NO_LONGER_AVAILABLENot Applicable

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

United States

Plain-language summary

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.

Who can participate

SexALL

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Inclusion criteria

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

Exclusion criteria

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

Trial details

NCT IDNCT05154851

SponsorHope Biosciences Research Foundation

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

View on ClinicalTrials.gov More Congenital Muscular Dystrophy Due to Lamin A/C Mutation trials