Who is sponsoring NCT04299321?

NCT04299321 is sponsored by Prothelia, Inc..

What condition does NCT04299321 study?

NCT04299321 studies Merosin Deficient Congenital Muscular Dystrophy.

Is NCT04299321 still recruiting?

NCT04299321 is currently completed. Last updated 7 February 2022.

Last reviewed 2022-02-07 · How we verify

NCT04299321: LAMA2 rNHS

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

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

Completed Last updated 7 February 2022

What this trial tests

trial in Merosin Deficient Congenital Muscular Dystrophy in 75 participants. Completed in 31 December 2021.

Timeline

24 April 2020

Primary endpoint
31 December 2021

31 December 2021

Quick facts

Lead sponsor	Prothelia, Inc.
Status	Completed
Study type	OBSERVATIONAL
Enrollment	75
Start date	24 April 2020
Primary completion	31 December 2021
Estimated completion	31 December 2021
Sites	1 location across United States

Conditions studied

Merosin Deficient Congenital Muscular Dystrophy — all drugs for Merosin Deficient Congenital Muscular Dystrophy →

Sponsor

Prothelia, Inc.

Who can join

Adults 2 to 21, any sex, with Merosin Deficient Congenital Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

An international retrospective early natural history study of LAMA2-related dystrophies.
Hinkley L, Orbach R, Park J, Alvarez R, et al · · 2024 · cited 5× · PMID 39177609 · DOI 10.3233/jnd-240048

Verify or expand the search:

Other recruiting trials for Merosin Deficient Congenital Muscular Dystrophy

Currently open trials in the same condition.

NCT06354790 — Natural History Study of Children With LAMA2-related Dystrophies · recruiting
NCT06924125 — Spanish Natural History Study for LAMA2 Muscular Dystrophy · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04299321 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Prothelia, Inc.
Last refreshed: 7 February 2022

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04299321.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing