Phase 3 N=24 Randomized Double-blind Treatment

Arimoclomol in Sporadic Inclusion Body Myositis

Inclusion Body Myositis

Bottom Line

View on ClinicalTrials.gov: NCT00769860 ↗

Enrolled (actual)

Serious AEs

4.2%

Results posted

Jan 2017

Primary outcome: Primary: Count of Adverse Events Reported — 109; 52 adverse events reported

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Arimoclomol (Drug); Placebo (Other)
Age: Adult, Older Adult · 50+ yrs
Sex: All
Sponsor: Richard Barohn, MD
Primary completion: Sep 2012

Outcome Measures

Outcome	Result	p-value
PRIMARY Count of Adverse Events Reported	109; 52	—
SECONDARY Heat Shock Protein 70 (HSP70) Levels in the Tissue	-110.72; -34.70	—
SECONDARY Muscle Strength Testing	-0.21; -0.35	—
SECONDARY Muscle Strength Testing	-0.21; -0.35	—
SECONDARY Muscle Strength Testing	-0.21; -0.35	—
SECONDARY Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score	-2.03; -3.50	—
SECONDARY Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score	-2.03; -3.50	—
SECONDARY Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score	-2.03; -3.50	—
SECONDARY Maximum Isometric Voluntary Contraction Testing (MVICT) Score	-1.21; 0.52	—
SECONDARY Maximum Isometric Voluntary Contraction Testing (MVICT) Score	-1.21; 0.52	—
SECONDARY Maximum Isometric Voluntary Contraction Testing (MVICT) Score	-1.21; 0.52	—

Summary

Inclusion body myositis (IBM) is the most common progressive and debilitating muscle disease beginning in persons over 50 years of age. This study will assess the safety and tolerability of Arimoclomol in IBM as compared to placebo over 4 months of treatment.

Eligibility Criteria

Inclusion Criteria

Meet the diagnostic criteria for definite or probable IBM (Griggs 1995)
Muscle function adequate for quantitative muscle testing
Age > 50 years
Women must be postmenopausal or status post hysterectomy
For any patient currently taking medication for IBM, they must remain on current dosage for the extent of the study and last dosage change must be > 30 days previous to enrollment

Exclusion Criteria

Presence of any one of the following medical conditions: diabetes mellitus or patients taking anti-diabetic medications, chronic infection, chronic renal insufficiency, cancer other than skin cancer less than 5 years previously, multiple sclerosis or prior episode or central nervous system demyelination, or other chronic serious medical illnesses
Presence of any of the following on routine blood screening: WBC 30 mg%, creatine > 1.5 mg%, symptomatic liver disease with serum albumin upper range of control values
Women who are pregnant or lactating
History of non-compliance with other therapies
Coexistence of other neuromuscular disease
Drug or alcohol abuse within the last 3 months
Inability to give informed consent
Known bleeding disorder
Use of potentially renal toxic drugs
Prior difficulties with local anesthetic

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00769860). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.