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

Development of an Autologous Myogenic Cell Therapy against the Neuromuscular Phenotype of Myotonic Dystrophy type 1. - Cellular therapy for DM1.

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON43189

Enrollment

Registered

2016-10-18

Start date

2018-03-23

Completion date

Unknown

Last updated

2024-04-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

inherited muscle disorder Steinert disease

Interventions

None listed

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

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

Exclusion criteria

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

Design outcomes

Primary

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

Measure

Time frame

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

—

Secondary

Measure	Time frame
Not applicable.	—

Countries

Netherlands

Outcome results