weakness of neck flexor muscles (due to cervical ataxia). It is well known that fibres of muscle spindles are receptors combining a specialized sub-set of muscle fibers with a specialized array of both sensory and motor nerve fibers. Spindles transduce into neural afferent discharges the muscle length and length changes. They are very dense in deep neck muscles, are crucial to body balance and gage orientation, and are severely affected in DM1. Preliminary results suggest that falls could reflect imbalance. These indicate that cervical ataxia may come into play because of muscle spindle fibre disruption. In light of the current knowledge on the physiology of balance and on the association between balance deficits and cervical dystonia in other clinical conditions (e.g., whiplash injury), a rationale is therefore offered to a confirmation of the hypothesis that DM1 patients may suffer from cervical ataxia.
The primary endpoint is the demonstration of an association between balance deficits in standing and cervical proprioception deficit in adults affected by Myotonic dystrophy 1.
Read more
Secondary endpoints are:
- the investigation of the correlation among the two deficits and the clinical conditions of patients,
- the definition of normative data in the measure of cervical proprioception in a sample of healthy participants.
It is expected that high scores in postural balance, obtained on the posturographic Equitest™-Sensory Organization Test-SOT, correspond to high levels of repositioning accuracy in tests of cervical repositioning and low SOT scores correspond to low accuracy. Moreover, it is expected that an association exists among the two deficits and the clinical situation of the patients. Results from the present pilot study will allow an estimate of the sample size for future experimental protocols. The evidence for an association between balance deficits and cervical ataxia would be of obvious relevance to the patients. This would also support the hypothesis that neck muscle spindles may be especially affected in DM1. This would highlight that muscles are also crucial sensory organs, involved in the perception of joint position, muscle strength, and fatigue. Results from the present study might allow the definition of new rehabilitative programs, such as treatments through a neck strengthening (and thus stiffening) exercise program. This study, therefore, might stimulate new research hypothesis at the neurophysiologic level and possibly lead to findings generalizable from DM1 to other forms of myopathy.
- Conditions
- Myotonic Dystrophy 1
- Being tested
- Healthy subjects, Pathologic group
- Keywords
- Myotonic Dystrophy 1, Accidental falls, Ataxia, Postural balance, Rehabilitation
- Medical subject headings
- Myotonic Dystrophy, Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Healthy subjectsOther
Participants will be tested for their foot dominance by means of the Waterloo footedness questionnaire-revised, their hand dominance by means of the Edinburgh inventory, and their eyedness Coren's Lateral Preference Inventory.
Participants will perform a cervical repositioning test. They will seat in a chair in front of a Plexiglas screen with the eye closed. The operator will guide the participant in four positions: at 30° right/left rotation and at 25° extension/flexion. The participant will be then asked to reproduce the angle. Each movement will be repeated four times in a random order. The whole sequence will be repeated by another second operator. Both the tests will be repeated after two weeks.
Head movements will be detected using an optoelectronic system using passive markers positioned on the head of the participant.
Pathologic groupOther
Clinical evaluation of the participants will be performed by means of the Myotonic Dystrophy Health Index (MDHI), the Rivermead Mobility Index (RMI), the Fall Events Questionnaire, and the Dizziness Handicap Inventory- short form (DHIsf).
Participants will perform a cervical repositioning test. Participants will seat in a chair in front of a Plexiglas screen with the eye closed. The operator will guide the participant in four positions: at 30° right/left rotation and at 25° extension/flexion. The participant will be then asked to reproduce the angle. Each movement will be repeated four times in a random order.
Head movements will be detected using an optoelectronic system using passive markers positioned on the head of the participant.
Participants will then perform test of balance in standing, using the EquiTest platform. Individuals will be requested to perform three different tasks: sensory organization test, adaptation test-upward tilt, and adaptation test-downward tilt.
| Study group | Role | What they receive |
|---|---|---|
| Healthy participants | At least 25 healthy participants aged from 18 to 50 years old. Participants will be excluded if pregnant. Receives: Other: Healthy subjects | |
| Pathologic group | At least 22 participants with diagnosis of Myotonic Dystrophy 1. Receives: Other: Pathologic group |
What is being measured
The primary outcome is the main question the study is designed to answer.
SOT score
The patient's task is to maintain an upright stance during 3 20 s trials under six different conditions, including platform and visual surround 'tuned' with individual's sagittal oscillation. The SOT score will be calculated by comparing the sagittal oscillation of the body's centre of mass (COM) to the maximal sagittal oscillation. Score is averaged across the six conditions (range 0 - 100 the higher the score, the lower the oscillation).
Measured: Day 1
Secondary outcomes (1)
Head Repositioning Accuracy, HRA
The head repositioning accuracy in percentage will be computed as the joint position error (JPE) divided by the target position. The JPE will be computed as the absolute difference between the target position and the measured position.
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2021
- Study start27 Oct 2020
- Primary completion13 Oct 2021
- Study completion31 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (16)Papers the sponsor has linked to this study.
- Other trials for Myotonic Dystrophy 1Studies of the same condition, open and under way.
- More trials run by Istituto Auxologico ItalianoThe 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
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- Istituto Auxologico Italiano
- Sponsor type
- University, hospital or other
- Collaborators
- Fondazione Serena Onlus - Centro Clinico NeMO Milano
- Responsible party
- Sponsor
- Investigators
- Luigi Tesio, MD, Full professor, Principal investigator, Istituto Auxologico Italiano
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04712422
- Sponsor's protocol ID
- 24C020
- Acronym
- CABLAMYD
- Status last verified
- Feb 2024
- First submitted
- 13 Jan 2021
- Last updated
- 20 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Publications
Galli M, Cimolin V, Crugnola V, Priano L, Menegoni F, Trotti C, Milano E, Mauro A. Gait pattern in myotonic dystrophy (Steinert disease): a kinematic, kinetic and EMG evaluation using 3D gait analysis. J Neurol Sci. 2012 Mar 15;314(1-2):83-7. doi: 10.1016/j.jns.2011.10.026. Epub 2011 Nov 25.
PubMed 22118863Jimenez-Moreno AC, Raaphorst J, Babacic H, Wood L, van Engelen B, Lochmuller H, Schoser B, Wenninger S. Falls and resulting fractures in Myotonic Dystrophy: Results from a multinational retrospective survey. Neuromuscul Disord. 2018 Mar;28(3):229-235. doi: 10.1016/j.nmd.2017.12.010. Epub 2017 Dec 27.
PubMed 29361394Pieterse AJ, Luttikhold TB, de Laat K, Bloem BR, van Engelen BG, Munneke M. Falls in patients with neuromuscular disorders. J Neurol Sci. 2006 Dec 21;251(1-2):87-90. doi: 10.1016/j.jns.2006.09.008. Epub 2006 Nov 9.
PubMed 17097113Hammaren E, Kjellby-Wendt G, Kowalski J, Lindberg C. Factors of importance for dynamic balance impairment and frequency of falls in individuals with myotonic dystrophy type 1 - a cross-sectional study - including reference values of Timed Up & Go, 10m walk and step test. Neuromuscul Disord. 2014 Mar;24(3):207-15. doi: 10.1016/j.nmd.2013.12.003. Epub 2013 Dec 15.
PubMed 24412157Proske U, Gandevia SC. The proprioceptive senses: their roles in signaling body shape, body position and movement, and muscle force. Physiol Rev. 2012 Oct;92(4):1651-97. doi: 10.1152/physrev.00048.2011.
PubMed 23073629Sansone VA, Lizio A, Greco L, Gragnano G, Zanolini A, Gualandris M, Iatomasi M, Heatwole C. The Myotonic Dystrophy Health Index: Italian validation of a disease-specific outcome measure. Neuromuscul Disord. 2017 Nov;27(11):1047-1053. doi: 10.1016/j.nmd.2017.07.004. Epub 2017 Jul 10.
PubMed 28890289Tesio L, Alpini D, Cesarani A, Perucca L. Short form of the Dizziness Handicap Inventory: construction and validation through Rasch analysis. Am J Phys Med Rehabil. 1999 May-Jun;78(3):233-41. doi: 10.1097/00002060-199905000-00009.
PubMed 10340421Tesio L, Rota V, Longo S, Grzeda MT. Measuring standing balance in adults: reliability and minimal real difference of 14 instrumental measures. Int J Rehabil Res. 2013 Dec;36(4):362-74. doi: 10.1097/MRR.0000000000000037.
PubMed 24185405Beinert K, Taube W. The effect of balance training on cervical sensorimotor function and neck pain. J Mot Behav. 2013;45(3):271-8. doi: 10.1080/00222895.2013.785928.
PubMed 23663191Loudon JK, Ruhl M, Field E. Ability to reproduce head position after whiplash injury. Spine (Phila Pa 1976). 1997 Apr 15;22(8):865-8. doi: 10.1097/00007632-199704150-00008.
PubMed 9127919Grip H, Sundelin G, Gerdle B, Karlsson JS. Variations in the axis of motion during head repositioning--a comparison of subjects with whiplash-associated disorders or non-specific neck pain and healthy controls. Clin Biomech (Bristol). 2007 Oct;22(8):865-73. doi: 10.1016/j.clinbiomech.2007.05.008. Epub 2007 Jul 9.
PubMed 17619066Ferrario VF, Sforza C, Serrao G, Grassi G, Mossi E. Active range of motion of the head and cervical spine: a three-dimensional investigation in healthy young adults. J Orthop Res. 2002 Jan;20(1):122-9. doi: 10.1016/S0736-0266(01)00079-1.
PubMed 11853078Mortensen JD, Vasavada AN, Merryweather AS. The inclusion of hyoid muscles improve moment generating capacity and dynamic simulations in musculoskeletal models of the head and neck. PLoS One. 2018 Jun 28;13(6):e0199912. doi: 10.1371/journal.pone.0199912. eCollection 2018.
PubMed 29953539Vasavada AN, Hughes E, Nevins DD, Monda SM, Lin DC. Effect of Subject-Specific Vertebral Position and Head and Neck Size on Calculation of Spine Musculoskeletal Moments. Ann Biomed Eng. 2018 Nov;46(11):1844-1856. doi: 10.1007/s10439-018-2084-9. Epub 2018 Jul 9.
PubMed 29987540Vasavada AN, Li S, Delp SL. Influence of muscle morphometry and moment arms on the moment-generating capacity of human neck muscles. Spine (Phila Pa 1976). 1998 Feb 15;23(4):412-22. doi: 10.1097/00007632-199802150-00002.
PubMed 9516695
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1
- ClinicalTrials.gov ID
- NCT04712422
- Acronym / sponsor ID
- CABLAMYD / 24C020
- Conditions
- Myotonic Dystrophy 1
- Keywords
- Myotonic Dystrophy 1, Accidental falls, Ataxia, Postural balance, Rehabilitation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- Istituto Auxologico ItalianoUniversity, hospital or other
- Collaborators
- Fondazione Serena Onlus - Centro Clinico NeMO Milano
- Study start
- 27 Oct 2020 (actual)
- Primary completion
- 13 Oct 2021 (actual)
- Study completion
- 31 Dec 2022 (actual)
- First posted
- 15 Jan 2021 (actual)
- 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.
Impairment of balance and gait are frequent complaints in patients with myotonic dystrophy type 1 (DM1). In these persons, there is an increased risk for stumbles and falls when compared to normal subjects. An underestimated cause of falls might be the weakness of neck flexor muscles (due to cervical ataxia). It is well known that fibres of muscle spindles are receptors combining a specialized sub-set of muscle fibers with a specialized array of both sensory and motor nerve fibers. Spindles transduce into neural afferent discharges the muscle length and length changes. They are very dense in deep neck muscles, are crucial to body balance and gage orientation, and are severely affected in DM1. Preliminary results suggest that falls could reflect imbalance. These indicate that cervical ataxia may come into play because of muscle spindle fibre disruption. In light of the current knowledge on the physiology of balance and on the association between balance deficits and cervical dystonia in other clinical conditions (e.g., whiplash injury), a rationale is therefore offered to a confirmation of the hypothesis that DM1 patients may suffer from cervical ataxia.
The primary endpoint is the demonstration of an association between balance deficits in standing and cervical proprioception deficit in adults affected by Myotonic dystrophy 1.
Secondary endpoints are:
- the investigation of the correlation among the two deficits and the clinical conditions of patients,
- the definition of normative data in the measure of cervical proprioception in a sample of healthy participants.
It is expected that high scores in postural balance, obtained on the posturographic Equitest™-Sensory Organization Test-SOT, correspond to high levels of repositioning accuracy in tests of cervical repositioning and low SOT scores correspond to low accuracy. Moreover, it is expected that an association exists among the two deficits and the clinical situation of the patients. Results from the present pilot study will allow an estimate of the sample size for future experimental protocols. The evidence for an association between balance deficits and cervical ataxia would be of obvious relevance to the patients. This would also support the hypothesis that neck muscle spindles may be especially affected in DM1. This would highlight that muscles are also crucial sensory organs, involved in the perception of joint position, muscle strength, and fatigue. Results from the present study might allow the definition of new rehabilitative programs, such as treatments through a neck strengthening (and thus stiffening) exercise program. This study, therefore, might stimulate new research hypothesis at the neurophysiologic level and possibly lead to findings generalizable from DM1 to other forms of myopathy.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Healthy participants | – | Other: Healthy subjectsAt least 25 healthy participants aged from 18 to 50 years old. Participants will be excluded if pregnant. |
| Pathologic group | – | Other: Pathologic groupAt least 22 participants with diagnosis of Myotonic Dystrophy 1. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- The participants will be recruited in the NeuroMuscolar Omniservice (NEMO) clinical centre, on an outpatient basis, from a physiatric referral, or from an inpatient rehabilitation unit.
Key inclusion criteria (6 listed)
- Diagnosis of DM1 since at least 5 years;
- Ability to stand erect with open eyes for at least 20 s;
- Rivermead Mobility Index (RMI) score ≥ 10/15;
- Visual acuity > 10/20, also with corrective lenses;
- Mini Mental State Examination (MMSE) score ≥ 26/30;
Key exclusion criteria (6 listed)
- neurological or orthopedic pathologies with impact on balance;
- pregnancy;
- previous orthopedic surgical intervention;
- head or neck trauma in the 6 months preceding the study;
- other pathological conditions which could alter balance;
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 |
|---|---|---|
| Healthy subjectsParticipants will be tested for their foot dominance by means of the Waterloo footedness questionnaire-revised, their hand dominance by means of the Edinburgh inventory, and their eyedness Coren's Lateral Preference Inventory. Participants will perform a cervical repositioning test. They will seat in a chair in front of a Plexiglas screen with the eye closed. The operator will guide the participant in four positions: at 30° right/left rotation and at 25° extension/flexion. The participant will be then asked to reproduce the angle. Each movement will be repeated four times in a random order. The whole sequence will be repeated by another second operator. Both the tests will be repeated after two weeks. Head movements will be detected using an optoelectronic system using passive markers positioned on the head of the participant. | Other | Healthy participants |
| Pathologic groupClinical evaluation of the participants will be performed by means of the Myotonic Dystrophy Health Index (MDHI), the Rivermead Mobility Index (RMI), the Fall Events Questionnaire, and the Dizziness Handicap Inventory- short form (DHIsf). Participants will perform a cervical repositioning test. Participants will seat in a chair in front of a Plexiglas screen with the eye closed. The operator will guide the participant in four positions: at 30° right/left rotation and at 25° extension/flexion. The participant will be then asked to reproduce the angle. Each movement will be repeated four times in a random order. Head movements will be detected using an optoelectronic system using passive markers positioned on the head of the participant. Participants will then perform test of balance in standing, using the EquiTest platform. Individuals will be requested to perform three different tasks: sensory organization test, adaptation test-upward tilt, and adaptation test-downward tilt. | Other | Pathologic group |
Primary outcome
SOT score
Time frame: Day 1
The patient's task is to maintain an upright stance during 3 20 s trials under six different conditions, including platform and visual surround 'tuned' with individual's sagittal oscillation. The SOT score will be calculated by comparing the sagittal oscillation of the body's centre of mass (COM) to the maximal sagittal oscillation. Score is averaged across the six conditions (range 0 - 100 the higher the score, the lower the oscillation).
1 secondary outcome
Head Repositioning Accuracy, HRA
Time frame: Day 1
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 20 Feb 2024.
- Background: PubMed 22118863Galli M, Cimolin V, Crugnola V, Priano L, Menegoni F, Trotti C, Milano E, Mauro A. Gait pattern in myotonic dystrophy (Steinert disease): a kinematic, kinetic and EMG evaluation using 3D gait analysis. J Neurol Sci. 2012 Mar 15;314(1-2):83-7. doi: 10.1016/j.jns.2011.10.026. Epub 2011 Nov 25.
- Background: PubMed 29361394Jimenez-Moreno AC, Raaphorst J, Babacic H, Wood L, van Engelen B, Lochmuller H, Schoser B, Wenninger S. Falls and resulting fractures in Myotonic Dystrophy: Results from a multinational retrospective survey. Neuromuscul Disord. 2018 Mar;28(3):229-235. doi: 10.1016/j.nmd.2017.12.010. Epub 2017 Dec 27.
- Background: PubMed 17097113Pieterse AJ, Luttikhold TB, de Laat K, Bloem BR, van Engelen BG, Munneke M. Falls in patients with neuromuscular disorders. J Neurol Sci. 2006 Dec 21;251(1-2):87-90. doi: 10.1016/j.jns.2006.09.008. Epub 2006 Nov 9.
- Background: PubMed 24412157Hammaren E, Kjellby-Wendt G, Kowalski J, Lindberg C. Factors of importance for dynamic balance impairment and frequency of falls in individuals with myotonic dystrophy type 1 - a cross-sectional study - including reference values of Timed Up & Go, 10m walk and step test. Neuromuscul Disord. 2014 Mar;24(3):207-15. doi: 10.1016/j.nmd.2013.12.003. Epub 2013 Dec 15.
- Background: PubMed 23073629Proske U, Gandevia SC. The proprioceptive senses: their roles in signaling body shape, body position and movement, and muscle force. Physiol Rev. 2012 Oct;92(4):1651-97. doi: 10.1152/physrev.00048.2011.
- Background: PubMed 28890289Sansone VA, Lizio A, Greco L, Gragnano G, Zanolini A, Gualandris M, Iatomasi M, Heatwole C. The Myotonic Dystrophy Health Index: Italian validation of a disease-specific outcome measure. Neuromuscul Disord. 2017 Nov;27(11):1047-1053. doi: 10.1016/j.nmd.2017.07.004. Epub 2017 Jul 10.
- Background: PubMed 10340421Tesio L, Alpini D, Cesarani A, Perucca L. Short form of the Dizziness Handicap Inventory: construction and validation through Rasch analysis. Am J Phys Med Rehabil. 1999 May-Jun;78(3):233-41. doi: 10.1097/00002060-199905000-00009.
- Background: PubMed 24185405Tesio L, Rota V, Longo S, Grzeda MT. Measuring standing balance in adults: reliability and minimal real difference of 14 instrumental measures. Int J Rehabil Res. 2013 Dec;36(4):362-74. doi: 10.1097/MRR.0000000000000037.
- Background: PubMed 23663191Beinert K, Taube W. The effect of balance training on cervical sensorimotor function and neck pain. J Mot Behav. 2013;45(3):271-8. doi: 10.1080/00222895.2013.785928.
- Background: PubMed 9127919Loudon JK, Ruhl M, Field E. Ability to reproduce head position after whiplash injury. Spine (Phila Pa 1976). 1997 Apr 15;22(8):865-8. doi: 10.1097/00007632-199704150-00008.
- Background: PubMed 17619066Grip H, Sundelin G, Gerdle B, Karlsson JS. Variations in the axis of motion during head repositioning--a comparison of subjects with whiplash-associated disorders or non-specific neck pain and healthy controls. Clin Biomech (Bristol). 2007 Oct;22(8):865-73. doi: 10.1016/j.clinbiomech.2007.05.008. Epub 2007 Jul 9.
- Background: PubMed 11853078Ferrario VF, Sforza C, Serrao G, Grassi G, Mossi E. Active range of motion of the head and cervical spine: a three-dimensional investigation in healthy young adults. J Orthop Res. 2002 Jan;20(1):122-9. doi: 10.1016/S0736-0266(01)00079-1.
- Individual participant dataSharing planned: Yes
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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.
Luigi Tesio, MD, Full professor principal investigator
Istituto Auxologico Italiano
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myotonic Dystrophy 1, run by Istituto Auxologico Italiano at 2 sites in Italy.
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 6 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 20 Feb 2024. 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 Italy. 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?
SOT score (time frame: Day 1). 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 NCT04712422, which also lists 16 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 20 Feb 2024.