HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.

NO_LONGER_AVAILABLE

Conditions studied: Congenital Muscular Dystrophy Due to Lamin A/C Mutation

In brief

This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD). In this expanded access, the patient will receive the investigational product through 14 intravenous infusions, followed by Follow-Up visit and an End of Study.

Key facts

Study ID

NCT05154851

Run by

Hope Biosciences Research Foundation

Last updated by the study team

2025-09-29

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subject diagnosed with LCMNA congenital muscular dystrophy.
• Subject must have banked his stem cells at Hope Biosciences LLC.

You may not qualify if…

• Subject has any active infection requiring medications.
• The subject has any known coagulation anomalies.

Where it is running

• Hope Biosciences Stem Cell Research Foundation — Sugar Land, Texas, United States

Full record on ClinicalTrials.gov

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