Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
Condition: Inclusion Body Myositis
MeSH terms: Myositis; Myositis, Inclusion Body
Intervention: Arimoclomol
Intervention description: 2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily during breakfast, early afternoon, and at bedtime
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 121.0
Sex: All
Minimum age: 45 Years
Healthy volunteers: No
Keywords: IBM

Summary:

      A multicenter, nonrandomized, open-label, uncontrolled clinical extension trial designed to
      compare the efficacy and safety of early versus delayed start of arimoclomol in the treatment
      of Inclusion Body Myositis (IBM)
    

Eligibility criteria:

        Inclusion Criteria:

          1. Patient is able to comprehend and is willing to provide written informed consent and
             is capable and willing to comply with trial procedures.

          2. Patient has completed the IBM4809 trial on treatment with Investigational Medicinal
             Product (IMP).

        Exclusion Criteria:

          1. Known or suspected allergy or intolerance to arimoclomol or its constituents.

          2. Exposure to any other investigational treatment within 30 days or <5 half-lives of the
             baseline visit or taking part or planning to take part in another interventional
             trial.

          3. Significant protocol deviation in the blinded IBM4809 trial based on the
             investigator's judgement in discussion with the medical monitor.

          4. Women who are lactating or pregnant, or men or women unwilling to use a highly
             effective method of birth control if not surgically sterile (defined as bilateral
             tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men)
             for female participants until 4 weeks after last dose and for male participants up to
             3 months after last dose. Premenopausal women must have a negative pregnancy test
             prior to dosing with trial medication. Acceptable methods of birth control are:

               -  Hormonal methods associated with inhibition of ovulation such as oral,
                  implantable, injectable, or transdermal contraceptives for a minimum of 1 full
                  cycle (based on the patient's usual menstrual cycle period) before arimoclomol
                  administration.

               -  Total abstinence from sexual intercourse since the last menses before arimoclomol
                  administration. (The reliability of sexual abstinence needs to be evaluated in
                  relation to the duration of the clinical trial and the preferred and usual
                  lifestyle of the patient. Periodic abstinence [calendar, symptothermal,
                  post-ovulation] methods are not acceptable methods of contraception).

               -  Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS).

          5. Any concurrent condition that in the investigator's opinion will significantly
             interfere with assessment of safety or efficacy.

          6. Inability to comply with the protocol-specified procedures/evaluations and scheduled
             visits as per the investigator.