Study of Arimoclomol in Inclusion Body Myositis (IBM)

Sponsor

KemPharm Denmark A/S (Industry)

Overall Status

Completed

CT.gov ID

NCT02753530

Collaborator

University of Kansas Medical Center (Other), University College, London (Other)

152

Enrollment

Locations

Arms

40.9

Actual Duration (Months)

12.7

Patients Per Site

0.3

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Funding Source - FDA OOPD. The purpose of this study is to evaluate the safety and efficacy of the study drug, Arimoclomol in IBM patients.

Condition or Disease	Intervention/Treatment	Phase
Inclusion Body Myositis	Drug: Arimoclomol Other: Placebo	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

152 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Double (Participant, Investigator)

Primary Purpose:

Treatment

Official Title:

Phase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM)

Actual Study Start Date :

Aug 16, 2017

Actual Primary Completion Date :

Jan 11, 2021

Actual Study Completion Date :

Jan 11, 2021

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Arimoclomol Participants will be asked to take 400mg arimoclomol three times a day.	Drug: Arimoclomol 2 200mg pills taken 3 times per day during breakfast, early afternoon and at bedtime
Placebo Comparator: Placebo Participants will be asked to take 400mg placebo three times a day.	Other: Placebo 2 matched placebo pills to be taken 3 times per day during breakfast, early afternoon and at bedtime

Arm

Intervention/Treatment

Experimental: Arimoclomol

Participants will be asked to take 400mg arimoclomol three times a day.

Drug: Arimoclomol

2 200mg pills taken 3 times per day during breakfast, early afternoon and at bedtime

Placebo Comparator: Placebo

Participants will be asked to take 400mg placebo three times a day.

Other: Placebo

2 matched placebo pills to be taken 3 times per day during breakfast, early afternoon and at bedtime

Outcome Measures

Primary Outcome Measures

Decline in Inclusion body myositis functional rating scale (IBMFRS) [Change from Baseline to Month 20]
Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.

Secondary Outcome Measures

Manual Muscle Testing (MMT) [Change from Baseline to Month 20]
Maximum voluntary isometric contraction (MVICT) of quadriceps [Change from Baseline to Month 20]
Grip and pinch test [Change from Baseline to Month 20]
modified timed up and go (mTUG) [Change from Baseline to Month 20]
6 minute walk test with 2 minute distance captured [Change from Baseline to Month 20]
Health Assessment Questionnaire (HAQ-DI) [Change from Baseline to Month 20]
SF-36 [Change from Baseline to Month 20]
Measured using the Short form health survey with 36 items (SF-36).
Count of adverse events [Change from Baseline to Month 20]
Decline in Inclusion body myositis functional rating scale (IBMFRS) [Change from Baseline to Month 12]
Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.

Eligibility Criteria

Criteria

Ages Eligible for Study:

45 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Meet any of the European Neuromuscular Centre Inclusion Body Myositis research diagnostic criteria 2011 categories for IBM
Demonstrate being able to arise from a chair without support from another person or device
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, can, crutches, or braces. They cannot be supported by another person and cannot use furniture or wall for support.
Body weight of >= 40 kg
Pre-menopausal women must have a negative pregnancy tst prior to dosing iwht study medication.
If a participant in the bimagrumab study, the participant must be off of the study medication for at least 6 months.
Able to give informed consent

Exclusion Criteria:

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.
Presence of any of the following on routine blood screening: WEB <3000; platelets < 100,000; hematocrit , 30%; BUN > 30 mg%; creatinine > 1.5 mg%; symptomatic liver disease with serum albumin < 3 g/dL.
History of most recent creatine kinase >15x the upper limit of normal without any other explanation besides IBM.
History of non-compliance with other therapies
Use of testosterone except for physiologic replacement doses in case of androgen deficiency. Participants must have documented proof of the androgen deficiency.
Coexistence of other disease that would be likely to affect outcome measures.
Drug or alcohol abuse within past three months
Participation in a recent drug study in the last 30 days prior to the screening visit or use of a biologic agent less than 6 months prior to the screening visit.
Women who are lactating or unwilling to use adequate method of birth control who are not surgically sterile. Adequate birth control includes use of intrauterine device, abstinence, or oral contraceptives or a double barrier method, e.g condom plas diaphragm will be necessary for both male and female participants.
Participants taking >7.5 mg prednisolone or equivalent or participants on IVIg or other immunosuppressants within the last 3 months.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Phoenix Neurological Associates	Phoenix	Arizona	United States	85018
2	University of California, Irvine	Irvine	California	United States	92697
3	University of Colorado School of Medicine	Aurora	Colorado	United States	80045
4	University of Kansas Medical Center	Kansas City	Kansas	United States	66160
5	Johns Hopkins University	Baltimore	Maryland	United States	21218
6	Brigham and Women's Hospital	Boston	Massachusetts	United States	02115
7	University of Rochester	Rochester	New York	United States	14642
8	The Ohio State University	Columbus	Ohio	United States	43221
9	Nerve and Muscle Center of Texas	Houston	Texas	United States	77030
10	University of Utah	Salt Lake City	Utah	United States	84112
11	University of Virginia	Charlottesville	Virginia	United States	22908
12	University College of London	London		United Kingdom	WC1N 3BG

Sponsors and Collaborators

KemPharm Denmark A/S
University of Kansas Medical Center
University College, London

Investigators

Principal Investigator: Mazen Dimachkie, MD, University of Kansas Medical Center
Principal Investigator: Michael Hanna, MD, University College, London

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

KemPharm Denmark A/S

ClinicalTrials.gov Identifier:

NCT02753530

Other Study ID Numbers:

STUDY00002461
R01FD004809

First Posted:

Apr 28, 2016

Last Update Posted:

Mar 24, 2021

Last Verified:

Mar 1, 2021

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Myositis

Myositis, Inclusion Body

Study Results

No Results Posted as of Mar 24, 2021