Arimoclomol in Sporadic Inclusion Body Myositis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00769860
• Date of registration: 08/10/2008
• Prospective Registration: No
• Primary sponsor: Richard Barohn, MD
• Public title: Arimoclomol in Sporadic Inclusion Body Myositis
• Scientific title: Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis
• Date of first enrolment: September 2008
• Target sample size: 24
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00769860
• Study type: Interventional
• Phase: Phase 2/Phase 3

Countries of recruitment

United Kingdom, United States

Key inclusion & exclusion 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 < 3000, platelets <
100,000, hematocrit < 30%, BUN > 30 mg%, creatine > 1.5 mg%, symptomatic liver disease
with serum albumin < 3 g/dl, PT or PTT > 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



Age minimum: 50 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Inclusion Body Myositis

Intervention(s)

Other: Placebo

Drug: Arimoclomol

Primary Outcome(s)

Count of Adverse Events Reported [Time Frame: Month 12]

Secondary Outcome(s)

Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score [Time Frame: Change from Baseline to Month 8]

Maximum Isometric Voluntary Contraction Testing (MVICT) Score [Time Frame: Change from Baseline to Month 8]

Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score [Time Frame: Change from Baseline to Month 4]

Maximum Isometric Voluntary Contraction Testing (MVICT) Score [Time Frame: Change from Baseline to Month 12]

Heat Shock Protein 70 (HSP70) Levels in the Tissue [Time Frame: Change from Baseline to Month 4]

Muscle Strength Testing [Time Frame: Change from Baseline to Month 4]

Muscle Strength Testing [Time Frame: Change from Baseline to Month 8]

Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score [Time Frame: Change from Baseline to Month 12]

Maximum Isometric Voluntary Contraction Testing (MVICT) Score [Time Frame: Change from Baseline to Month 4]

Muscle Strength Testing [Time Frame: Change from Baseline to Month 12]

Secondary ID(s)

10656

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 19/01/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00769860