Trial · NCT07201636
Natural History Study for Patients With Nemaline Myopathy in Belgium
NCT07201636, Not applicable, Not yet recruiting, Centre Hospitalier Universitaire de Liege
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Natural History Study for Patients With Nemaline Myopathy in Belgium. Trials, record NCT07201636. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07201636
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Natural History Study for Patients With Nemaline Myopathy in Belgium
- NCT number
- NCT07201636
- Status
- Not yet recruiting
- Phases
- Not applicable
- Enrollment
- 10 participants, estimated
- Lead sponsor
- Centre Hospitalier Universitaire de Liege
- Conditions
- Nemaline Myopathy
- Interventions
- Motor Function Assessments, Syde, Respiratory Assessments, Bulbar Function Questionnaires, Clinical Global Impression (CGI), Quality of Life Questionnaires, Blood Sample Collection
- First posted
- 2025-10-01
- Last updated
- 2026-03-25
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Motor Function Measure (MFM32) Score | Over 3 years | Difference in MFM32 score from baseline to each follow-up visit. The MFM32 is a validated scale (range 0-96; higher scores indicate better motor function). |
| Primary | Change in Ventilation Hours per Day | Over 3 years | Evolution of the number of hours of assisted ventilation per day compared to baseline. |
| Secondary | Change in Quality of Life Score (PROMIS-29 Profile v2.1) | Over 3 years | Difference in PROMIS-29 Profile v2.1 score from baseline to follow-up visits. PROMIS-29 includes 29 items across 7 domains (physical function, anxiety, depression, fatigue, sleep disturbance, social roles, pain interference) plus pain intensity. Higher T-scores indicate more of the concept being measured (e.g., higher fatigue score = more fatigue). |
| Secondary | Drop-out Rate | Over 3 years | Percentage of participants who discontinue participation before study completion. |
| Other | Change in Health Utilities Index 3 (HUI3) Score | Over 3 years | Difference in HUI3 score from baseline to follow-up visits (range -0.36 to 1.00; higher scores indicate better health status). |
| Other | Distance Walked per Hour | 6 recording periods of 1 month over 3 years | Average distance walked per hour measured by wearable sensors. |
| Other | Stride Speed Variability | 6 recording periods of 1 month over 3 years | Coefficient of variation of stride speed measured by wearable sensors. |
| Other | Number of Falls | 6 recording periods of 1 month over 3 years | Number of falls recorded by wearable sensors during continuous monitoring in ambulant patients. |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT07201636
GET https://conductscience.org/api/trials/v1/NCT07201636/registered