A multicenter, nonrandomized, open-label, uncontrolled clinical extension trial designed to compare the efficacy and safety of early versus delayed start of arimoclomol in the treatment of Inclusion Body Myositis (IBM)
This was planned to be a 40-month open-label extension trial of the 20-month randomized, double-blind, placebo-controlled IBM4809 trial. The open-label trial was terminated early by the sponsor as a consequence of the results of IBM4809 which did not meet any of its efficacy endpoints. Therefore, the actual average duration of open-label treatment was approximately 28 weeks.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
121
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
Phoenix Neurological Associates
Phoenix, Arizona, United States
University of California
Irvine, California, United States
University of Colorado School of Medicine
Aurora, Colorado, United States
Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
The Inclusion Body Myositis Functional Rating Scale (IBMFRS) includes 10 measures (swallowing, handwriting, cutting food and handling utensils, fine motor tasks, dressing, hygiene, turning in bed and adjusting covers, changing position from sitting to standing, walking, and climbing stairs), each graded on a Likert scale from 0 (being unable to perform) to 4 (normal). The sum of the 10 items gives a value between 0 and 40. A higher score represents less functional limitation. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively.
Time frame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Change in Six Minutes Walking Distance Test; Distance at 6 Minutes (6MWD)
Patients were instructed to walk down one side of a track and back along the opposite side as quickly and safely as possible for 6 minutes. Patients were allowed to take breaks as needed during the walking period, but timing continued during breaks. The distance walked in meters was recorded after 6 minutes. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively.
Time frame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Change in Modified Timed Up and Go (mTUG)
The Modified Timed Up and Go (mTUG) measures the patient's ability to get up from a chair (allowing patients to use their arms), walk 3 meters, turn around, walk back to the chair, and sit down. The use of nearby walls or assistance from a caregiver was not allowed. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively.
Time frame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
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University of Kansas Medical Center
Kansas City, Kansas, United States
Johns Hopkins University
Baltimore, Maryland, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
University of Rochester
Rochester, New York, United States
The Ohio State University
Columbus, Ohio, United States
Nerve and Muscle Center of Texas
Houston, Texas, United States
University of Utah
Salt Lake City, Utah, United States
...and 2 more locations
Change in Quadriceps Muscle Strength
Maximal voluntary isometric contraction testing (MVICT) of the patient's quadriceps muscle (extensor strength of the knee) were performed using a hand myometer which is a hand-held device that allows the examiner to push against a muscle while the patient resists. The test was performed on each side. The results for the stronger knee are reported here. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively.
Time frame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Change in Hand Grip Strength
Hand grip strength was assessed using a dynamometer. The test was performed on each hand. The grip strength of the stronger hand is reported here. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively.
Time frame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Change in the 36-Item Short Form Health Survey (SF-36)
The 36-Item Short Form Health Survey (SF-36) is a 36-item, patient-reported survey of health status. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol. No derived scores were calculated for SF-36 and no summary tabulation was done.
Time frame: Change from Baseline in IBM-OLE to Early Termination Visit
Number of Falls and Near Falls
Patients recorded the number of falls and near falls in a falls diary. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol. No tabulation of falls and near falls was performed.
Time frame: Baseline to Early Termination Visit