Back to resultsArimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
Primary Purpose
Inclusion Body Myositis
Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Arimoclomol
Sponsored by
About this trial
This is an interventional treatment trial for Inclusion Body Myositis focused on measuring IBM
Eligibility Criteria
Inclusion Criteria:
- Patient is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures.
- Patient has completed the IBM4809 trial on treatment with IMP. -
Exclusion Criteria:
- Known or suspected allergy or intolerance to arimoclomol or its constituents.
- Exposure to any other investigational treatment within 30 days or <5 half-lives of the baseline visit or taking part or planning to take part in another interventional trial.
- Significant protocol deviation in the blinded IBM4809 trial based on the investigator's judgement in discussion with the medical monitor.
Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants up to 3 months after last dose. Premenopausal women must have a negative pregnancy test prior to dosing with trial medication. Acceptable methods of birth control are:
- Hormonal methods associated with inhibition of ovulation such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the patient's usual menstrual cycle period) before arimoclomol administration.
- Total abstinence from sexual intercourse since the last menses before arimoclomol administration. (The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient. Periodic abstinence [calendar, symptothermal, post-ovulation] methods are not acceptable methods of contraception).
- Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS).
- Any concurrent condition that in the investigator's opinion will significantly interfere with assessment of safety or efficacy.
- Inability to comply with the protocol-specified procedures/evaluations and scheduled visits as per the investigator.
Sites / Locations
- Phoenix Neurological Associates
- University of California
- University of Colorado School of Medicine
- University of Kansas Medical Center
- Johns Hopkins University
- Brigham and Women's Hospital
- University of Rochester
- The Ohio State University
- Nerve and Muscle Center of Texas
- University of Utah
- University of Virginia
- University College of London
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Arimoclomol
Arm Description
248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
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.
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.
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.
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.
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.
Full Information
NCT ID
NCT04049097
First Posted
April 1, 2019
Last Updated
August 22, 2023
Sponsor
ZevraDenmark
Collaborators
University of Kansas Medical Center, University College, London
1. Study Identification
Unique Protocol Identification Number
NCT04049097
Brief Title
Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
Official Title
An Open-label, Non-randomized Trial to Investigate the Efficacy and Safety of Early Versus Delayed Start of Arimoclomol in Patients With Sporadic Inclusion Body Myositis Who Have Completed the IBM4809 Trial
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Terminated
Why Stopped
As a consequence of the results of IBM4809 which did not meet any of its efficacy endpoints. The planned duration of open-label treatment was 40 months. After termination, the actual mean duration of treatment was approx. 28 weeks.
Study Start Date
May 20, 2019 (Actual)
Primary Completion Date
November 15, 2021 (Actual)
Study Completion Date
November 15, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ZevraDenmark
Collaborators
University of Kansas Medical Center, University College, London
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
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)
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Inclusion Body Myositis
Keywords
IBM
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
121 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Arimoclomol
Arm Type
Experimental
Arm Description
248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
Intervention Type
Drug
Intervention Name(s)
Arimoclomol
Intervention Description
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
Primary Outcome Measure Information:
Title
Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
Description
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).
Secondary Outcome Measure Information:
Title
Change in Six Minutes Walking Distance Test; Distance at 6 Minutes (6MWD)
Description
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).
Title
Change in Modified Timed Up and Go (mTUG)
Description
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).
Title
Change in Quadriceps Muscle Strength
Description
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).
Title
Change in Hand Grip Strength
Description
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).
Title
Change in the 36-Item Short Form Health Survey (SF-36)
Description
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
Title
Number of Falls and Near Falls
Description
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
45 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patient is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures.
Patient has completed the IBM4809 trial on treatment with Investigational Medicinal Product (IMP).
Exclusion Criteria:
Known or suspected allergy or intolerance to arimoclomol or its constituents.
Exposure to any other investigational treatment within 30 days or <5 half-lives of the baseline visit or taking part or planning to take part in another interventional trial.
Significant protocol deviation in the blinded IBM4809 trial based on the investigator's judgement in discussion with the medical monitor.
Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants up to 3 months after last dose. Premenopausal women must have a negative pregnancy test prior to dosing with trial medication. Acceptable methods of birth control are:
Hormonal methods associated with inhibition of ovulation such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the patient's usual menstrual cycle period) before arimoclomol administration.
Total abstinence from sexual intercourse since the last menses before arimoclomol administration. (The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient. Periodic abstinence [calendar, symptothermal, post-ovulation] methods are not acceptable methods of contraception).
Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS).
Any concurrent condition that in the investigator's opinion will significantly interfere with assessment of safety or efficacy.
Inability to comply with the protocol-specified procedures/evaluations and scheduled visits as per the investigator.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mazen M Dimachkie
Organizational Affiliation
University of Kansas Medical Center
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Michael Hanna
Organizational Affiliation
University College, London
Official's Role
Principal Investigator
Facility Information:
Facility Name
Phoenix Neurological Associates
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85018
Country
United States
Facility Name
University of California
City
Irvine
State/Province
California
ZIP/Postal Code
92697
Country
United States
Facility Name
University of Colorado School of Medicine
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Johns Hopkins University
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21218
Country
United States
Facility Name
Brigham and Women's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
University of Rochester
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
The Ohio State University
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43221
Country
United States
Facility Name
Nerve and Muscle Center of Texas
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
University of Virginia
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
University College of London
City
London
ZIP/Postal Code
WC1N 3BG
Country
United Kingdom
12. IPD Sharing Statement
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Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
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