Study of Arimoclomol in Inclusion Body Myositis (IBM)

Study Description

Go to 

Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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

Go to 

Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	150 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 :	August 9, 2017
Estimated Primary Completion Date :	December 2021
Estimated Study Completion Date :	December 2021

Arms and Interventions

Go to 

Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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

Go to 

Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :

1. Decline in Inclusion body myositis functional rating scale (IBMFRS) [ Time Frame: 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.

Secondary Outcome Measures :

1. Manual Muscle Testing (MMT) [ Time Frame: Change from Baseline to Month 20 ]
2. Maximum voluntary isometric contraction (MVICT) of quadriceps [ Time Frame: Change from Baseline to Month 20 ]
3. Grip and pinch test [ Time Frame: Change from Baseline to Month 20 ]
4. modified timed up and go (mTUG) [ Time Frame: Change from Baseline to Month 20 ]
5. 6 minute walk test with 2 minute distance captured [ Time Frame: Change from Baseline to Month 20 ]
6. Health Assessment Questionnaire (HAQ-DI) [ Time Frame: Change from Baseline to Month 20 ]
7. SF-36 [ Time Frame: Change from Baseline to Month 20 ]
   Measured using the Short form health survey with 36 items (SF-36).
8. Count of adverse events [ Time Frame: Change from Baseline to Month 20 ]
9. Decline in Inclusion body myositis functional rating scale (IBMFRS) [ Time Frame: 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.

Eligibility Criteria

Go to 

Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Ages Eligible for Study:	45 Years and older (Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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

Go to 

Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Contacts

Contact: Laura Herbelin	(913) 588-5095	LHERBELIN@kumc.edu

Locations

United States, Arizona
Phoenix Neurological Associates	Recruiting
Phoenix, Arizona, United States, 85018
Contact: Janira Ramos 602-258-2863 janira.ramos@honorhealth.com
Principal Investigator: David Saperstein, MD
United States, California
University of California, Irvine	Recruiting
Irvine, California, United States, 92697
Contact: Veena Mathew 714-456-2864 vmathew@uci.edu
Principal Investigator: Tahseen Mozaffar, MD
United States, Colorado
University of Colorado School of Medicine	Recruiting
Aurora, Colorado, United States, 80045
Contact: Nicola Haakonsen 303-724-4644 Nicola.Haakonsen@ucdenver.edu
Principal Investigator: Matthew P. Wicklund, MD
United States, Kansas
University of Kansas Medical Center	Recruiting
Kansas City, Kansas, United States, 66160
Contact: Laura Herbelin 913-588-5095 lherbelin@kumc.edu
Contact: Tina Liu 913-945-9932 tliu@kumc.edu
Principal Investigator: Mazen Dimachkie, MD
United States, Massachusetts
Brigham and Women's Hospital	Recruiting
Boston, Massachusetts, United States, 02115
Contact: Kristen Roe 617-525-6763 kroe@bwh.harvard.edu
Principal Investigator: Anthony Amato, MD
United States, New York
University of Rochester	Recruiting
Rochester, New York, United States, 14642
Contact: Liz Luebbe 585-275-7867 elizabeth_luebbe@urmc.rochester.edu
Principal Investigator: Emma Ciafaloni, MD
United States, Ohio
The Ohio State University	Recruiting
Columbus, Ohio, United States, 43221
Principal Investigator: Miriam Freimer, MD
United States, Texas
Nerve and Muscle Center of Texas	Recruiting
Houston, Texas, United States, 77030
Contact: Zinah Rasheed, BS 713-795-0033 ext 16 ataher@aol.com
Principal Investigator: Aziz Shaibani, MD
United States, Utah
University of Utah	Recruiting
Salt Lake City, Utah, United States, 84112
Contact: Crystal Neate 801-213-1018 crystal.neate@hsc.utah.edu
Principal Investigator: Summer Gibson, MD
United Kingdom
University College of London	Recruiting
London, United Kingdom, WC1N 3BG
Contact: Iwona Skorupinska, BSc, RGN 0203-108-7515 iwona.skorupinska@nhs.net
Principal Investigator: Pedro Machado, MD

Sponsors and Collaborators

Orphazyme

University of Kansas Medical Center

University College, London

Investigators

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

More Information

Go to 

Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Orphazyme
ClinicalTrials.gov Identifier:	NCT02753530 History of Changes
Other Study ID Numbers:	STUDY00002461; R01FD004809-01A2 ( U.S. FDA Grant/Contract )
First Posted:	April 28, 2016
Last Update Posted:	November 5, 2018
Last Verified:	November 2018

Studies a U.S. FDA-regulated Drug Product:		Yes
Studies a U.S. FDA-regulated Device Product:		No

Additional relevant MeSH terms:

Myositis; Myositis, Inclusion Body; Muscular Diseases	Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases