Cell Therapy for IBM by Muscle Injection of ADSVF (ADSVF-in-IBM)
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|ClinicalTrials.gov Identifier: NCT05032131|
Recruitment Status : Recruiting
First Posted : September 2, 2021
Last Update Posted : March 22, 2023
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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|
|Actual Study Start Date :||February 1, 2023|
|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)
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|Ages Eligible for Study:||45 Years to 80 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- With an age ≥ 45 and ≤ 80 yo.
- Man or menopausal woman. - With IBM defined by the Lloyd criteria (Lloyd et al., 2014): muscle weakness of finger flexors or quadriceps, and endomysial inflammatory infiltrates on muscle biopsy, and presence of invaded fibers or rimmed vacuoles on muscle biopsy.
- Who gave their written informed consent
- Affiliated to a social security regime (expected AME) And for: -group 1: treated by sirolimus since at least 6 months (but still disabled ) - group 2: currently (for at least 3 months) without specific treatment for inclusion myositis.
- Impossibility to walk 10 meters
- Grip evaluated by MRC5 MMT at 0 OR 1.
- Body mass index < 18
- Not able to stop any anticoagulant, or antiaggregant drugs within the week before and the 48 hours before the liposuction
- Severe respiratory insufficiency (FVC < 50% and/or FEV1 < 50%)
- Severe chronic kidney disease (Estimated Glomerular Filtration Rate < 15 ml/min and/or proteinuria > 0.5 g/24h)
- Cancer non in remission (necessitating specific treatment) during the past 12 months
- Connective Tissue Disease non in remission (necessitating specific treatment) during the past 12 months - Bone marrow transplantation
- Connective Tissue disease non in remission (necessitating specific treatment) during the past 12months - Immunosuppressive drugs except sirolimus, ongoing or stopped in less than 3 months
- Polyvalent immunoglobulins (IV or sub-cut) ongoing or stopped in less than 3 months
- Any biotherapies (mAbs) such as ant-CD20, CTLA4Ig, anti-TNF, anti-IL6R, anti-IL1 etc… ongoing or stopped in less than 6 months.
- Seropositivity for HIV, HCV or HBV
- Contraindication to muscle MRI
- Contraindications to the liposuction: eg coagulation disorders, etc… - Contraindications to anaesthetics
- Documented conventional antibiotics severe allergy such as ß-lactam (cephalosporin), cyclins, macrolides (for example metronidazole), quinolones
- Participation in another trial (Jardé 1 or Jardé 2)
- Legal protection (curatorship or tutorship) or safety measure
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|
|Paris, France, 75013|
|Contact: Olivier BENVENISTE 0142161699|
|Contact: Anne RADENNE|
|Principal Investigator:||Olivier Benveniste, Professor||APHP|
|Responsible Party:||Assistance Publique - Hôpitaux de Paris|
|Other Study ID Numbers:||
2020-005876-36 ( EudraCT Number )
|First Posted:||September 2, 2021 Key Record Dates|
|Last Update Posted:||March 22, 2023|
|Last Verified:||February 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Yes|
Data are available upon reasonable request. The procedures carried out with the French data privacy authority (CNIL, Commission nationale de l'informatique et des libertés) do not provide for the transmission of the database, nor do the information and consent documents signed by the patients.
Consultation by the editorial board or interested researchers of individual participant data that underlie the results reported in the article after deidentification may nevertheless be considered, subject to prior determination of the terms and conditions of such consultation and in respect for compliance with the applicable regulations.
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
|Time Frame:||Beginning 3 months and ending 3 years following article publication. Requests out of these time frame can also be submitted to the sponsor|
|Access Criteria:||Researchers who provide a methodologically sound proposal|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Inclusion Body Myositis
Myositis, Inclusion Body
Nervous System Diseases