Inclusion Body MyositisPhase 1ADSVF-in-IBMNCT05032131
Cell Therapy for IBM by Muscle Injection of ADSVF
A Phase 1 study testing ADSVF in people with Inclusion Body Myositis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 45 to 80 years
- Conditions the study is for
- Inclusion Body Myositis
- What is being tested?
- ADSVF
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 1 siteFrance
- Study dates
- 1 Feb 2023to Apr 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 45 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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)
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- 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
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Olivier BENVENISTE
Paris, 75013
Olivier BENVENISTE, 0142161699
Anne RADENNE
About this study
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
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.
Read the detailed description
The main objective of this study is to evaluate the tolerance of escalating doses of ADSVF, one month after intramuscular injection in the finger flexors, in the non-dominant forearm. The second objective of this study is to evaluate the efficacy in term of muscle repair (regenerative properties of ADSVF) and in term of muscle inflammation control, during 6 months, by functional strength tests, quantitative MRI and PBMC monitoring. This research is a phase I trial evaluating first the tolerance and second the efficacy of 3 escalating doses of ADSVF intramuscularly injected in the non-dominant forearm. The volume of injection will be of 1 ml in each of the 5 sites of the finger flexoses, in line at 1 cm apart, for a total dose of 5 millions (low dose) or 10 millions (intermediate dose) or 20 millions (high dose) of viable nucleated cells.These doses are chosen because of the perfect tolerance of intra-muscle injection of (in average) one million ADSVF per millilitre, with a total dose of viable nucleated cells injected between 2.5 and 8.6 millions.The cell treatment will be prepared from autologous uncultured Adipose-Derived Stromal Vascular Fraction (ADSVF) isolated by centrifugation of adipose tissue obtain by liposuction. The study population will be adult patients suffering of an Inclusion Body Myositis (IBM) fulfilling the Lloyd criteria treated by sirolimus since at least 6 months (but still disabled) - who are part of group 1 or currently (for at least 3 months) without specific treatment for inclusion myositis - who are part of group 2. The main inclusion criteria are : Patients: with an age ≥ 45 and ≤ 80 years old, with IBM defined by the Lloyd criteria: 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 - group 2. The duration of participation of the patients will be 7 months, included one month between maximum the inclusion visit and the injection visit, and 6 month of follow-up periof after ADSVF injection.
- Conditions
- Inclusion Body Myositis
- Being tested
- ADSVF
- Keywords
- Inclusion Body Myositis, Cell Therapy, ADSVF
- Medical subject headings
- Myositis, Inclusion Body
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ADSVFBiological
Group 1 and 2 : Escalating dose of ADSVF injection (5 millions of cells, 10 millions of cells and 20 millions of cells).
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Biological: ADSVF |
What is being measured
The primary outcome is the main question the study is designed to answer.
In each group, tolerance of escalating doses (3+3) of ADSVF in the non-dominant forearm
By determining the dose-limiting toxicity (DLT)
Measured: 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
By research of adverse events
Measured: 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)
Functional muscle evaluations
Measured: At day 30, 3 month and 6 month
In each group, efficacy in term of muscle repair (regenerative properties of ADSVF)
Muscle mass, fatty replacement and inflammation by quantitative NMRI
Measured: At 6 month
In each group, evaluation of muscle inflammation control
Immunomonitoring of the peripheral blood mononuclear cells (PBMC)
Measured: At 6 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2021
- Study start1 Feb 2023
- Primary completionOct 2023 (estimated)
- Study completionApr 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inclusion Body MyositisStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Olivier Benveniste, Professor, Principal investigator, APHP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05032131
- Sponsor's protocol ID
- APHP180593
- Other IDs
- 2020-005876-36
- Acronym
- ADSVF-in-IBM
- Status last verified
- Feb 2023
- First submitted
- 27 Aug 2021
- Last updated
- 22 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 22 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05032131
- Acronym / sponsor ID
- ADSVF-in-IBM / APHP180593
- Conditions
- Inclusion Body Myositis
- Keywords
- Inclusion Body Myositis, Cell Therapy, ADSVF
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 1 Feb 2023 (actual)
- Primary completion
- Oct 2023 (estimated)
- Study completion
- Apr 2024 (estimated)
- First posted
- 2 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The main objective of this study is to evaluate the tolerance of escalating doses of ADSVF, one month after intramuscular injection in the finger flexors, in the non-dominant forearm. The second objective of this study is to evaluate the efficacy in term of muscle repair (regenerative properties of ADSVF) and in term of muscle inflammation control, during 6 months, by functional strength tests, quantitative MRI and PBMC monitoring. This research is a phase I trial evaluating first the tolerance and second the efficacy of 3 escalating doses of ADSVF intramuscularly injected in the non-dominant forearm. The volume of injection will be of 1 ml in each of the 5 sites of the finger flexoses, in line at 1 cm apart, for a total dose of 5 millions (low dose) or 10 millions (intermediate dose) or 20 millions (high dose) of viable nucleated cells.These doses are chosen because of the perfect tolerance of intra-muscle injection of (in average) one million ADSVF per millilitre, with a total dose of viable nucleated cells injected between 2.5 and 8.6 millions.The cell treatment will be prepared from autologous uncultured Adipose-Derived Stromal Vascular Fraction (ADSVF) isolated by centrifugation of adipose tissue obtain by liposuction. The study population will be adult patients suffering of an Inclusion Body Myositis (IBM) fulfilling the Lloyd criteria treated by sirolimus since at least 6 months (but still disabled) - who are part of group 1 or currently (for at least 3 months) without specific treatment for inclusion myositis - who are part of group 2. The main inclusion criteria are : Patients: with an age ≥ 45 and ≤ 80 years old, with IBM defined by the Lloyd criteria: 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 - group 2. The duration of participation of the patients will be 7 months, included one month between maximum the inclusion visit and the injection visit, and 6 month of follow-up periof after ADSVF injection.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental | Experimental | Biological: ADSVFGroup 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 |
Who may qualify
- Ages
- 45 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (17 listed)
- 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%)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ADSVFGroup 1 and 2 : Escalating dose of ADSVF injection (5 millions of cells, 10 millions of cells and 20 millions of cells). | Biological | Experimental |
Primary outcome
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)
4 secondary outcomes
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)
In each group, efficacy in term of muscle repair (regenerative properties of ADSVF)
Time frame: At day 30, 3 month and 6 month
In each group, efficacy in term of muscle repair (regenerative properties of ADSVF)
Time frame: At 6 month
In each group, evaluation of muscle inflammation control
Time frame: At 6 month
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Mar 2023.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Olivier Benveniste, Professor principal investigator
APHP
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ADSVF in people with Inclusion Body Myositis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 22 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Olivier Benveniste, Professor (01 42 16 10 88, olivier.benveniste@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Mar 2023.