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

Phase 1

• Conditions: Sporadic Inclusion Body Myositis (sIBM); MedDRA version: 21.1Level: LLTClassification code 10075052Term: Sporadic inclusion body myositisSystem Organ Class: 100000004859; Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

• Registration Number: EUCTR2019-000749-11-GB
• Lead Sponsor: Orphazyme A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-06-20
• Last Update Posted: 9 November 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 130

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 IMP.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 75
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 75

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 sexually active female subjects of child-bearing potential* intending to become pregnant or unwilling to use a highly effective method of contraception** during the trial through 1 month after the last dose of trial medication. Sexually active males with female partners of child-bearing potential* unwilling to use a condom with or without spermicide in addition to the birth control used by their partners during the trial until 3 months after the last dose of trial medication unless surgically sterile (vasectomy).
* Non child-bearing potential is defined as post-menopausal (minimum of 12 months with no menses and follicle-stimulating hormone in the post-menopausal range) or sterilisation (hysterectomy, oophorectomy, or bilateral tubal ligation).
** Highly effective methods of contraception include combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable); intrauterine device; intrauterine hormone-releasing system; bilateral tubal occlusion; and vasectomised partner.
According to the recommendations from the Clinical Trial Facilitation Group (CTFG, 2014), sexual abstinence is considered a highly effective birth control method only if it is defined as refraining from heterosexual intercourse during the trial until 1 month after the last dose of trial medication (for female subjects of child-bearing potential) and for 3 months after the last dose of trial medication (for male subjects with female partners of child-bearing potential). The reliability of sexual abstinence needs to be evaluated by the investigator in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject.
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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To determine the efficacy (based on the Inclusion Body Myositis Functional Rating Scale [IBMFRS]) of early versus delayed start (i.e., at 20 months) of arimoclomol treatment of sIBM.;Secondary Objective: Secondary:<br>• To determine the safety and tolerability of long-term treatment of sIBM with arimoclomol<br>• To determine the efficacy (on secondary efficacy endpoints) of early versus delayed start of arimoclomol treatment of sIBM<br>Exploratory:<br>• To establish the long-term efficacy of arimoclomol as determined by magnetic resonance imaging (MRI) parameters.<br>• To explore population pharmacokinetics (popPK) and popPK/PD.;Primary end point(s): Efficacy endpoints:<br>• Primary efficacy endpoint: Change from baseline to Month 20 in the IBMFRS total score<br>;Timepoint(s) of evaluation of this end point: Endpoint evaluation will be checked at every visit for Efficacy.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • Secondary efficacy endpoints: Change from baseline to Month 20 in secondary efficacy parameters<br>Exploratory endpoints:<br>• Population pharmacokinetics (popPK) and popPK/PD (reported separately)<br>Safety endpoints:<br>• Safety parameters (AEs, SAEs, clinical safety laboratory values, vital signs, C-SSRS) assessed throughout the extension trial;Timepoint(s) of evaluation of this end point: Endpoint evaluation will be checked at every visit for Safety.<br>Exploratory endpoints will be evaluated at Month 9.