E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
SKIN FIBROSIS IN PATIENTS WITH SYSTEMIC SCLEROSIS |
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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 | 10039710 |
E.1.2 | Term | Scleroderma |
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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 asses the efficacy of P144 topically administered in skin lesions of systemic sclerosis patients throw the measure of soluble collagen content and durometry over three months. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives are to evaluate the safety, quality of life and pharmacokinetics of P144 topically administered one a day for up to three months. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
· Patients male and female ≥18 years at time of consent. · History of systemic sclerosis (including diffuse scleroderma and limited scleroderma) for less than three years of evolution from the onset of cutaneous manifestations. Patients will also be eligible for treatment if, despite a history of systemic sclerosis longer than three years from the onset of cutaneous manifestations, they have active skin disease, defined as a worsening of the Modified Rodnan Skin Score (MRSS) during the last year of follow-up or by the clinical judgement of its attending physician. · Symmetric lesions in forearms. The extension of the selected symmetric lesions must be at least 15 square cm. · Stable therapy for at least one month, except in the case of patients under treatment with putative disease modifying agents (immunosupressants like cyclophosphamide, methotrexate or azathioprine) that will need at least three months of stable therapy, without the expectation of treatment modifications during the trial period. · Capable of understanding and willing to provide signed and dated written voluntary informed consent before any protocol specific procedures are performed. · For female subjects with childbearing potential: use of a known highly effective method of birth control, defined as those which results in a low failure rate: i.e. less 1% per year, (contraceptive pills, intrauterine contraceptive device, implants, vasectomized partner or sexual abstinence), for at least three consecutive months prior to the study, during the study and one month after the end of the study.
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E.4 | Principal exclusion criteria |
o Systemic sclerosis sine scleroderma. o Localized scleroderma. o Eosinophilic fascitis, eosinophilia myalgia syndrome. · Any other definable connective tissue disease, such as rheumatoid arthritis, systemic lupus erythematosus, polymyositis, or dermatomyositis. · Clinically significant overlap condition. · Significant existing internal organ damage (Kidney, Cardiovascular disease, Pulmonary disease, Gastrointestinal disease) as defined in “Guidelines for clinical trial in systemic sclerosis (scleroderma) “ · History of skin cancer. · Other skin diseases affecting the treatment area. · Patients with substantial history of environmental exposure to tainted rapeseed oil, vinyl chloride, L- tryptophan, bleomycin, trichoroethylene, or silica. · PUVA therapy within 1 month of study drug initiation. · Concurrent interventional therapy that might independently influence outcome of trial, such as D-penicillamine, cyclosporine, interferon-g or photopheresis. · Topical corticosteroids treatment affecting the selected area. · Cosmetics over the treatment area. · Pregnant or breast-feeding women. · Reasonable expectation that the subject will not be able to satisfactorily complete the study: o History of or current psychiatric illness that would interfere with the subject’s ability to comply with protocol requirements or give informed consent. o History of alcohol or drug abuse that would interfere with the subject’s ability to comply with protocol requirements. o Receipt of any investigational drug within three months of screening visit. o Documented noncompliance.
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E.5 End points |
E.5.1 | Primary end point(s) |
The percentage of reduction in the soluble collagen content and skin hardness after three months of treatment relative to baseline. |
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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 | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
INTRAINDIVIDUALLY PLACEBO CONTROLLED |
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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
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 14 |
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 | 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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 12 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 12 |
E.8.9.2 | In all countries concerned by the trial days | 0 |