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Congenital Myotonic Dystrophy clinical trials

View clinical trials related to Congenital Myotonic Dystrophy.

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NCT ID: NCT05224778 Recruiting - Clinical trials for Congenital Myotonic Dystrophy

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

ASPIRE-DM1
Start date: August 24, 2022
Phase:
Study type: Observational

The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and childhood myotonic dystrophy type 1, and develop biomarkers for the condition.

NCT ID: NCT05004129 Recruiting - Clinical trials for Congenital Myotonic Dystrophy

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

REACH CDM X
Start date: August 23, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.

NCT ID: NCT03059264 Recruiting - Clinical trials for Congenital Myotonic Dystrophy

Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy

TREAT-CDM
Start date: December 14, 2016
Phase:
Study type: Observational [Patient Registry]

Congenital Myotonic Dystrophy (CDM) is a multi-systemic, dominantly inherited disorder caused by a trinucleotide repeat expansion (CTGn) in the DMPK gene. CDM occurs when the CTGn increases between the adult myotonic dystrophy type-1 (DM1) parent and the child. Children with CDM present at birth with respiratory insufficiency, talipes equinovarus, feeding difficulties and hypotonia. There is a 30% mortality rate in the first year of life. As children grow, they are at risk for intellectual impairment, autistic features, gastrointestinal symptoms, and motor delay. The investigators will enroll children with CDM between ages 0-15 with visits at baseline and one year to evaluate appropriate physical functional outcomes, cognitive function and quality of life over time. Functional outcome measures will be correlated with potential biomarkers in the children. Completion of these specific aims will extend the understanding of disease progression in CDM and will provide the requisite information for successful therapeutic trials in children with DM.

NCT ID: NCT02398786 Recruiting - Clinical trials for Myotonic Dystrophy 1

Myotonic Dystrophy Family Registry

MDFR
Start date: February 2013
Phase:
Study type: Observational [Patient Registry]

The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) to aid researchers in developing new, effective treatments and help identify participants for research studies and clinical trials.

NCT ID: NCT00082108 Recruiting - Muscular Dystrophy Clinical Trials

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

Start date: September 2000
Phase:
Study type: Observational

Myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD) are inherited disorders characterized by progressive muscle weakness and loss of muscle tissue. The purpose of this registry is to connect people with DM or FSHD with researchers studying these diseases. The registry will offer individuals with DM and FSHD an opportunity to participate in research that focuses of their diseases. The registry will also help scientists to accomplish research on DM and FSHD and to distribute their findings to patients and care providers.