Congenital Muscle Disease Study of Patient and Family Reported Medical Information
Public ClinicalTrials.gov record NCT01403402. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Congenital Muscle Disease Patient and Proxy Reported Outcome Study
Study identification
- NCT ID
- NCT01403402
- Recruitment status
- Recruiting
- Study type
- Observational
- Phase
- Not listed
- Lead sponsor
- Cure CMD
- Other
- Enrollment
- 4,000 participants
Conditions and interventions
Conditions
- Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations)
- Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan and Epilepsy)
- Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan)
- Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy)
- Alpha-Dystroglycanopathy (Dystroglycanopathy, Congenital With or Without Mental Retardation (Formerly MDC1C))
- Alpha-Dystroglycanopathy (Fukuyama CMD)
- Alpha-Dystroglycanopathy (LGMDR09 FKRP Related (Formerly LGMD2I))
- Alpha-Dystroglycanopathy (LGMDR11 POMT1 Related (Formerly LGMD2K))
- Alpha-Dystroglycanopathy (LGMDR13 FKTN Related (Formerly LGMD2M))
- Alpha-Dystroglycanopathy (LGMDR14 POMT2 Related (Formerly LGMD2N))
- Alpha-Dystroglycanopathy (LGMDR15 POMGnT1 Related (Formerly LGMD2O))
- Alpha-Dystroglycanopathy (LGMDR19 GMPPB Related (Formerly LGMD2T))
- Alpha-Dystroglycanopathy (LGMDR20 ISPD Related (Formerly LGMD2U))
- Alpha-Dystroglycanopathy (LGMDR24 POMGnT2 Related)
- Alpha-Dystroglycanopathy (Muscle Eye Brain Disease (MEB))
- Alpha-Dystroglycanopathy (Walker Warburg Syndrome (WWS))
- Choline Kinase B Receptor - CHKB
- Collagen VI Related Disorders
- Collagen XII Related Disorders
- Congenital Muscular Dystrophy Not Otherwise Specified (Including Merosin Positive)
- Congenital Muscular Dystrophy With Cataracts and Intellectual Disability (MDCCAID)
- Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency
- Congenital Muscular Dystrophy With Joint Hyperlaxity
- Congenital Muscular Dystrophy With Rigid Spine Related to ACTA1
- Congenital Myasthenic Syndrome
- Emery-Dreifuss Muscular Dystrophy
- GOLGA2-related Congenital Muscle Dystrophy With Brain Involvement
- LGMDD01 - DNAJB6 (Formerly LGMD1D)
- LGMDD05 - Collagen VI Related Bethlem Myopathy (Dominant)
- LGMDR07 - Telethonin (TCAP) Related (Formerly LGMD2G)
- LGMDR08 - TRIM Related (Formerly LGMD2H)
- LGMDR09 - FKRP Related (Formerly LGMD2I)
- LGMDR10 - Titin (TTN) Related (Formerly LGMD2J)
- LGMDR11 - POMT1 Related (Formerly LGMD2K)
- LGMDR13 - Fukutin (FKTN) Related (Formerly LGMD2M)
- LGMDR14 - POMT2 Related (Formerly LGMD2N)
- LGMDR15 - POMGnT1 Related (Formerly LGMD2O)
- LGMDR16 - DAG1 Related Dystroglycanopathy (Formerly LGMD2P)
- LGMDR17 - Plectin (PLEC) Related (Formerly LGMD2Q)
- LGMDR18 - TRAPPC11 Related (Formerly LGMD2S)
- LGMDR19 - GMPPB Related (Formerly LGMD2T)
- LGMDR20 - ISPD Related (Formerly LGMD2U)
- LGMDR22 - Collagen VI Related Bethlem Myopathy (Recessive)
- LGMDR23 - LAMA2 Related
- LGMDR24 - POMGnT2 Related
- LMNA Related Disorders
- Limb-Girdle Muscular Dystrophy
- Merosin Deficient CMD (Full or Partial)
- Nesprin Related MD (SYNE1)
- SELENON Related Disorders (Previously Known as SEPN1)
- SELENON Related Myopathy (Aka SEPN1)
- Telethonin CMD
Interventions
Not listed
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Aug 31, 2009
- Primary completion
- Aug 31, 2029
- Completion
- Aug 31, 2029
- Last update posted
- Aug 8, 2021
2009 – 2029
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Congenital Muscle Disease International Registry (www.cmdir.org) | Lakewood | California | 90712 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01403402, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 8, 2021 · Synced May 20, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01403402 live on ClinicalTrials.gov.