Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial

Study Purpose

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 sIBM

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	45 Years and Over
Gender	All

More Inclusion & Exclusion 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 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 Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT04049097
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 3
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Orphazyme
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Mazen M DimachkieMichael Hanna
Principal Investigator Affiliation	University of Kansas Medical CenterUniversity College, London
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry, Other
Overall Status	Enrolling by invitation
Countries	United Kingdom, United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Inclusion Body Myositis

Arms & Interventions

Arms

Experimental: Arimoclomol

1200 mg/day arimoclomol citrate (400 mg t.i.d.)

Interventions

Drug: - Arimoclomol

1200 mg/day arimoclomol citrate (400 mg t.i.d.)

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Phoenix Neurological Associates, Phoenix, Arizona

Status

Address

Phoenix Neurological Associates

Phoenix, Arizona, 85018

Aurora, Colorado

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Address

University of Colorado School of Medicine

Aurora, Colorado, 80045

University of Kansas Medical Center, Kansas City, Kansas

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Address

University of Kansas Medical Center

Kansas City, Kansas, 66160

Johns Hopkins University, Baltimore, Maryland

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Address

Johns Hopkins University

Baltimore, Maryland, 21218

Brigham and Women's Hospital, Boston, Massachusetts

Status

Address

Brigham and Women's Hospital

Boston, Massachusetts, 02115

University of Rochester, Rochester, New York

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University of Rochester

Rochester, New York, 14642

The Ohio State University, Columbus, Ohio

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Address

The Ohio State University

Columbus, Ohio, 43221

Nerve and Muscle Center of Texas, Houston, Texas

Status

Address

Nerve and Muscle Center of Texas

Houston, Texas, 77030

University of Utah, Salt Lake City, Utah

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Address

University of Utah

Salt Lake City, Utah, 84112

University of Virginia, Charlottesville, Virginia

Status

Address

University of Virginia

Charlottesville, Virginia, 22908

International Sites

University College of London, London, United Kingdom

Status

Address

University College of London

London, , WC1N 3BG

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