Development of an Autologous Myogenic Cell Therapy against the Neuromuscular Phenotype of Myotonic Dystrophy type 1.

Completed

• Conditions: inherited muscle disorder; Steinert disease; 10027664; 10028302

• Registration Number: NL-OMON43189
• Lead Sponsor: Radboud Universitair Medisch Centrum

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 11

Inclusion Criteria

- Written informed consent
- Age: 18+
- Sex: male/female
- Genetically confirmed diagnosis with DM1

Exclusion Criteria

- Psychiatric or other disorders likely to impact on the informed consent
- Patients unable and/or unwilling to comply with the study instructions
- Concurrent illness
- Ongoing participation in other clinical trials
- Major surgery within 4 weeks of the visit

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Isolate, culture and expand genetically corrected pericytes derived from a<br /><br>human skeletal muscle biopsy<br /><br>* Determine the efficiency of pericyte generation: number of pericytes/ mg<br /><br>skeletal muscle biopsy<br /><br>* Determine the percentage of edited pericytes generated by CRISPR-Cas9<br /><br>modification: percentage of pericytes with complete *clean* removal of the<br /><br>entire repeat sequence<br /><br>* Determine the maximum amount of amplification: number of passages possible<br /><br>with edited pericytes</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Not applicable. </p><br>