E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
SISTEMIC SCLEROSIS |
SCLEROSI SISTEMICA |
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E.1.1.1 | Medical condition in easily understood language |
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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 | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10042953 |
E.1.2 | Term | Systemic sclerosis |
E.1.2 | System Organ Class | 100000004859 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To confirm the efficacy, the safety and the feasibility of the slow infusion of Iloprost done at home, using the portable syringe pump INFONDE® in a group of patients affected by systemic sclerosis.
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Confermare l’efficacia, la sicurezza e la fattibilità dell’infusione lenta di Iloprost a domicilio mediante pompa siringa portatile INFONDE® in un gruppo di pazienti affetti da sclerosi sistemica |
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E.2.2 | Secondary objectives of the trial |
• Patients’ Quality of Life, measured with the HAQ ( Health Assessment Questionnaire • Pharmacoeconomic assessments, related to the probable resources savings (direct and indirect costs), linked to the treatment made at home. |
Qualità della vita dei pazienti, misurata mediante il questionario HAQ (Health Assessment Questionnaire) Valutazioni di farmacoeconomia, relative al probabile risparmio di risorse (costi diretti ed indiretti) , ottenibile con il trattamento domiciliare |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Informed Consent personally signed and dated by the patient, before any study procedure is implemented 2. Diagnosis of Raynaud’ Disease, with or without presence of acral ulcers 3. Age ≥ 18 and ≤ 80 years 4. Check for proper venous assets (rated by experienced personnel) or central venous access 5. Women of childbearing potential not willing to become pregnant during the course of the study and for 1 month following its completion 6. Men having a relationship with women of childbearing age, who are willing not to get pregnant during the course of the study and for 1 month following its completion |
Consenso Informato firmato e datato dal paziente, prima di iniziare qualsiasi procedura prevista dallo studio 2. Diagnosi di sclerodermia con Fenomeno di Raynaud, con presenza o meno di ulcere acrali 3. Età ≥ 18 anni e ≤ 80 anni 4. Verifica di patrimonio venoso adeguato (valutato da personale esperto) o dell’accesso venoso centrale 5. Donne in età fertile disposte a non iniziare una gravidanza durante il corso dello studio e per 1 mese successivo al suo completamento 6. Uomini che hanno una relazione con donne in età fertile che sono disposti a non procurare una gravidanza durante il corso dello studio e per 1 mese successivo al suo completamento |
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E.4 | Principal exclusion criteria |
1. Patients with a disease characterized by advanced stages of deformation 2. Contraindications to treatment with Iloprost and / or hypersensitivity to the active substance or to any of the excipients (trometamol, ethanol, 96% (v / v), sodium chloride, hydrochloric acid) 3. Chronic renal failure (GFR <30 ml / min 4. Liver Cirrhosis 5. Surgery planned or executed by less than 3 months after initiation of treatment to the central nervous system and / or the eye; trauma surgery associated with large wounds exposed 6. Severe Thrombocytopenia (PLT <20,000 / mmc) or severe anemia (Hb <8 g / dl) 7. Severe and not controlled arterial hypertension 8. Orthostatic hypotension 9. Cephalalgia type of pathology and / or chronic vertigo evaluated by a reference specialist and / or for which the patient is undergoing a drug treatment 10. Gastrointestinal pathology in acute phase 11. Pregnancy and / or breast-feeding 12. Severe coronary heart disease or unstable angina, myocardial infarction within six months preceding the start of treatment, acute or chronic congestive heart failure (NYHA II - IV), severe arrhythmias or relevant for prognosis 13. Severe ischemic disorders of the lower limbs (grade III and IV) 14. Smoking Patients with high levels of daily consumption and not willing to abstain during the treatment period 15. Severe coagulation disorders, who need a stable regimen of treatment with anticoagulants or platelet aggregation inhibitors 16. Patients with a neurological condition that makes them incapable of understanding the nature, purpose and possible consequences of the study 17. Congestive heart failure |
Pazienti con malattia caratterizzata da stati di deformazione avanzati 2. Controindicazioni al trattamento con Iloprost e/o ipersensibilità al principio attivo o ad uno qualsiasi degli eccipienti (trometamolo; etanolo, 96% (v/v); cloruro di sodio; acido cloridrico) 3. Insufficienza renale cronica (VFG< 30 ml/min) 4. Cirrosi epatica 5. Intervento chirurgico previsto o eseguito da meno di 3 mesi dall’inizio del trattamento al sistema nervoso centrale e/o all’occhio; chirurgia traumatica associata a grandi ferite esposte 6. Piastrinopenia severa (PLT <20000/mmc) o anemia severa (Hb <8 g/dl) 7. Ipertensione arteriosa grave non controllata 8. Ipotensione ortostatica 9. Patologia di tipo cefalgico e/o vertigini croniche valutate da specialista di riferimento e/o per le quali il paziente risulta in trattamento farmacologico 10. Patologia gastrointestinale in fase acuta 11. Gravidanza e/o allattamento 12. Coronaropatie gravi o angina instabile, infarto del miocardico nei sei mesi precedenti l’inizio del trattamento, insufficienza cardiaca congestizia acuta o cronica (NYHA II - IV), aritmie gravi o rilevanti per la prognosi 13. Patologie ischemiche severe degli arti inferiori (grado III e IV) 14. Pazienti fumatori, con elevati livelli di consumo quotidiano e non disposti all’astensione durante il periodo del trattamento 15. Patologie coagulative severe, che necessitino un regime stabile di trattamento con anticoagulanti o inibitori dell’aggregazione piastrinica 16. Pazienti affetti da una condizione neurologica che li renda incapaci di comprendere la natura, lo scopo e le possibili conseguenze dello studio 17. Scompenso cardiaco congestizio |
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E.5 End points |
E.5.1 | Primary end point(s) |
Raynaud’s Disease intensity, evaluated through the Raynaud’s Condition Score (RCS) |
Intensità del Fenomeno di Raynaud (FR) valutato mediante la Raynaud’s Condition Score (RCS) |
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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) |
Ischemic ulcers incidence observed at 6 months of follow-up Recovery time of the observed ischemic ulcers Frequency and duration of the Raynaud’s Disease, evaluated through the Raynaud’s Condition Score (RCS) Patients’ Quality of Life, measured with the HAQ ( Health Assessment Questionnaire Completion rate of the entire home infusion cycle Adverse Events incidence |
Incidenza di ulcere ischemiche a 6 mesi di follow-up Tempo di guarigione delle ulcere ischemiche in studio Frequenza e durata del Fenomeno di Raynaud (FR), valutato mediante la Raynaud’s Condition Score (RCS) Qualità della vita misurata mediante il questionario HAQ (Health Assessment Questionnaire) Tasso di completamento dell’intero ciclo di infusione a domicilio Incidenza di eventi avversi |
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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 | Yes |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
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 | 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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
STESSO FARMACO MA SOMMINISTRATO ATTRAVERSO POMPA DI INFUSIONE |
SAME DRUG ADMINISTRATED BY MEDICAL DEVICE |
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E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 16 |
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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CLINICAL STUDY REPORT |
DISPONIBILITA' DEL CLINICAL STUDY REPORT |
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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 | 21 |
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 | 21 |
E.8.9.2 | In all countries concerned by the trial days | 0 |