NCT02196467

Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients

Status unknown Phase 1/Phase 2 Last updated 28 January 2021

What this trial tests

Phase 1/Phase 2 trial testing Myoblast transplantation in Duchenne Muscular Dystrophy in 10 participants. Status unknown.

Timeline

1 May 2014

Primary endpoint
1 January 2024

1 January 2024

Quick facts

Lead sponsor	CHU de Quebec-Universite Laval
Phase	Phase 1/Phase 2
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	randomized
Design	single group
Masking	quadruple
Primary purpose	treatment
Enrollment	10
Start date	1 May 2014
Primary completion	1 January 2024
Estimated completion	1 January 2024
Sites	2 locations across Canada

Drugs / interventions tested

• Myoblast transplantation — full drug profile →
• Saline injection

Conditions studied

• Duchenne Muscular Dystrophy — all drugs for Duchenne Muscular Dystrophy →

Sponsor

CHU de Quebec-Universite Laval — full company profile →

Who can join

16 and older, male only, with Duchenne Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Number of Participants with Serious and Non-Serious Adverse Events as a measure of safety.
  Time frame: Up to 6 months
  The patients will be monitored for local and systemic potential adverse effects due to the transplantation and for adverse effects associated with immunosuppression with tacrolimus.

Sponsor's own description

This Phase I/II of the clinical trial is to investigate whether the transplantation of normal myoblasts throughout one muscle (the extensor carpi radialis) of the patients is safe and will improve the strength of that muscle. During this Phase I/II, the patients will be transplanted with myoblasts grown from the muscle biopsy of a donor and kept frozen in liquid nitrogen. Thirty million myoblasts will be injected per cm cube in a progressively higher surface of the radialis (i.e., 3, 6 and 9 cm2). The contralateral muscle will be injected with saline to serve as a control. The strength of both muscles will be measured at 3 months post transplantation to verify whether the myoblast transplantation improved the strength of the muscle. If there is no significant strength improvement, the protocol will be terminated immediately for that patient. If there is a significant strength improvement, the patient will be maintained under immunosuppression until 6 months post transplant and his strength will be re-evaluated.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Tissue Stem Cells: Architects of Their Niches.
   Fuchs E, Blau HM. · · 2020 · cited 231× · PMID 33007238 · DOI 10.1016/j.stem.2020.09.011
2. Stem cell therapy for muscular dystrophies.
   Biressi S, Filareto A, Rando TA. · · 2020 · cited 69× · PMID 32946430 · DOI 10.1172/jci142031
3. Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials.
   Shimizu-Motohashi Y, Miyatake S, Komaki H, Takeda S, et al · · 2016 · cited 68× · PMID 27398133
4. Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches.
   Shimizu-Motohashi Y, Komaki H, Motohashi N, Takeda S, et al · · 2019 · cited 62× · PMID 30621068 · DOI 10.3390/jpm9010001
5. Progress and prospects of gene therapy clinical trials for the muscular dystrophies.
   Bengtsson NE, Seto JT, Hall JK, Chamberlain JS, et al · · 2016 · cited 48× · PMID 26450518 · DOI 10.1093/hmg/ddv420
6. A Brief Review of Duchenne Muscular Dystrophy Treatment Options, with an Emphasis on Two Novel Strategies.
   Heydemann A, Siemionow M. · · 2023 · cited 18× · PMID 36979809 · DOI 10.3390/biomedicines11030830
7. Dystrophin Dp71ab is monoclonally expressed in human satellite cells and enhances proliferation of myoblast cells.
   Farea M, Rani AQM, Maeta K, Nishio H, et al · · 2020 · cited 18× · PMID 33051488 · DOI 10.1038/s41598-020-74157-y
8. Portrait of Dysferlinopathy: Diagnosis and Development of Therapy.
   Bouchard C, Tremblay JP. · · 2023 · cited 15× · PMID 37762951 · DOI 10.3390/jcm12186011