Clinical trial NCT04049097: 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
Overall status: TERMINATED
Phase: PHASE3
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: 2019-08-07
Enrollment: 121
Sex: ALL
Minimum age (years): 45
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

1. Patient is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures.
2. Patient has completed the IBM4809 trial on treatment with Investigational Medicinal Product (IMP).

Exclusion Criteria:

1. Known or suspected allergy or intolerance to arimoclomol or its constituents.
2. 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.
3. Significant protocol deviation in the blinded IBM4809 trial based on the investigator's judgement in discussion with the medical monitor.
4. 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).
5. Any concurrent condition that in the investigator's opinion will significantly interfere with assessment of safety or efficacy.
6. Inability to comply with the protocol-specified procedures/evaluations and scheduled visits as per the investigator.

Primary outcomes:
measure: Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
timeFrame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Secondary outcomes:
measure: Change in Six Minutes Walking Distance Test; Distance at 6 Minutes (6MWD)
timeFrame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
measure: Change in Modified Timed Up and Go (mTUG)
timeFrame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
measure: Change in Quadriceps Muscle Strength
timeFrame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
measure: Change in Hand Grip Strength
timeFrame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
measure: Change in the 36-Item Short Form Health Survey (SF-36)
timeFrame: Change from Baseline in IBM-OLE to Early Termination Visit
measure: Number of Falls and Near Falls
timeFrame: Baseline to Early Termination Visit
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Neurological Associates
state: Arizona
city: Irvine
country: United States
facility: University of California
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 M Dimachkie
role: PRINCIPAL_INVESTIGATOR
affiliation: University College, London
name: Michael Hanna
role: PRINCIPAL_INVESTIGATOR
References: