E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Raynaud's phenomenon and acral ulcers in systemic sclerosis |
Fenomeno di raynaud e ulcere acrali secondarie a sclerosi sistemica |
|
E.1.1.1 | Medical condition in easily understood language |
Manifestations caused by a low blood flow in hands and feet in patients affected by fibrosis of various tissues. |
Manifestazioni dovute a basso flusso sanguigno in pazienti affetti da fibrosi dei tessuti |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
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 |
feasibility of the home infusion therapy with endoprost |
fattibilità dell'infusione domiciliare di Iloprost |
|
E.2.2 | Secondary objectives of the trial |
Safety and efficacy of the new method |
Sicurezza ed efficacia della nuova metodica |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Acral ulcers and / or Raynaud's phenomenon in patients affected by systemic sclerosis
- A usable peripheral venous access or an avalaible central venous access.
- Age above 18 years and below 80 years.
- Informed consent signed by the patient
- Women of childbearing potential who are willing to practice effective contraception for the infusion period
|
Ulcere acrali e/o fenomeno di Raynaud secondario a Sclerodermia.
-Patrimonio venoso adeguato (valutato da personale infermieristico esperto) o accesso venoso centrale.
-Età superiore a 18 anni ed età inferiore ai 80 anni.
-Consenso informato sottoscritto dal paziente
-Donne in età fertile che sono disposte a praticare efficace contraccezione per il periodo della sperimentazione
|
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E.4 | Principal exclusion criteria |
Hypersensitivity to the active substance or to any of the excipients.
- Pregnancy / Lactation.
- Severe coronary heart disease or unstable angina.
- Myocardial infarction within the previous six months.
- Acute or chronic congestive heart failure (NYHA II - IV).
- Serious arrhythmias.- Suspicion of pulmonary congestion.
- Cerebrovascular events in the last 3 months.
- Chronic renal failure (GFR <30 ml / min).
- Cirrhosis .
- Therapy with vasodilators
- Active bleeding
- Severe thrombocytopenia (PLT <20000/mmc). Severe anemia (Hb <8 g / dl).
- Severe arterial hypertension not controlled.
- Orthostatic hypotension.
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-Ipersensibilità al principio attivo o ad uno qualsiasi degli eccipienti.
-Gravidanza/Allattamento.
-Coronaropatie gravi o angina instabile.
-Infarto miocardico nei sei mesi precedenti.
-Insufficienza cardiaca congestizia acuta o cronica (NYHA II - IV).
-Aritmie gravi o rilevanti per la prognosi.
-Sospetto di congestione polmonare.
-Eventi cerebro-vascolari negli ultimi 3 mesi.
-Insufficienza renale cronica (VFG< 30 ml/min).
-Cirrosi epatica.
-Terapia con farmaci vasodilatatori
-Sanguinamento in atto
-Piastrinopenia severa (PLT <20000/mmc). Anemia severa (Hb <8 g/dl).
-Ipertensione arteriosa grave non controllata.
-Ipotensione ortostatica.
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|
E.5 End points |
E.5.1 | Primary end point(s) |
75% of patients completed the six-month treatment |
75% dei pazienti a termine del trattamento |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Response of Raynaud's phenomenon and acral ulcers, patient satisfaction for the new method
|
Risposta di fenomeno di raynaud e ulcere acrali, soddisfazione del paziente per la nuova metodica |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1°,2°,3°,4°,5°,6° months |
1°,2°,3°,4°,5°,6° mese |
|
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 | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
home therapy |
Terapia domiciliare |
|
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 | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
studio pilota |
pilot study |
|
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.8.2.4 | Number of treatment arms in the trial | 1 |
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
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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 | |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |