E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Systemic sclerosis |
Systemsklerose |
|
E.1.1.1 | Medical condition in easily understood language |
Scleroderma |
Sklerodiermie |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10059040 |
E.1.2 | Term | Autologous peripheral haematopoietic stem cell transplant |
E.1.2 | System Organ Class | 10042613 - Surgical and medical procedures |
|
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 optimise the treatment for severe systemic sclerosis with high dose chemotherapy and autologous stem cell transplantation by adapting the procedure to the individual organ manifestation. |
|
E.2.2 | Secondary objectives of the trial |
Reduction of treatment related mortality |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Limited or diffuse cutaneous systemic sclerosis with organ manifestation (lung/heart or kidney) Progressive disease despite cyclophosphamide treatment Cyclophosphamide i.v.: at least 3 x 500-1000 mg/m² every 3-4 weeks or Oral Cyclophosphamide with at least 100mg/day for at least 2 months or or contraindications for a cyclophosphamide treatment Written informed consent
|
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E.4 | Principal exclusion criteria |
Age < 18 years Pregnancy or unwillingsness to use contraception Concomittend severe disease Heart insuffiency with EF < 30% (echo) Pulmonary arterial hypertension with papSys > 50mmHg Kidney insiffiency with Clearance for creatinine < 30ml/min Severe limitation in lung function test IVC < 50% DLCO ≤ 30% Bone marrow defect Leukopenia < 2.000/ul Thrombopenia < 100.000/ul Cyclophosphamide sum dose > 50g (relative) Chronic infection (Hepatitis B/C, HIV, salmonella, lues, tuberculosis (relative) Severe psychiatric disease Abuse of nicotine, alcohol or any illegal drugs Poor compliance
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E.5 End points |
E.5.1 | Primary end point(s) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Safety (treatment related mortality/toxicity) Progression free survival Frequency of cardiac manifestations Efficacy (mRSS; sHAQ, DLCO/IVC)
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | 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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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 | No |
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.8.5 | 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 | No |
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
|
Last patient in (evaluation after 36 months follow up) |
|
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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |