Inclusion Body Myositis (IBM)Phase 2/3RAPAMINCT02481453
Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis
A Phase 2/3 study testing Rapamycin in people with Inclusion Body Myositis (IBM), run by Institut National de la Santé Et de la Recherche Médicale, France at 1 site in France.
Trial summary
Sporadic Inclusion Body Myositis (IBM) is the most frequent inflammatory myopathy in patients over 50. It is a slowly progressive, but today untreatable (notably by classical immunosuppressants) disease. Read more about this study
- Who is it for?
- Ages 45 to 85 years
- Conditions the study is for
- Inclusion Body Myositis (IBM)
- What is being tested?
- Rapamycin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 44 participants
- Where
- 1 siteFrance
- Study dates
- 15 Jul 2015to 22 Jan 2018
- Phase
- Phase 2/3
- 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 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- IBM defined by the Benveniste \& Hilton-Jones ( Neuromuscul Disord. 2010;20: 414-21) or Llyod criteria (Neurology 2014; 83: 426-433)
Who cannot join
- Impossiblility to walk 10 meters
- Hypersensitivity to rapamycin or one compound of the oral solution
- Severe respiratory insufficiency (FVC < 50% and/or FEV1 < 50%)
- Severe chronic kidney disease (Estimated Glomerular Filtration Rate < 15 ml/min and/or proteinuria > 0.3 g/24h)
- Chronic liver disease (cirrhosis and/or ALT/AST > 2.5 normal values)
- Cancer non in remission (necessitating specific treatment) during the past 12 months
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- Connective Tissue Disease non in remission (necessitating specific treatment) during the past 12 months
- Pregnancy
- Seropositivity for HIV, HCV or HBV
- Total cholesterolemia > 8 mmol/l
- Triglyceridemia > 5 mmol/l
- Hemoglobinemia < 11 g/dL
- Thrombopenia < 100 000/mm3
- Neutropenia < 1500/ mm3
- Lymphopenia < 1000/ mm3
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Étude de l'Effet de la Rapamycine Sur la Force Musculaire et la réponse Immunitaire au Cours de la Myosite à Inclusions: étude RAPAMI"
Sporadic Inclusion Body Myositis (IBM) is the most frequent inflammatory myopathy in patients over 50. It is a slowly progressive, but today untreatable (notably by classical immunosuppressants) disease.
Rapamycin used in organ transplantation blocks the activity of T effector cells, preserves T regulatory cells and induces autophagy (protein degradation), all parameters impaired during IBM.
RAPAMI is a prospective, randomised, controlled, double blind, monocentric, phase IIb trial evaluating rapamycine against placebo.
- Conditions
- Inclusion Body Myositis (IBM)
- Being tested
- Rapamycin, Placebo
- 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.
RapamycinDrug
Also called Sirolimus, Rapamune
Experimental: rapamycin oral solution, 2 mg/day during one year Comparator: placebo
PlaceboDrug
Also called Phosal
Comparator: placebo
| Study group | Role | What they receive |
|---|---|---|
| Rapamycin | Experimental | rapamycin 1 mg/ml oral solution, 2 mg/day (2 ml/day), once a day, during one year Receives: Drug: Rapamycin |
| Placebo | Placebo comparator | Placebo oral solution, 2 ml/day, once a day, during one year Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
stabilization of quadiceps strength measured by myometry
Measured: 52 weeks
Secondary outcomes (7)
stabilization of hand grip strength measured by myometry
Measured: 52 weeks
comparison of 6 minutes walking test
Measured: 52 weeks
composite measure of the handicap
Rivermead Mobility Index (RMI), scale Walton, Inclusion Body Myositis Weakness Composite Index (IBMWCI), Inclusion Body Myositis Functional rating scale (IBMFRS)
Measured: 52 weeks
Quality of life by different scales
Health Assessment Questionnaire (HAQ), Instrumental activities of daily living (IADL),Individualized Neuromuscular Quality of Life Questionnaire (INQol)
Measured: 52 weeks
measures of muscle fatty replacement by MRI
Measured: 52 weeks
Measure of the tolerance
Efficacy will be measured by:
check list of the known side effect of rapamycin.
Measured: 52 weeks
measure of inflammation by MRI
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2015 (estimated)
- Study start15 Jul 2015
- Primary completion22 Jan 2018
- Study completion22 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Inclusion Body Myositis (IBM)Studies of the same condition, open and under way.
- More trials run by Institut National de la Santé Et de la Recherche Médicale, FranceThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02481453
- Sponsor's protocol ID
- C12-66
- Other IDs
- 2013-003485-14
- Acronym
- RAPAMI
- Status last verified
- Jan 2017
- First submitted
- 13 May 2015
- Last updated
- 2 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Benveniste O, Hilton-Jones D. International Workshop on Inclusion Body Myositis held at the Institute of Myology, Paris, on 29 May 2009. Neuromuscul Disord. 2010 Jun;20(6):414-21. doi: 10.1016/j.nmd.2010.03.014. Epub 2010 Apr 21. No abstract available.
PubMed 20413309Lloyd TE, Mammen AL, Amato AA, Weiss MD, Needham M, Greenberg SA. Evaluation and construction of diagnostic criteria for inclusion body myositis. Neurology. 2014 Jul 29;83(5):426-33. doi: 10.1212/WNL.0000000000000642. Epub 2014 Jun 27.
PubMed 24975859Benveniste O, Hogrel JY, Belin L, Annoussamy M, Bachasson D, Rigolet A, Laforet P, Dzangue-Tchoupou G, Salem JE, Nguyen LS, Stojkovic T, Zahr N, Hervier B, Landon-Cardinal O, Behin A, Guilloux E, Reyngoudt H, Amelin D, Uruha A, Mariampillai K, Marty B, Eymard B, Hulot JS, Greenberg SA, Carlier PG, Allenbach Y. Sirolimus for treatment of patients with inclusion body myositis: a randomised, double-blind, placebo-controlled, proof-of-concept, phase 2b trial. Lancet Rheumatol. 2021 Jan;3(1):e40-e48. doi: 10.1016/S2665-9913(20)30280-0. Epub 2020 Oct 12.
PubMed 38273639
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Étude de l'Effet de la Rapamycine Sur la Force Musculaire et la réponse Immunitaire au Cours de la Myosite à Inclusions: étude RAPAMI"
- ClinicalTrials.gov ID
- NCT02481453
- Acronym / sponsor ID
- RAPAMI / C12-66
- Conditions
- Inclusion Body Myositis (IBM)
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Institut National de la Santé Et de la Recherche Médicale, FranceOther government
- Study start
- 15 Jul 2015 (actual)
- Primary completion
- 22 Jan 2018 (actual)
- Study completion
- 22 Jan 2018 (actual)
- First posted
- 25 Jun 2015 (estimated)
- 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.
Sporadic Inclusion Body Myositis (IBM) is the most frequent inflammatory myopathy in patients over 50. It is a slowly progressive, but today untreatable (notably by classical immunosuppressants) disease.
Rapamycin used in organ transplantation blocks the activity of T effector cells, preserves T regulatory cells and induces autophagy (protein degradation), all parameters impaired during IBM.
RAPAMI is a prospective, randomised, controlled, double blind, monocentric, phase IIb trial evaluating rapamycine against placebo.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Rapamycin | Experimental | Drug: Rapamycinrapamycin 1 mg/ml oral solution, 2 mg/day (2 ml/day), once a day, during one year |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo oral solution, 2 ml/day, once a day, during one year |
Who may qualify
- Ages
- 45 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- IBM defined by the Benveniste \& Hilton-Jones ( Neuromuscul Disord. 2010;20: 414-21) or Llyod criteria (Neurology 2014; 83: 426-433)
Key exclusion criteria (15 listed)
- Impossiblility to walk 10 meters
- Hypersensitivity to rapamycin or one compound of the oral solution
- Severe respiratory insufficiency (FVC < 50% and/or FEV1 < 50%)
- Severe chronic kidney disease (Estimated Glomerular Filtration Rate < 15 ml/min and/or proteinuria > 0.3 g/24h)
- Chronic liver disease (cirrhosis and/or ALT/AST > 2.5 normal values)
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 |
|---|---|---|
| RapamycinAlso: Sirolimus, RapamuneExperimental: rapamycin oral solution, 2 mg/day during one year Comparator: placebo | Drug | Rapamycin |
| PlaceboAlso: PhosalComparator: placebo | Drug | Placebo |
Primary outcome
stabilization of quadiceps strength measured by myometry
Time frame: 52 weeks
7 secondary outcomes
stabilization of hand grip strength measured by myometry
Time frame: 52 weeks
comparison of 6 minutes walking test
Time frame: 52 weeks
composite measure of the handicap
Time frame: 52 weeks
Quality of life by different scales
Time frame: 52 weeks
measures of muscle fatty replacement by MRI
Time frame: 52 weeks
Measure of the tolerance
Time frame: 52 weeks
measure of inflammation by MRI
Time frame: 52 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2025.
- Background: PubMed 20413309Benveniste O, Hilton-Jones D. International Workshop on Inclusion Body Myositis held at the Institute of Myology, Paris, on 29 May 2009. Neuromuscul Disord. 2010 Jun;20(6):414-21. doi: 10.1016/j.nmd.2010.03.014. Epub 2010 Apr 21. No abstract available.
- Background: PubMed 24975859Lloyd TE, Mammen AL, Amato AA, Weiss MD, Needham M, Greenberg SA. Evaluation and construction of diagnostic criteria for inclusion body myositis. Neurology. 2014 Jul 29;83(5):426-33. doi: 10.1212/WNL.0000000000000642. Epub 2014 Jun 27.
- Results publication: PubMed 38273639Benveniste O, Hogrel JY, Belin L, Annoussamy M, Bachasson D, Rigolet A, Laforet P, Dzangue-Tchoupou G, Salem JE, Nguyen LS, Stojkovic T, Zahr N, Hervier B, Landon-Cardinal O, Behin A, Guilloux E, Reyngoudt H, Amelin D, Uruha A, Mariampillai K, Marty B, Eymard B, Hulot JS, Greenberg SA, Carlier PG, Allenbach Y. Sirolimus for treatment of patients with inclusion body myositis: a randomised, double-blind, placebo-controlled, proof-of-concept, phase 2b trial. Lancet Rheumatol. 2021 Jan;3(1):e40-e48. doi: 10.1016/S2665-9913(20)30280-0. Epub 2020 Oct 12.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Rapamycin in people with Inclusion Body Myositis (IBM), run by Institut National de la Santé Et de la Recherche Médicale, France 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 1 inclusion and 15 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: finished, as of the sponsor's last update on 2 Sep 2025. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
stabilization of quadiceps strength measured by myometry (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02481453, which also lists 3 linked publications.
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 2 Sep 2025.