Cell Therapy for IBM by Muscle Injection of ADSVF

NCT05032131 | Unknown Phase 1 DMC

• 2 other identifiers: APHP1805932020-005876-36
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Inclusion Body Myositis

Keywords

Inclusion Body Myositis; Cell Therapy; ADSVF

Outcome Measures

Primary Outcomes (1)

• In each group, tolerance of escalating doses (3+3) of ADSVF in the non-dominant forearm

  By determining the dose-limiting toxicity (DLT)

  Days 0 (day of the injection) to day 30

Secondary Outcomes (4)

• In each group, tolerance of escalating doses (3+3) of ADSVF in the non-dominant forearm

  Days 0 (day of the injection) to 6 months (end of participation)

• In each group, efficacy in term of muscle repair (regenerative properties of ADSVF)

  At day 30, 3 month and 6 month

• In each group, efficacy in term of muscle repair (regenerative properties of ADSVF)

  At 6 month

• In each group, evaluation of muscle inflammation control

  At 6 month

Study Arms (1)

Experimental

EXPERIMENTAL

Group 1: Patients treated by sirolimus since at least 6 months (but still disabled) Group 2: Patients currently (for at least 3 months) without specific treatment for inclusion myositis

Biological: ADSVF

Interventions

ADSVF BIOLOGICAL

Group 1 and 2 : Escalating dose of ADSVF injection (5 millions of cells, 10 millions of cells and 20 millions of cells).

Experimental

Eligibility Criteria

• Age: 45 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Olivier BENVENISTE

Paris, 75013, France

RECRUITING

MeSH Terms

Conditions

Myositis, Inclusion Body

Condition Hierarchy (Ancestors)

Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Study Officials

• Olivier Benveniste, Professor

  APHP

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Olivier Benveniste, Professor

CONTACT

01 42 16 10 88; olivier.benveniste@aphp.fr

Anne Radenne, Manager

CONTACT

01 42 16 16 99; anne.radenne@aphp.fr

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 27, 2021
• First Posted: September 2, 2021
• Study Start: February 1, 2023
• Primary Completion: October 1, 2023
• Study Completion: April 1, 2024
• Last Updated: March 22, 2023

Record last verified: 2023-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, 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

Locations

FR (1)