E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10066407 |
E.1.2 | Term | Inclusion body myositis |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
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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E.2.2 | Secondary objectives of the trial |
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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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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
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E.4 | Principal 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 |
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E.5 End points |
E.5.1 | 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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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | No |
E.8.5.1 | Number of sites anticipated in the EEA | 1 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The end of the trial will be defined as the completion of all trial procedures by the participants (ie. the last follow-up visit of the last subject undergoing the trial). |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |