Inclusion Body MyositisPhase 2/3NCT00769860
Arimoclomol in Sporadic Inclusion Body Myositis
A Phase 2/3 study testing Arimoclomol in people with Inclusion Body Myositis, run by Richard Barohn, MD at 2 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Inclusion Body Myositis
- What is being tested?
- Arimoclomol
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Sep 2008to Sep 2012
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 10Show fewer
- Inability to give informed consent
- Known bleeding disorder
- Use of potentially renal toxic drugs
- Prior difficulties with local anesthetic
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
University of Kansas Medical Center
Kansas City, Kansas, 66160
United Kingdom1 site
University College London, MRC Centre for Neuromuscular Disease
London
About this study
Official title: Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis
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.
Read the detailed description
IBM is a chronic disorder in which muscles become inflamed (swollen) and cause muscle weakening. The cause is unknown. There is new evidence to suggest that the pathology in IBM results from cellular changes induced by a variety of stressful events and diseases. In response to these stressful events the body's normal response is to increase the levels of Heat Shock Proteins (HSP) to help counteract and stop these cellular changes. In people with IBM this increase does not appear sufficient enough to reverse these toxic cellular changes. Arimoclomol causes the body to make more of this HSP protein. By increasing HSP levels in IBM patients we hope to reverse the toxic cellular changes that might be responsible for the pathology of IBM.
- Conditions
- Inclusion Body Myositis
- Being tested
- Arimoclomol, Placebo
- Keywords
- myositis, IBM, inclusion body myositis
- Medical subject headings
- Myositis, Inclusion Body, Myositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ArimoclomolDrug
Arimoclomol 100 mg TID for 4 months
PlaceboOther
Placebo for 4 months
| Study group | Role | What they receive |
|---|---|---|
| 1 | Active comparator | Arimoclomol Receives: Drug: Arimoclomol |
| 2 | Placebo comparator | Not described Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Count of Adverse Events Reported
Measure reflects the total number of adverse events reported during course of the study.
Measured: Month 12
Secondary outcomes (10)
Heat Shock Protein 70 (HSP70) Levels in the Tissue
Biopsy taken from participants at baseline and month 4 visits. Measured change in HSP70 levels in the tissue.
Measured: Change from Baseline to Month 4
Muscle Strength Testing
Change in MMT score for the period. The MMT score range is 0-5. A score of 0 is the lowest and represents no visible or palpable contraction. A score of 5 is the highest and represents full range of motion against gravity, maximal resistance.
Measured: Change from Baseline to Month 4
Muscle Strength Testing
Change in MMT score for the period. The MMT score range is 0-5. A score of 0 is the lowest and represents no visible or palpable contraction. A score of 5 is the highest and represents full range of motion against gravity, maximal resistance.
Measured: Change from Baseline to Month 8
Muscle Strength Testing
Change in MMT score for the period. The MMT score range is 0-5. A score of 0 is the lowest and represents no visible or palpable contraction. A score of 5 is the highest and represents full range of motion against gravity, maximal resistance.
Measured: Change from Baseline to Month 12
Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Measured change for the period. The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient.
Measured: Change from Baseline to Month 4
Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Measured change for the period. The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient.
Measured: Change from Baseline to Month 8
Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Measured change for the period. The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient.
Measured: Change from Baseline to Month 12
Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Measured MVICT using the Quantitative Muscle Assessment (QMA) system designed by Computer Source, Atlanta, GA. The system uses an adjustable cuff to attach the patient's arm or leg to an inelastic strap that is connected to force transducer with a load of 0.5 to 1,000 Newtons. Maximum force is recorded.
Measured: Change from Baseline to Month 4
Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Measured MVICT using the Quantitative Muscle Assessment (QMA) system designed by Computer Source, Atlanta, GA. The system uses an adjustable cuff to attach the patient's arm or leg to an inelastic strap that is connected to force transducer with a load of 0.5 to 1,000 Newtons. Maximum force is recorded.
Measured: Change from Baseline to Month 8
Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Measured MVICT using the Quantitative Muscle Assessment (QMA) system designed by Computer Source, Atlanta, GA. The system uses an adjustable cuff to attach the patient's arm or leg to an inelastic strap that is connected to force transducer with a load of 0.5 to 1,000 Newtons. Maximum force is recorded.
Measured: Change from Baseline to Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2008 (estimated)
- Study startSep 2008
- Primary completionSep 2012
- Study completionSep 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Inclusion Body MyositisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Richard Barohn, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Richard Barohn, MD, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00769860
- Sponsor's protocol ID
- 10656
- Status last verified
- Nov 2016
- First submitted
- 8 Oct 2008
- Last updated
- 19 Jan 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis
- ClinicalTrials.gov ID
- NCT00769860
- Acronym / sponsor ID
- 10656
- Conditions
- Inclusion Body Myositis
- Keywords
- myositis, IBM, inclusion body myositis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Richard Barohn, MDUniversity, hospital or other
- Study start
- Sep 2008
- Primary completion
- Sep 2012 (actual)
- Study completion
- Sep 2012 (actual)
- First posted
- 9 Oct 2008 (estimated)
- Results
- Posted 19 Jan 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
IBM is a chronic disorder in which muscles become inflamed (swollen) and cause muscle weakening. The cause is unknown. There is new evidence to suggest that the pathology in IBM results from cellular changes induced by a variety of stressful events and diseases. In response to these stressful events the body's normal response is to increase the levels of Heat Shock Proteins (HSP) to help counteract and stop these cellular changes. In people with IBM this increase does not appear sufficient enough to reverse these toxic cellular changes. Arimoclomol causes the body to make more of this HSP protein. By increasing HSP levels in IBM patients we hope to reverse the toxic cellular changes that might be responsible for the pathology of IBM.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Active comparator | Drug: ArimoclomolArimoclomol |
| 2 | Placebo comparator | Other: Placebo |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ArimoclomolArimoclomol 100 mg TID for 4 months | Drug | 1 |
| PlaceboPlacebo for 4 months | Other | 2 |
Primary outcome
Count of Adverse Events Reported
Time frame: Month 12
Measure reflects the total number of adverse events reported during course of the study.
10 secondary outcomes
Heat Shock Protein 70 (HSP70) Levels in the Tissue
Time frame: Change from Baseline to Month 4
Muscle Strength Testing
Time frame: Change from Baseline to Month 4
Muscle Strength Testing
Time frame: Change from Baseline to Month 8
Muscle Strength Testing
Time frame: Change from Baseline to Month 12
Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Time frame: Change from Baseline to Month 4
Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Time frame: Change from Baseline to Month 8
Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Time frame: Change from Baseline to Month 12
Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Time frame: Change from Baseline to Month 4
Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Time frame: Change from Baseline to Month 8
Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Time frame: Change from Baseline to Month 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jan 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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The Aim of the Study is to Characterize Patients Exhibiting Muscular Atrophy and Weakness Who Require a Muscle Biopsy for Diagnosis (IBM vs Atypical MND), Through an Innovative Technique Which Enables the Evaluation of the Neuromuscular Junction Involvement in Each Condition
Inclusion Body Myositis (IBM), Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Arimoclomol in people with Inclusion Body Myositis, run by Richard Barohn, MD at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Jan 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Count of Adverse Events Reported (time frame: Month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Jan 2017.