Inclusion Body Myositis, SporadicPhase 2BIGTIMNCT06536166
Ruxolitinib Treatment in Inclusion Body Myositis
A Phase 2 study testing Ruxolitinib in people with Inclusion Body Myositis, Sporadic, run by Assistance Publique - Hôpitaux de Paris at 21 sites in France.
Trial summary
Refer to the "Detailed Description" section. Read more about this study
- Who is it for?
- Ages 45 years and older
- Conditions the study is for
- Inclusion Body Myositis, Sporadic
- What is being tested?
- Ruxolitinib
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 21 sitesFrance
- Study dates
- 27 May 2025to 10 Sep 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 45 years
- Effective contraception for the duration of the clinical trial for fertile women of childbearing age
- Defined diagnosis of IBM according to data-derived criteria (Llyod et al, 2014): Patient must fulfill the three following criteria for being diagnosed as IBM: (1) finger flexor or quadriceps weakness; and (2) muscle biopsy showing endomysial inflammation; and (3) muscle biopsy showing invasion of nonnecrotic muscle fibers or rimmed vacuoles
- To be able to walk 6 min without assistance from another person (external assist devices permitted [e.g., canes, walkers, or rollators])
- Patient informed and having signed the consent for participation, possibly assisted by a trusted person
Who cannot join
- Pregnancy or breastfeeding
- Patient under guardianship, curatorship, safeguard of justice or deprived of liberty
- Patient with cognitive disorders or unable, according to the investigator, to understand the study and/or to give informed consent
- Quadriceps weakness (manual muscle testing, MRC) below or equal 1
- Forced vital capacity (FVC) or forced expiratory volume (FEV) < 50% of predicted value
- Concomitant use of immunomodulatory drugs including previous treatment with JAK inhibitor, or medications acting on muscle anabolism or catabolism
Show all 24Show fewer
- Live vaccine within the 4 weeks before starting treatment
- Comorbidity or active chronic disease which contraindicate ruxolitinib:
- Lipid parameters abnormalities/elevations
- Severe renal impairment (stage 4) and end-stage renal disease (stage 5)
- Hepatic impairment
- Cytopenia
- Recent history (<6 months) of cardiovascular or thromboembolic disease (documented coronaropathy or hospitalization for acute arterial thrombosis or stroke or deep venous thrombosis or pulmonary embolism)
- Active smoking more than 20 pack-years or history of respiratory or skin cancer or recent history (<6 months) of other neoplastic disease
- Very high cardiovascular risk (red color) at SCORE 2 in case of recent history (<6 months) of cardiovascular or thromboembolic disease and non-controlled cardiovascular risk factors
- History of Stevens-Johnson's syndrome or Lyell's syndrome
- Active SARS-CoV-2 infection (patient can be included once infection resolved)
- Any medical condition which limits the ability of participant to participate in study
- Necessity to use a drug incompatible with ruxolitinib
- Hypersensitivity to the IMP's active substance (ruxolitinib) or to any of the excipients
- Non-affiliation to a social security scheme or to another social protection scheme, patient on state medical aid
- Foreseeable inability, according to the investigator, to participate in all the visits, treatments and measures provided for in the protocol
- Concomitant participation in another clinical trial on medical product for human use, to a clinical investigation on a medical device, to interventional study involving human participants or in the exclusion period at the end of a previous clinical trial on medical product for human use, a clinical investigation on a medical device, or study involving human participants.
- Participation in non-interventional research is permitted.
Trial locations and contacts
21 sites in 1 country. Contact the study team or a site near you to ask about taking part.
21 sites in 1 country
France21 sites, 16 recruiting
- Enrolling by invitation
CHU d'Angers
Angers, France, 49100
- Enrolling by invitation
Centre hospitalier de la Côte Basque
Bayonne, France, 64109
- Enrolling by invitation
Hôpital CHU Jean Minjoz, CHU Besançon
Besançon, France, 25030
- Active, not recruiting
Hôpital Pellegrin - Tripode, CHU de Bordeaux
Bordeaux, France, 33076
- Enrolling by invitation
Hôpital de la Cavale Blanche, CHU de Brest
Brest, France, 29200
- Active, not recruiting
Hôpital Pierre Wertheimer, CHU de Lyon
Bron, France, 69500
- Recruiting
CHU Caen Normandie
Caen, France, 14000
Maxime FOURNIER, Dr., 02.33.06.31.66 ext. +33, fournier-m@chu-caen.fr
- Recruiting
Hôpital Henri-Mondor, APHP
Créteil, France, 94010
François Jérôme AUTHIER, Pr, 01.49.81.27.35 ext. +33, françois-jerome.authier@aphp.fr
- Active, not recruiting
Hôpital Raymond Poincaré, APHP
Garches, France, 92380
- Recruiting
Hôpital Roger Salengro, CHU de Lille
Lille, France, 59037
Céline TARD, Dr., 03.20.44.58.08 ext. +33, celine.tard@chu-lille.fr
- Recruiting
Hôpital Dupuytren, CHU de Limoges
Limoges, France, 87000
Laurent MAGY, Pr., 05.55.05.65.61 ext. +33, laurent.magy@chu-limoges.fr
- Not yet recruiting
Hôpital de la Timone, APHM
Marseille, France, 13005
Shahram ATTARIAN, Pr., 04.91.38.65.79 ext. +33, shahram.attarian@ap-hm.fr
- Enrolling by invitation
Hôpital Gui de Chauliac, CHU de Montpellier
Montpellier, France, 34000
- Recruiting
CHU Nancy
Nancy, France, 54035
Maud MICHAUD, Dr., 03.83.85.94.88 ext. +33, m.michaud@chu-nancy.fr
- Active, not recruiting
Hôtel-Dieu, CHU Nantes
Nantes, France, 44093
- Recruiting
Hôpital Pasteur, CHU de Nice
Nice, France, 06001
Sabrina SACCONI, Pr., 04.92.03.90.02 ext. +33, sacconi.s@chu-nice.fr
- Recruiting
Hôpital Pitié-Salpêtrière, APHP
Paris, France, 75013
Olivier BENVENISTE, Pr., 01.42.16.10.88 ext. +33, olivier.benveniste@aphp.fr
- Recruiting
Hôpital Christian Cabrol, CHU Reims
Reims, France, 51100
Loïs BOLKO, Dr., 03.26.78.44.70 ext. +33, lbolko@chu-reims.fr
- Recruiting
Hôpital Bellevue, CHU Saint-Etienne
Saint-Etienne, France, 42270
Léonard FEASSON, Pr., 04.77.82.91.89 ext. +33, leonard.feasson@univ-st-etienne.fr
- Active, not recruiting
Hôpital de Hautepierre, Hôpitaux Universitaires de Strasbourg
Strasbourg, France, 67089
- Recruiting
Hôpital Pierre-Paul Riquet, CHU de Toulouse
Toulouse, France, 31059
Pascal CINTAS, Dr., 05.61.77.94.40 ext. +33, cintas.p@chu-toulouse.fr
About this study
Official title: Blocking Interferon-γ by Ruxolitinib for Treating Inclusion Body Myositis: a Phase IIb Trial
Refer to the "Detailed Description" section.
Read the detailed description
A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of Ruxolitinib in the Treatment of Subjects with Inclusion Body Myositis (IBM) IBM is the most frequent idiopathic immune myopathy (IIM) over age 45, pathologically characterized by the combination of intramuscular inflammation and degenerative features. It differs from other IIMs by its chronic evolution and refractoriness to common immunomodulatory drugs leading to marked disability and poor quality of life. Histological and molecular analyses of muscle biopsies from IBM patients showed intense muscular type II interferon (IFNγ) signature, stronger than observed in other IIMs. In vitro and in vivo experimental studies showed that IFNγ exerts myosuppressive effects through JAK/STAT pathway activation mimicking the degenerative features observed in IBM, and that these effects can be prevented by JAK-inhibitor ruxolitinib.
Hypothesis/Objective : Ruxolitinib could be an effective therapy for IBM. Objective is to evaluate its therapeutic effects in IBM.
Method : Comparative, multicenter, randomized, parallel-group, superiority, placebo-controlled, double-blind, phase 2 trial. 60 IBM patients able to walk during at least 6mn will be randomized in two groups (30/group) and received either ruxolitinib 15mgx2/d or placebo during 1 yr. Evaluation includes 6MWT, muscle strength quantification, functional scales, respiratory functional test and muscle MRI.
- Conditions
- Inclusion Body Myositis, Sporadic
- Being tested
- Ruxolitinib, Placebo
- Keywords
- Inclusion body myositis, Idiopathic immune myopathy, JAK-inhibitor, Ruxolitinib
- 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.
RuxolitinibDrug
IBM patients treated by ruxolitinib (JAKAVI®), 15mg per os, twice a day, during 12 months.
PlaceboDrug
IBM patients treated by placebo, twice a day, during 12 months.
| Study group | Role | What they receive |
|---|---|---|
| Experimental group | Experimental | Randomization in experimental group. Receives: Drug: Ruxolitinib |
| Control group | Placebo comparator | Randomization in control group. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
6 minutes-walk distance (6MWT): A distance walked in 6 min. superior in treated patients compared to placebo group
The 6MWT is performed in a corridor, between two cones separated by a distance of 25 m.
Measured: 12 months
Secondary outcomes (8)
Adverse events (safety and tolerability) of ruxolitinib in IBM patients
Adverse events collected according to the MedDRA classification
Measured: Through study completion that is to say 15 months
Therapeutic muscular efficacy of ruxolitinib on muscle strength
Quantification of muscle strength using dynamometer
Measured: Until last consultation that is to say 12 months
Therapeutic muscular efficacy of ruxolitinib on overall muscle status
Measurement of overall muscle status using scales and serum creatine kinase (CK) levels
Measured: Until last consultation that is to say 12 months
Respiratory ability
Forced vital capacity (FVC) measurement
Measured: 12 months
Evaluate swallowing using Swallowing Disturbance Questionnaire (SDQ)
Measurement of the swallowing disorders via Swallowing Disturbance Questionnaire (SDQ). 15 items. Overall score from 0,5 to 44,5. The score increase with the swallowing disorders.
Measured: 12 months
Lower limb quantification of fat replacement of muscle tissue, residual muscle tissue and markers of disease activity using MRI
Measurement of the differences in fat fraction value calculated on thigh Dixon MRI pictures
Measured: 12 months
Evaluate quality of life using Health Assessment Questionnaire without Disability Index (HAQ-DI)
Measurement of the difference in the quality of life measured by Health Assessment Questionnaire without Disability Index (HAQ-DI). 8 dimensions rated from 0 (without any difficulty) to 3 (unable to do). Overall score from 0 to 3. The higher the score, the lower the quality of life.
Measured: 12 months
Evaluate quality of life using Duke health profile
Measurement of the difference in the quality of life measured by Duke health profile - The Duke. 10 dimensions. Overall score from 0 to 100. The score increase with the quality of life.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2024
- Study start27 May 2025
- Primary completion10 Jun 2028 (estimated)
- Study completion10 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inclusion Body Myositis, SporadicStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- François Jérôme AUTHIER, Pr, Study director, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06536166
- Sponsor's protocol ID
- APHP220829
- Acronym
- BIGTIM
- Status last verified
- Sep 2025
- First submitted
- 26 Jul 2024
- Last updated
- 29 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Datas are own by assistance publique - hopitaux de paris, please contact sponsor for further information.
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Blocking Interferon-γ by Ruxolitinib for Treating Inclusion Body Myositis: a Phase IIb Trial
- ClinicalTrials.gov ID
- NCT06536166
- Acronym / sponsor ID
- BIGTIM / APHP220829
- Conditions
- Inclusion Body Myositis, Sporadic
- Keywords
- Inclusion body myositis, Idiopathic immune myopathy, JAK-inhibitor, Ruxolitinib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 27 May 2025 (actual)
- Primary completion
- 10 Jun 2028 (estimated)
- Study completion
- 10 Sep 2028 (estimated)
- First posted
- 2 Aug 2024 (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.
Refer to the "Detailed Description" section.
Detailed description
A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of Ruxolitinib in the Treatment of Subjects with Inclusion Body Myositis (IBM) IBM is the most frequent idiopathic immune myopathy (IIM) over age 45, pathologically characterized by the combination of intramuscular inflammation and degenerative features. It differs from other IIMs by its chronic evolution and refractoriness to common immunomodulatory drugs leading to marked disability and poor quality of life. Histological and molecular analyses of muscle biopsies from IBM patients showed intense muscular type II interferon (IFNγ) signature, stronger than observed in other IIMs. In vitro and in vivo experimental studies showed that IFNγ exerts myosuppressive effects through JAK/STAT pathway activation mimicking the degenerative features observed in IBM, and that these effects can be prevented by JAK-inhibitor ruxolitinib.
Hypothesis/Objective : Ruxolitinib could be an effective therapy for IBM. Objective is to evaluate its therapeutic effects in IBM.
Method : Comparative, multicenter, randomized, parallel-group, superiority, placebo-controlled, double-blind, phase 2 trial. 60 IBM patients able to walk during at least 6mn will be randomized in two groups (30/group) and received either ruxolitinib 15mgx2/d or placebo during 1 yr. Evaluation includes 6MWT, muscle strength quantification, functional scales, respiratory functional test and muscle MRI.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Experimental group | Experimental | Drug: RuxolitinibRandomization in experimental group. |
| Control group | Placebo comparator | Drug: PlaceboRandomization in control group. |
Who may qualify
- Ages
- 45 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥ 45 years
- Effective contraception for the duration of the clinical trial for fertile women of childbearing age
- Defined diagnosis of IBM according to data-derived criteria (Llyod et al, 2014): Patient must fulfill the three following criteria for being diagnosed as IBM: (1) finger flexor or quadriceps weakness; and (2) muscle biopsy showing endomysial inflammation; and (3) muscle biopsy showing invasion of nonnecrotic muscle fibers or rimmed vacuoles
- To be able to walk 6 min without assistance from another person (external assist devices permitted [e.g., canes, walkers, or rollators])
- Patient informed and having signed the consent for participation, possibly assisted by a trusted person
Key exclusion criteria (24 listed)
- Pregnancy or breastfeeding
- Patient under guardianship, curatorship, safeguard of justice or deprived of liberty
- Patient with cognitive disorders or unable, according to the investigator, to understand the study and/or to give informed consent
- Quadriceps weakness (manual muscle testing, MRC) below or equal 1
- Forced vital capacity (FVC) or forced expiratory volume (FEV) < 50% of predicted value
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 |
|---|---|---|
| RuxolitinibIBM patients treated by ruxolitinib (JAKAVI®), 15mg per os, twice a day, during 12 months. | Drug | Experimental group |
| PlaceboIBM patients treated by placebo, twice a day, during 12 months. | Drug | Control group |
Primary outcome
6 minutes-walk distance (6MWT): A distance walked in 6 min. superior in treated patients compared to placebo group
Time frame: 12 months
The 6MWT is performed in a corridor, between two cones separated by a distance of 25 m.
8 secondary outcomes
Adverse events (safety and tolerability) of ruxolitinib in IBM patients
Time frame: Through study completion that is to say 15 months
Therapeutic muscular efficacy of ruxolitinib on muscle strength
Time frame: Until last consultation that is to say 12 months
Therapeutic muscular efficacy of ruxolitinib on overall muscle status
Time frame: Until last consultation that is to say 12 months
Respiratory ability
Time frame: 12 months
Evaluate swallowing using Swallowing Disturbance Questionnaire (SDQ)
Time frame: 12 months
Lower limb quantification of fat replacement of muscle tissue, residual muscle tissue and markers of disease activity using MRI
Time frame: 12 months
Evaluate quality of life using Health Assessment Questionnaire without Disability Index (HAQ-DI)
Time frame: 12 months
Evaluate quality of life using Duke health profile
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2026.
- Individual participant dataSharing planned: No. Datas are own by assistance publique - hopitaux de paris, please contact sponsor for further information.
Related trials for Inclusion Body Myositis, Sporadic
Active, not recruitingObservational study
Sporadic Inclusion Body Myositis Natural History Study
Sporadic Inclusion Body Myositis, Inclusion Body Myositis
RecruitingEarly Phase 1
Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis
Sporadic Inclusion Body Myositis (sIBM), Idiopathic Inflammatory Myopathies
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.
François Jérôme AUTHIER, Pr study director
Assistance Publique - Hôpitaux de Paris
21 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ruxolitinib in people with Inclusion Body Myositis, Sporadic, run by Assistance Publique - Hôpitaux de Paris at 21 sites 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 5 inclusion and 24 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: looking for participants now, as of the sponsor's last update on 29 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact François Jérôme AUTHIER, Pr (+33 1 4981 2735, francois-jerome.authier@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
6 minutes-walk distance (6MWT): A distance walked in 6 min. superior in treated patients compared to placebo group (time frame: 12 months). 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 29 May 2026.