Study of Arimoclomol in Inclusion Body Myositis (IBM)

Zevra Therapeutics

Status and phase: Completed; Phase 2

Conditions: Inclusion Body Myositis

Treatments: Drug: Arimoclomol; Other: Placebo

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02753530

STUDY00002461

R01FD004809

Details and patient eligibility

About

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.

Full description

A Phase 2/3, randomized, double-blind, placebo-controlled, international, 20-month intervention study in patients with IBM.

Enrollment: 152 patients

Sex: All

Ages: 45+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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152 participants in 2 patient groups, including a placebo group

Arimoclomol

Experimental group

Description:

248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily

Treatment:

Drug: Arimoclomol

Placebo

Placebo Comparator group

Description:

248 mg matching placebo 3 times daily

Treatment:

Other: Placebo