Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial

Zevra Therapeutics

Status and phase: Terminated; Phase 3

Conditions: Inclusion Body Myositis

Treatments: Drug: Arimoclomol

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT04049097

IBM-OLE

Details and patient eligibility

About

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)

Full 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.

Enrollment: 121 patients

Sex: All

Ages: 45+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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121 participants in 1 patient group

Arimoclomol

Experimental group

Description:

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

Treatment:

Drug: Arimoclomol