Clinical trial NCT02753530: Study of Arimoclomol in Inclusion Body Myositis (IBM)
Official title: Phase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Inclusion Body Myositis
Keywords: IBM
Lead sponsor: ZevraDenmark
Sponsor class: INDUSTRY
Collaborators: name: University of Kansas Medical Center
name: University College, London
First posted: 2016-04-28
Enrollment: 152
Sex: ALL
Minimum age (years): 45
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

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

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.

Primary outcomes:
measure: Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
timeFrame: Change from Baseline to Month 20
Secondary outcomes:
measure: Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
timeFrame: Change from Baseline to Month 12
measure: Grip Strength
timeFrame: Change from Baseline to Month 12 and 20
measure: Modified Timed up and go (mTUG)
timeFrame: Change from Baseline to Month 12 and Month 20
measure: Manual Muscle Testing (MMT), Total Score
timeFrame: Change from Baseline to Month 12 and Month 20
measure: 6 Minute Walk Test (6MWT); Distance After 6 Minutes (6MWD)
timeFrame: Change from Baseline to Month 12 and Month 20
measure: Short Form-36 (SF-36) Physical Component Score
timeFrame: Change from Baseline to Month 12 and Month 20
measure: Maximum Voluntary Isometric Contraction (MVICT) of Quadriceps
timeFrame: Change from Baseline to Month 12 and Month 20
measure: Health Assessment Questionnaire - Disability Index (HAQ-DI)
timeFrame: Change from Baseline to Month 12 and Month 20
measure: 2 Minute Walk Test (2MWT)
timeFrame: Change from Baseline to Month 12 and Month 20
measure: Short Form-36 (SF-36) Mental Component Score
timeFrame: Change from Baseline to Month 12 and Month 20
measure: Patient Global Impression of Severity (PGIS)
timeFrame: Change from Baseline to Month 12 and Month 20
measure: Patient Global Impression of Change (PGIC)
timeFrame: Change from Baseline to Month 12 and Month 20
measure: Clinician Global Impression of Severity (CGIS)
timeFrame: Change from Baseline to Month 12 and Month 20
measure: Clinician Global Impression of Change (CGIC)
timeFrame: Change from Baseline to Month 12 and Month 20
measure: Falls and Near Falls
timeFrame: Accumulated number from Baseline to Month 20
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Neurological Associates
state: Arizona
city: Irvine
country: United States
facility: University of California, Irvine
state: California
city: Aurora
country: United States
facility: University of Colorado School of Medicine
state: Colorado
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Columbus
country: United States
facility: The Ohio State University
state: Ohio
city: Houston
country: United States
facility: Nerve and Muscle Center of Texas
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: London
country: United Kingdom
facility: University College of London
Study officials:
affiliation: University of Kansas Medical Center
name: Mazen Dimachkie, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University College, London
name: Michael Hanna, MD
role: PRINCIPAL_INVESTIGATOR
References: