Study of Arimoclomol in patients with Inclusion Body Myositis (IBM)

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: EUCTR2017-004903-33-GB
• Lead Sponsor: Orphazyme A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-04-05
• Last Update Posted: 29 June 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

1. Meet any of the European Neuromuscular Centre Inclusion Body Myositis research diagnostic criteria 2011 categories for IBM.1 (as per Appendix 1 of the protocol)
2. Demonstrate being able to arise from a chair without support from another person or device
3. Able to ambulate at least 20 ft/6 meters with or without assistive device. Once arisen from the chair, participant may use any walking device, i.e. walker/frame, cane, crutches, or braces. They cannot be supported by another person and cannot use furniture or wall for support.
4. Age at onset of weakness > 45 years
5. Body weight of > 40 kgs
6. Able to give informed consent

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. History of any of the following excludes subject participation in the study: chronic infection particularly HIV or Hepatitis B or C; cancer other than basal cell cancer less than five years prior, or other chronic serious medical illnesses.
2. Presence of any of the following on routine blood screening: WBC < 3000; platelets < 100,000; hematocrit < 30%; BUN > 30 mg; creatinine > 1.5 x upper limit of normal; symptomatic liver disease with serum albumin < 30mg/dl
3. History of most recent creatine kinase >15x the upper limit of normal without any other explanation besides IBM.
4. History of non-compliance with other therapies.
5. Use of testosterone except for physiologic replacement doses in case of androgen deficiency. Participants must have documented proof of the androgen deficiency.
6. Coexistence of any other disease that would likely to affect outcome measures.
7. Drug or alcohol abuse within past three months. Patient has recent history (within 6 months before Screening) of chronic alcohol or drug abuse which may compromise the patient’s safety or ability to participate in study activities. Cannabis for IBM symptoms will be allowed (where legal).
8. Participation in a recent drug study in the last 30 days prior to the screening visit or use of biologic agents less than 6 months prior to the screening visit.
9. 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.
10. Participants taking >7.5 mg prednisolone or equivalent or participants on intravenous immunoglobulin (IVIg) or other immunosuppressants within the last 3 months. Topical, nasal, and ocular corticosteroids are allowed unless they are being widely applied or

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of arimoclomol at a daily dosage of 1200 mg (400 mg t.i.d) compared to placebo in the treatment of sporadic IBM at 20 months. <br><br>;Secondary Objective: ;Primary end point(s): Primary efficacy endpoint is the change from baseline to Month 20 in the IBM Functional Rating Scale (IBMFRS) total score.<br>;Timepoint(s) of evaluation of this end point: Efficacy endpoint will be checked at Visit at Month 20<br><br>