Clinical trialNCT04299321CompletedObservational

Retrospective Natural History Study of Infants and Toddlers With LAMA2-CMD

SponsorProthelia, Inc.Enrollment75Merosin Deficient Congenital Muscular Dystrophy

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

This retrospective chart review study of 75-120 LAMA2-CMD patients will expand the investigators understanding of the natural history of this disease. Current and pending publications cover research performed only in ages 5-16 years; there is currently no documented natural history for patients ages 0-5 years. Data collected in this study has the potential to inform the design of future interventional studies that draw nearer to clinical trial readiness every day.

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Alan Beggs, PhD
Boston Children's Hospital
Study Director
clinicaltrials.gov ↗
Carsten Bönnemann, MD
NINDS/NIH
Study Director
clinicaltrials.gov ↗
Gustavo Dziewczapolski, PhD
Cure CMD
Principal Investigator
clinicaltrials.gov ↗
Oscar H Mayer, MD
Children's Hospital of Philadelphia
Study Director
clinicaltrials.gov ↗
Rachel Alvarez, BS
Cure CMD
Study Director
clinicaltrials.gov ↗
Reghan Foley, MD
NINDS/NIH
Study Director
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

1 registered facility, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

Cure Cmd, Inc.
Lakewood, California, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study — it may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 24 Apr 2020
Primary completion: 31 Dec 2021
Completion: 31 Dec 2021
Last update posted: 7 Feb 2022
Registered enrollment: 75
Registry id: NCT04299321

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.