NCT05154851

Brief 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.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 9, 2021

Completed
4 days until next milestone

First Posted

Study publicly available on registry

December 13, 2021

Completed
Last Updated

September 29, 2025

Status Verified

September 1, 2025

First QC Date

December 9, 2021

Last Update Submit

September 24, 2025

Conditions

Congenital Muscular Dystrophy Due to Lamin A/C Mutation

Keywords

CMDMuscular DystrophyCongenitalStem cellsL-CMD

Interventions

HB-adMSCsBIOLOGICAL

HB-adMSCs (Hope Biosciences autologous adipose-derived mesenchymal stem cells) is manufactured by Hope Biosciences, L.L.C., a biotechnology company headquartered in Sugar Land, Texas.

Eligibility Criteria

Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hope Biosciences Stem Cell Research Foundation

Sugar Land, Texas, 77478, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Congenital, Lmna-RelatedMuscular Dystrophies

Condition Hierarchy (Ancestors)

Muscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Djamchid Lotfi, MD

    Investigator

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 9, 2021

First Posted

December 13, 2021

Last Updated

September 29, 2025

Record last verified: 2025-09

Locations

US(1)