Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial

NCT04049097 | Terminated Phase 3 Results FDA Drug

• 1 other identifier: IBM-OLE
• Study Type: interventional
• Target: 121
• Locations: 2 countries
• Sites: 12

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)

Conditions

Inclusion Body Myositis

Keywords

IBM

Outcome Measures

Primary Outcomes (1)

• 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.

  Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).

Secondary Outcomes (6)

• Change in Six Minutes Walking Distance Test; Distance at 6 Minutes (6MWD)

  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)

  Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).

• Change in Quadriceps Muscle Strength

  Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).

• Change in Hand Grip Strength

  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)

  Change from Baseline in IBM-OLE to Early Termination Visit

Study Arms (1)

Arimoclomol

EXPERIMENTAL

248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily

Drug: Arimoclomol

Interventions

Arimoclomol DRUG

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

Arimoclomol

Eligibility Criteria

• Age: 45 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• ZevraDenmark: lead
• University of Kansas Medical Center: collaborator
• University College, London: collaborator

Study Sites (12)

Phoenix Neurological Associates

Phoenix, Arizona, 85018, United States

Location

University of California

Irvine, California, 92697, United States

Location

University of Colorado School of Medicine

Aurora, Colorado, 80045, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21218, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Rochester

Rochester, New York, 14642, United States

Location

The Ohio State University

Columbus, Ohio, 43221, United States

Location

Nerve and Muscle Center of Texas

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84112, United States

Location

University of Virginia

Charlottesville, Virginia, 22908, United States

Location

University College of London

London, WC1N 3BG, United Kingdom

Location

MeSH Terms

Conditions

Myositis, Inclusion Body

Interventions

arimoclomol

Condition Hierarchy (Ancestors)

Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Results Point of Contact

• Title: Medical Affairs
• Organization: Zevra Denmark A/S

medicalaffairs@zevra.com

+1-888-289-5607

Study Officials

• Mazen M Dimachkie

  University of Kansas Medical Center

  PRINCIPAL INVESTIGATOR

• Michael Hanna

  University College, London

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 1, 2019
• First Posted: August 7, 2019
• Study Start: May 20, 2019
• Primary Completion: November 15, 2021
• Study Completion: November 15, 2021
• Last Updated: September 15, 2023
• Results First Posted: September 15, 2023

Record last verified: 2023-08

Locations

GB (1); US (11)