E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
HTLV-I-associated myelopathy |
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MedDRA Classification |
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 |
To determine in an open, pilot, proof of principle study whether ciclosporin A improves the clinical measures of patients with ‘early’ or ‘progressing’ ‘definite’ HAM/TSP |
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E.2.2 | Secondary objectives of the trial |
To determine in patients with HAM/TSP the safety of ciclosporin A the effect of ciclosporin A on viral load and expression the effect of ciclosporin A on markers of activation and proliferation To explore the mechanism of pathogenesis of HAM/TSP by understanding the effect of ciclosporin A on the severity and progression of HAM/TSP
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Have ‘definite’ HAM/TSP that is either early or progressing disease as defined below Are able to give informed consent Are 16 years or older
“HAM/TSP” Patients with HAM/TSP will be included if they fulfil the criteria of “Definite HAM/TSP” as agreed in Belem 2003. See annex.
“Early HAM/TSP” Less than 2 years history of disease, excluding the duration of bladder symptoms if these were the original and only presenting symptoms as assessed by history. Must have motor disability (minimum of stiffness or weakness).
“Progressing HAM/TSP” New or worsening (must be current within 3 months) motor symptoms in a patient with symptoms of greater than 2 years duration. Including a documented deterioration in timed walk by 50%. Patients with new symptoms under follow up could be included immediately. New patients presenting for initially with symptoms > 2 yrs would be eligible upon review if progression according to these criteria were met.
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E.4 | Principal exclusion criteria |
Hepatitis B or hepatitis C infection HIV infection Badly controlled hypertension (BP diastolic > 95 mmHg) Overt sepsis Prior exposure to corticosteroids (other than 3 days courses of pulsed methylprednisolone) or other immunomodulating therapy for HAM/TSP or within 2 years if not for HAM/TSP Active TB (untreated or on treatment) Strongyloides stercoralis (untreated)
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Incidence of clinical failure at 48 weeks. 2. Time to clinical failure (Kaplan-Meier)
Clinical Failure will be defined as any one of the following three: 1. Lack of any objective improvement (timed walk, spasticity, bladder function or pain) after three months of therapy 2. 1 point deterioration in the IPEC scale (an scale designed for the assessment of disability in patients with HAM) compared with baseline (2 separate measurements) at any time. 3. 30% deterioration in timed walk compared with baseline (2 separate measurements) at any time.
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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 | Yes |
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 | Information not present in EudraCT |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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 trial will be complete when the last patient to enter the trial has completed follow-up or withdrawn from the study |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |