NCT00769860

Brief Summary

Inclusion body myositis (IBM) is the most common progressive and debilitating muscle disease beginning in persons over 50 years of age. This study will assess the safety and tolerability of Arimoclomol in IBM as compared to placebo over 4 months of treatment.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Sep 2008

Typical duration for phase_2

Geographic Reach
2 countries

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2008

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

October 8, 2008

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 9, 2008

Completed
3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2012

Completed
4.4 years until next milestone

Results Posted

Study results publicly available

January 19, 2017

Completed
Last Updated

January 19, 2017

Status Verified

November 1, 2016

Enrollment Period

4 years

First QC Date

October 8, 2008

Results QC Date

November 22, 2016

Last Update Submit

November 22, 2016

Conditions

Inclusion Body Myositis

Keywords

myositisIBMinclusion body myositis

Outcome Measures

Primary Outcomes (1)

  • Count of Adverse Events Reported

    Measure reflects the total number of adverse events reported during course of the study.

    Month 12

Secondary Outcomes (10)

  • Heat Shock Protein 70 (HSP70) Levels in the Tissue

    Change from Baseline to Month 4

  • Muscle Strength Testing

    Change from Baseline to Month 4

  • Muscle Strength Testing

    Change from Baseline to Month 8

  • Muscle Strength Testing

    Change from Baseline to Month 12

  • Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score

    Change from Baseline to Month 4

  • +5 more secondary outcomes

Study Arms (2)

1

ACTIVE COMPARATOR

Arimoclomol

Drug: Arimoclomol

2

PLACEBO COMPARATOR
Other: Placebo

Interventions

ArimoclomolDRUG

Arimoclomol 100 mg TID for 4 months

1
PlaceboOTHER

Placebo for 4 months

2

Eligibility Criteria

Age50 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Meet the diagnostic criteria for definite or probable IBM (Griggs 1995)
  • Muscle function adequate for quantitative muscle testing
  • Age \> 50 years
  • Women must be postmenopausal or status post hysterectomy
  • For any patient currently taking medication for IBM, they must remain on current dosage for the extent of the study and last dosage change must be \> 30 days previous to enrollment

You may not qualify if:

  • Presence of any one of the following medical conditions: diabetes mellitus or patients taking anti-diabetic medications, chronic infection, chronic renal insufficiency, cancer other than skin cancer less than 5 years previously, multiple sclerosis or prior episode or central nervous system demyelination, or other chronic serious medical illnesses
  • Presence of any of the following on routine blood screening: WBC \< 3000, platelets \< 100,000, hematocrit \< 30%, BUN \> 30 mg%, creatine \> 1.5 mg%, symptomatic liver disease with serum albumin \< 3 g/dl, PT or PTT \> upper range of control values
  • Women who are pregnant or lactating
  • History of non-compliance with other therapies
  • Coexistence of other neuromuscular disease
  • Drug or alcohol abuse within the last 3 months
  • Inability to give informed consent
  • Known bleeding disorder
  • Use of potentially renal toxic drugs
  • Prior difficulties with local anesthetic

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

University College London, MRC Centre for Neuromuscular Disease

London, United Kingdom

Location

MeSH Terms

Conditions

Myositis, Inclusion BodyMyositis

Interventions

arimoclomol

Condition Hierarchy (Ancestors)

Muscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System Diseases

Results Point of Contact

Title
Dr. Richard Barohn
Organization
University of Kansas Medical Center
RBAROHN@kumc.edu
(913) 588-6094

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Gertrude and Dewey Zeigler Professor of Neurology and Chair

Study Record Dates

First Submitted

October 8, 2008

First Posted

October 9, 2008

Study Start

September 1, 2008

Primary Completion

September 1, 2012

Study Completion

September 1, 2012

Last Updated

January 19, 2017

Results First Posted

January 19, 2017

Record last verified: 2016-11

Locations

GB(1)US(1)