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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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EUCTR |
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Last refreshed on:
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25 November 2019 |
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Main ID: |
EUCTR2008-008208-42-GB |
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Date of registration:
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29/05/2009 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Randomised, Double-Blind Placebo-Controlled Pilot Study Assessing the Safety and Tolerability of Arimoclomol in Adult with Sporadic Inclusion Body Myositis - Arimoclomol for Inclusion Body Myositis
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Scientific title:
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A Randomised, Double-Blind Placebo-Controlled Pilot Study Assessing the Safety and Tolerability of Arimoclomol in Adult with Sporadic Inclusion Body Myositis - Arimoclomol for Inclusion Body Myositis |
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Date of first enrolment:
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26/06/2009 |
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Target sample size:
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24 |
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Recruitment status: |
Not Recruiting |
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URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-008208-42 |
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Study type:
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Interventional clinical trial of medicinal product |
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Study design:
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Controlled: yes
Randomised: yes
Open: no
Single blind: no
Double blind: yes
Parallel group: yes
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: yes
Other: no
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): yes
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): no
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Countries of recruitment
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United Kingdom
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Contacts
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Key inclusion & exclusion criteria
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Inclusion criteria:
1. Meet the diagnostic criteria for definite or probable IBM (Griggs 1995)
2. Muscle function adequate for quantitative muscle testing. At least 8 of the following 16 muscle groups must have a MMT muscle grade greater or equal 3- or greater on the modified Medical Research Council Scale: Shoulder abductors, Elbow Flexors, Elbow Extensors, Wrist Flexors, Knee Extensors, Ankle Dorsiflexors, Knee Flexors, Neck Flexors (unilateral) and Neck Extensors (unilateral).
3. Age>50 years
4. Women must be post-menopausal (no menses in > 12 months) or status - post hysterectomy
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
Exclusion criteria:
1. 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 of central nervous system demyelination, or other chronic serious medical illnesses
2. Presence of any of the following on routine blood screening: WBC<3000/cm3, platelets<100,000/cm3, hematocrit < 30 %, urea > 10 millimoles/litre, creatinne > 150 micromoles/litre, symptomatic liver disease with serum albumin < 30g/L, prothrombin time or activated partial thromboplastin time > upper range of control values
3. Currently taking Riluzole
4. Women who are pregnant or lactating
5. History of non-compliance with other therapies
6. Coexistence of other neuromuscular disease
7. Drug or alcohol abuse within last 3 months
8. Inability to give informed consent
9. Known bleeding disorder
10. Use of potentially renal-toxic drugs
11. Prior difficulties with local anesthetic
Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
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Health Condition(s) or Problem(s) studied
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The research project proposes to investigate Sporadic Inclusion Body Myositis (IBM).
IBM is the commonest acquired disease of muscle affecting people aged 50 years and over. It is a progressive and debilitating disease which causes both muscle weakness and wasting, characteristically of the quadriceps and finger flexors. Over time it can lead to severe disability, falls and swallowing impairment. IBM is currently without a clinically proven, effective pharmacological treatment.
MedDRA version: 9.1
Level: LLT
Classification code 10066407
Term: Inclusion body myositis
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Intervention(s)
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Product Name: Arimoclomol
Product Code: BRX-345
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Arimoclomol
CAS Number: 289893-25-0
Current Sponsor code: BRX - 345
Other descriptive name: Arimoclomol
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use
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Primary Outcome(s)
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Main Objective: The primary objective is to assess the safety and tolerability of Arimoclomol taken as a 100 mg capsule, three times per day, for four months as compared with placebo in 12 patients with Sporadic Inclusion Body Myositis.
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Primary end point(s): The Primary outcome measures will be adverse event reporting. This would include significant treatment emergent adverse events or abnormalities on physical examination, vital signs, electocardiogram and laboratory findings including clinical chemistry, haematology and urinalysis.
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Secondary Objective: Secondary research objectives include the following outcome measures:
1. Muscle strength (assessed by muscle testing and also by quantitative myometry)
2. Muscle mass measures (by DEXA Scan)
3. IBM Functional Rating Scale
4. Concentrations of Heat Shock Protein 70 in muscle tissue, before and after treatment with arimoclomol
5. Change in microscopic pathological features of muscle tissue
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Source(s) of Monetary Support
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Ethics review
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Status: Approved
Approval date:
Contact:
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