RUXOLITINIB for Inclusion body myositis

Inclusion criteria

• {"criterion_text":"-\tAge ≥ 45 years\n-\tEffective contraception for the duration of the clinical trial for fertile women of childbearing age. The participant agrees to follow the contraceptive requirements detailed in the protocol.\n-\tDefined 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\n-\tTo be able to walk 6 min without assistance from another person (external assist devices permitted [e.g., canes, walkers, or rollators])\n-\tPatient informed and having signed the consent for participation, possibly assisted by a trusted person"}

Exclusion criteria

• {"criterion_text":"-\tPregnancy or breastfeeding\n-\tAny medical condition which limits the ability of participant to participate in study\n-\tNecessity to use a drug incompatible with ruxolitinib (see 7.4)\n-\tHypersensitivity to the IMP’s active substance (ruxolitinib) or to any of the excipients (Cellulose microcrystalline, magnesium stearate, silica colloidal anhydrous, sodium carboxymethyl starch (Type A), povidone K30, hydroxypropylcellulose 300 to 600 cps, lactose monohydrate)\n-\tNon-affiliation to a social security scheme or to another social protection scheme, patient on AME (state medical aid)\n-\tForeseeable inability, according to the investigator, to participate in all the visits, treatments and measures provided for in the protocol\n-\tConcomitant 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.\n-\tPatient under guardianship, curatorship, safeguard of justice or deprived of liberty\n-\tPatient with cognitive disorders or unable, according to the investigator, to understand the study and/or to give informed consent According to the appreciation of the investigator, non-French speaking patients may be included if a close one is able to translate the information provided. The patient must be able to benefit from the accompaniment of a relative for all of their visits (in teleconsultation and in hospital).\n-\tQuadriceps weakness (manual muscle testing, MRC) below or equal 1\n-\tForced vital capacity (FVC) or forced expiratory volume (FEV) < 50% of predicted value\n-\tConcomitant use of immunomodulatory drugs including previous treatment with JAK inhibitor, or medications acting on muscle anabolism or catabolism\n-\tLive vaccine within the 4 weeks before starting ruxolitinib therapy\n-\tComorbidity or active chronic disease which contraindicate ruxolitinib: •\tIf the results of the biological assessment including blood count, blood formula and biochemistry and dating back less than three months are available, see the non-inclusion criteria below. For patients whose results of the biological assessment carried out on the day of the inclusion and randomization visit are not yet available, these criteria constitute secondary exclusion criteria to be checked upon receipt of the results and before the randomization. \tLipid parameters abnormalities/elevations (in lack of cardiovascular risk factors, normal values with or without lipid-lowering treatment are: CTtotal cholesterol < 2 g/L; LDL-C < 1.6 g/L; HDL-C > 0.4 g/L; TGtriglycerides < 1.5 g/L) \tSevere renal impairment (stage 4) and end-stage renal disease (stage 5): GFR < 30mL/min/1.73m2 \tHepatic impairment: AST/ALT > 3 ULN and bilirubin > 1.5 ULN \tCytopenia (polymorphonuclear neutrophilsPNN ≤ 1.5 Giga/L or platelets ≤ 75 Giga/L or hemoglobin ≤ 10 g/dL)"}

Primary endpoints

• {"endpoint_text":"- Improvement of 60 meters for the distance walked during 6-minute (6WMD) from baseline to M12 (6MWD).","definition_or_measurement_approach":"Change in distance walked during 6-minute walk test from baseline to month 12; improvement defined as an increase of 60 meters."}

Secondary endpoints

• {"endpoint_text":"- 1. Safety and tolerance of ruxolitinib in IBM patients: Adverse events according to the MedDRA classification.","definition_or_measurement_approach":"Adverse events classified and reported using MedDRA terms."}
• {"endpoint_text":"- Therapeutic muscular efficacy of ruxolitinib through change from baseline to M12 of the parameters depending on skeletal muscle involvement: (i) Muscle strengt (ii) Overall muscle status, (iii) Respiratory ability, (iv) Swallowing, (v) Lower limb Quantification of fat replacement of muscle tissue, residual muscle tissue and markers of disease activity using MRI","definition_or_measurement_approach":"Changes from baseline to month 12 in measures of muscle strength (maximal voluntary isometric strength for specified muscle groups), overall motor function, respiratory measures (e.g., FVC), swallowing assessments, and MRI quantification of fat replacement/residual muscle and activity markers."}
• {"endpoint_text":"- 3. Improvement or stability between baseline and M12 in quality of life assessed by the scoring through Health Assessment Questionnaire without Disability Index (HAQ-DI), Duke health profile.","definition_or_measurement_approach":"Change or stability from baseline to month 12 in QoL scores using HAQ-DI and Duke Health Profile instruments."}
• {"endpoint_text":"- 4. Quality of the blinding assessed by the New Blinding Index (Bang, 2004).","definition_or_measurement_approach":"Assessment of blinding quality using the New Blinding Index (Bang, 2004)."}

Methods

• Internet recruitment support (document: L1_Support-recrutement-internet) — online outreach targeting patients with inclusion body myositis in France
• Recruitment posters/flyers (document: L1_Affiche-recrutement) — displayed in participating neuromuscular/reference centers and hospitals in France
• Formal RecruitmentProcedure document present (RecruitmentProcedure_2023-507666-32-00)

France

Earliest CTIS Part Ii Submission Date

18-09-2024

Latest Decision Or Authorization Date

28-08-2025

Processing Time Days

344

Number Of Sites

16

Number Of Participants

80

Primary sponsor

Full Name

Assistance Publique Hopitaux De Paris

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France

Third parties

• {"country":"France","full_name":"Programme Hospitalier de Recherche Clinique - PHRC (French Ministry of Health)","duties_or_roles":"Monetary support / funding","organisation_type":"Government / Funding body"}
• {"country":"","full_name":"NOVARTIS EUROPHARM LIMITED","duties_or_roles":"Organisation named in product information for Jakavi (product holder/MA organisation listed in product dictionary)","organisation_type":""}