Cell Therapy for IBM by Muscle Injection of ADSVF: a Phase I Trial (ADSVF-in-IBM)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT05032131|
Recruitment Status : Not yet recruiting
First Posted : September 2, 2021
Last Update Posted : September 2, 2021
Inclusion Body Myositis is a slowly but disabling myopathy, the most frequent in patients over 50 years old. No treatments (in particular immunosuppressive) are known to be efficient.
Autologous uncultured adipose-derived stromal vascular fraction (ADSVF) is recognized as an easily accessible (by a standard liposuction to obtain adipose tissue, from which ADSVF are isolated by centrifugation), safe and well tolerated source of cells with angiogenic, anti-inflammatory, immunomodulatory and regenerative properties. The purpose of our ADSVF in IBM phase I trial is to evaluate, for the first time in human diseased muscle, first the tolerance of autologous ADSVF cells locally injected in affected forearm muscles and second their capability to repair those muscles. With always the goals of tolerance first and second muscle repair, we will recruit in parallel two groups of IBM patients: the first treated by sirolimus since at least 6 months (but still disabled) and the second currently (for at least 3 months) without specific treatment for inclusion myositis.
|Condition or disease||Intervention/treatment||Phase|
|Inclusion Body Myositis||Biological: ADSVF||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||32 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Cell Therapy for Inclusion Body Myositis (IBM) by Muscle Injection of Autologous Uncultured Adipose-Derived Stromal Vascular Fraction (ADSVF): a Phase I Trial|
|Estimated Study Start Date :||September 2021|
|Estimated Primary Completion Date :||October 2023|
|Estimated Study Completion Date :||April 2024|
Patients treated by sirolimus since at least 6 months (but still disabled)
Patients currently (for at least 3 months) without specific treatment for inclusion myositis
Group 1 and 2 : Escalating dose of ADSVF injection (5 millions of cells, 10 millions of cells and 20 millions of cells).
- In each group, tolerance of escalating doses (3+3) of ADSVF in the non-dominant forearm [ Time Frame: Days 0 (day of the injection) to day 30 ]By determining the dose-limiting toxicity (DLT)
- In each group, tolerance of escalating doses (3+3) of ADSVF in the non-dominant forearm [ Time Frame: Days 0 (day of the injection) to 6 months (end of participation) ]By research of adverse events
- In each group, efficacy in term of muscle repair (regenerative properties of ADSVF) [ Time Frame: At day 30, 3 month and 6 month ]Functional muscle evaluations
- In each group, efficacy in term of muscle repair (regenerative properties of ADSVF) [ Time Frame: At 6 month ]Muscle mass, fatty replacement and inflammation by quantitative NMRI
- In each group, evaluation of muscle inflammation control [ Time Frame: At 6 month ]Immunomonitoring of the peripheral blood mononuclear cells (PBMC)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05032131
|Contact: Olivier Benveniste, Professor||01 42 16 10 firstname.lastname@example.org|
|Contact: Anne Radenne, Manager||01 42 16 16 email@example.com|
|Principal Investigator:||Olivier Benveniste, Professor||APHP|