Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial

NCT ID: NCT04049097

Last Updated: 2023-09-15

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 121 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-05-20
• Study Completion Date: 2021-11-15

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.

Conditions

Inclusion Body Myositis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Arimoclomol

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

Group Type: EXPERIMENTAL

Arimoclomol

Intervention Type: 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

Interventions

Arimoclomol

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

Intervention Type: DRUG

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.

• Minimum Eligible Age: 45 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Kansas Medical Center

OTHER

Sponsor Role: collaborator

University College, London

OTHER

Sponsor Role: collaborator

ZevraDenmark

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Mazen M Dimachkie

Role: PRINCIPAL_INVESTIGATOR

University of Kansas Medical Center

Michael Hanna

Role: PRINCIPAL_INVESTIGATOR

University College, London

Locations

Phoenix Neurological Associates

Phoenix, Arizona, United States

University of California

Irvine, California, United States

University of Colorado School of Medicine

Aurora, Colorado, United States

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

University of Virginia

Charlottesville, Virginia, United States

University College of London

London, , United Kingdom

Countries

United States; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

IBM-OLE

Identifier Type: -

Identifier Source: org_study_id