Study of Arimoclomol in Inclusion Body Myositis (IBM)
Condition: Inclusion Body Myositis
MeSH terms: Myositis; Myositis, Inclusion Body
Intervention: Arimoclomol; Placebo
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; 2 matched placebo capsules taken 3 times daily during breakfast, early afternoon, and at bedtime
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 152.0
Sex: All
Minimum age: 45 Years
Healthy volunteers: No
Keywords: IBM

Summary:

      Funding Source - FDA Office of Orphan Products Development (OOPD). The purpose of this study
      is to evaluate the safety and efficacy of the study drug, arimoclomol in IBM patients.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Meet any of the European Neuromuscular Centre Inclusion Body Myositis research
             diagnostic criteria 2011 categories for IBM.

          -  Demonstrate being able to arise from a chair without support from another person or
             device.

          -  Able to ambulate at least 20 ft/6 meters with or without assistive device. Once arisen
             from the chair, participant may use any walking device, i.e. walker/frame, can,
             crutches, or braces. They cannot be supported by another person and cannot use
             furniture or wall for support.

          -  Age at onset of weakness >45 years.

          -  Body weight of >= 40 kg.

          -  Able to give informed consent.

        Exclusion Criteria:

          -  History of any of the following excludes subject participation in the study: chronic
             infection particularly HIV or Hepatitis B or C; cancer other than basal cell cancer
             less than five years prior; or other chronic serious medical illnesses.

          -  Presence of any of the following on routine blood screening: White blood cells (WBC)
             <3000; platelets < 100,000; hematocrit <30%; blood urea nitrogen (BUN) >30 mg/dL;
             creatinine >1.5 x upper limit of normal; symptomatic liver disease with serum albumin
             <3 g/dL.

          -  History of most recent creatine kinase >15x the upper limit of normal without any
             other explanation besides IBM.

          -  History of non-compliance with other therapies.

          -  Use of testosterone except for physiologic replacement doses in case of androgen
             deficiency. Participants must have documented proof of the androgen deficiency.

          -  Coexistence of other disease that would be likely to affect outcome measures

          -  Drug or alcohol abuse within past three months.

          -  Participation in a recent drug study in the last 30 days prior to the screening visit
             or use of a biologic agent less than 6 months prior to the screening visit.

          -  Women who are lactating or unwilling to use adequate method of birth control who are
             not surgically sterile. Adequate birth control includes use of intrauterine device,
             abstinence, or oral contraceptives or a double barrier method, e.g condom plus
             diaphragm will be necessary for both male and female participants.

          -  Participants taking >7.5 mg prednisolone or equivalent or participants on Intravenous
             Immunoglobulin Therapy (IVIg) or other immunosuppressants within the last 3 months.

          -  Clinically significant renal or hepatic disease, as indicated by clinical laboratory
             assessment.