E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Fibrotic and atrophic cutaneous lesions in localized scleroderma diseases. |
Lesioni fibrotiche e atrofiche cutanee nelle malattie sclerodermiformi localizzate. |
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E.1.1.1 | Medical condition in easily understood language |
Fibrotic and atrophic cutaneous lesions in localized scleroderma diseases. |
Lesioni fibrotiche e atrofiche cutanee nelle malattie sclerodermiformi localizzate. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10039711 |
E.1.2 | Term | Scleroderma and associated disorders |
E.1.2 | System Organ Class | 100000004870 |
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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 evaluate the efficacy on clinical improvement of cutaneous lesions following treatment with PLACENTEX ¿ Polydeoxyribonucleotide 5.625 mg/3 ml for parenteral use. |
Valutazione dell'efficacia del trattamento con PLACENTEX ¿ Polidesossiribonucleotide sul miglioramento clinico di lesioni cutanee. |
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E.2.2 | Secondary objectives of the trial |
Efficacy: 1. To evaluate the improvement of tele-thermographic profile of target cutaneous lesion following treatment with drug.
2. To evaluate the improvement of ecographic profile (through ultrasound test) of target cutaneous lesion following treatment with drug.
3. To evaluate the histology improvement of target cutaneous lesion following treatment with drug.
4. To evaluate the improvement of the dermatology life quality index (DLQI) of patient (through self-administered Dermatology Life Quality Index (DLQI)).
Safety: 5. To evaluate the potential clinical worsening following treatment with drug. |
Efficacia: 1. Valutazione del miglioramento del profilo tele-termografico della lesione cutanea target dopo il trattamento con il farmaco.
2. Valutazione del miglioramento del profilo ecografico della lesione cutanea target in seguito al trattamento con il farmaco.
3. Valutazione del miglioramento istologico della lesione cutanea target dopo il trattamento con il farmaco. 4. Valutazione del miglioramento dell'indice di qualit¿ della vita dermatologia (DLQI) del paziente dopo il trattamento con il farmaco.
Sicurezza: 5. Valutazione dell¿eventuale peggioramento clinico del paziente dopo il trattamento con il farmaco. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female age > 18 years. 2. Patients diagnosed with localized scleroderma diseases during inactive stage with fibrotic and atrophic cutaneous lesions confirmed histologically. 3. Understanding the nature of the study and Signature of the written informed consent. 4. Negative pregnancy test at study entry for females of child bearing potential. 5. If the patient is a female of childbearing potential (less than 24 months since the last menstrual bleeding), she is using an acceptable and effective method of contraception* during the study period.
* Methods of birth control which result in a low failure rate (i.e. less than 1% per year) when used consistently and correctly such as: implants, injectables, some intrauterine devices (IUDs), condom (for the partner), sexual abstinence or vasectomized partner.
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1. Maschio o femmina > 18 anni. 2. Pazienti con diagnosi di malattie sclerodermiche localizzate in fase di inattivitá, con lesioni cutanee fibrotiche e atrofiche confermate istologicamente. 3. Comprensione della natura dello studio e firma del consenso informato scritto. 4. Test di gravidanza negativo all'inizio dello studio, per femmine in età fertile. 5. In caso di pazienti donne in etá fertile (meno di 24 mesi dall'ultimo sanguinamento mestruale), utilizzo di un metodo contraccettivo accettabile ed efficace* durante il periodo di studio.
* Metodi contraccettivi con un dimostrato basso livello di errore (meno di 1% in un anno) quando utilizzati correttamente secondo l'indicazione, tra cui: impianti, iniettabili, alcuni dispositivi intra-uterini (IUD), preservativo (per il partner), astinenza sessuale o vasectomia (per il partner). |
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E.4 | Principal exclusion criteria |
1. Patients under treatment with steroid therapy and/or systemic immunosuppressive therapy within 1 month prior to screening. 2. Patients with ongoing infectious processes at the level of target lesions. 3. Women who are pregnant or breast feeding. 4. Known allergy or hypersensitivity to the active principle of the investigational drug or to one of its excipients. 5. Patients with a condition or concurrent severe and/or uncontrolled medical disease which could compromise his/her participation, compliance with and/or completion of study procedures. |
1. Pazienti sotto trattamento con terapia steroidea e/o terapia immunosoppressiva sistemica, entro 1 mese prima dello screening. 2. Pazienti con processi infettivi in atto a livello delle lesioni target. 3. Donne durante gravidanza o in allattamento. 4. Nota allergia o ipersensibilità al principio attivo del farmaco sperimentale o ad uno dei suoi eccipienti. 5. Pazienti con una condizione o malattia grave concomitante e/o non controllata che potrebbe compromettere la loro partecipazione, l’aderenza e/o il completamento delle procedure di studio. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The percentage of patients with a clinical improvement has been chosen as an efficacy endpoint of primary interest. The clinical improvement of cutaneous lesions following treatment with drug will be determined through a validated score (Localized Scleroderma Cutaneous Assessment Tool – LOSCAT) ranging with regard to the presence of atrophy and sclerosis, according to Investigator’s judgement. |
Percentuale di pazienti con miglioramento clinico a livello delle lesioni cutanee riscontrate alla visita di selezione, dopo trattamento con il farmaco in studio. Tale percentuale verrá determinata attraverso lo score validato LOSCAT (Localized Scleroderma Cutaneous Assessment Tool), che misura il grado di atrofia e sclerosi, in base al giudizio dello Sperimentatore. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1) Screening Visit (day 0) as baseline / End of Treatment Visit (day 90) / Follow-up Visit (day 180). |
1) Visita di Screening (giorno 0) al basale / Visita di Fine Trattamento (giorno 90) / Visita di Controllo (giorno 180). |
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E.5.2 | Secondary end point(s) |
Efficacy: 1. Changes in the tele-thermographic profile of target cutaneous lesion following treatment with drug, according to Investigator¿s judgement. ; Efficacy: 2. Changes in the ultrasound profile of target cutaneous lesion following treatment with drug, according to Investigator¿s judgement. ; Efficacy: 3. Measurement of histology improvement of target cutaneous lesion following treatment with drug through a validated score ranging from 0 (none) to 3 (high) with regard to the presence of epidermal, dermal, hypodermic and skin appendages atrophy as well as dermal and hypodermic sclerosis, according to Investigator¿s judgement. ; Efficacy: 4. Evaluation of patient¿s satisfaction in terms of functional incapacity and improvement of ambulation through a validated self-administered Dermatology Life Quality Index (DLQI). ; Safety: 5. Clinical worsening following treatment with drug, according to Investigator¿s judgement. ; Exploratory Endpoint: 6. Changes in the degree of induration of skin (in particular, subcutaneous tissue) at the level of target cutaneous lesion through a SkinFibrometer, following treatment with drug, according to Investigator¿s judgement. |
Efficacia: 1. Variazioni nel profilo tele-termografico della lesione cutanea target dopo il trattamento con il farmaco, in base al giudizio dello Sperimentatore. ; Efficacia: 2. Variazioni nel profilo ecografico della lesione cutanea target dopo il trattamento con il farmaco, in base al giudizio dello Sperimentatore. ; Efficacia: 3. Misurazione del miglioramento istologico della lesione cutanea target dopo il trattamento con il farmaco, attraverso una scala validate, che va da 0 (nessuno) a 3 (alto), per quanto riguarda la presenza di atrofia di annessi cutanei epidermici, dermici, e ipodermici, nonch¿ di sclerosi dermica ed ipodermica. ; Efficacia: 4. Valutazione della soddisfazione del paziente, attraverso il questionario validato auto-somministrato Dermatology Life Quality Index (DLQI). ; Sicurezza: 5. Variazioni nei parametri di peggioramento clinico dopo il trattamento con il farmaco, secondo il giudizio dello Sperimentatore. ; Endpoint Esploratorio: 6. Variazioni nel grado di indurimento della pelle (in particolare a del tessuto sub-cutaneo) a livello della lesione cutanea target, attraverso l¿utilizzo di uno SkinFibrometer, in seguito al trattamento con il farmaco, secondo il giudizio dello Sperimentatore. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Screening Visit (day 0) as baseline / End of Treatment Visit (day 90) / Follow-up Visit (day 180). ; Screening Visit (day 0) as baseline / End of Treatment Visit (day 90) / Follow-up Visit (day 180). ; Screening Visit (day 0) as baseline / End of Treatment Visit (day 90). ; Screening Visit (day 0) as baseline / End of Treatment Visit (day 90) / Follow-up Visit (day 180). ; End of Treatment Visit (day 90) / Follow-up Visit (day 180). ; Screening Visit (day 0) as baseline / End of Treatment Visit (day 90) / Follow-up Visit (day 180).
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Visita di Screening (giorno 0) al basale / Visita di Fine Trattamento (giorno 90) / Visita di Controllo (giorno 180). ; Visita di Screening (giorno 0) al basale / Visita di Fine Trattamento (giorno 90) / Visita di Controllo (giorno 180). ; Visita di Screening (giorno 0) al basale / Visita di Fine Trattamento (giorno 90). ; Visita di Screening (giorno 0) al basale / Visita di Fine Trattamento (giorno 90) / Visita di Controllo (giorno 180). ; Visita di Fine Trattamento (giorno 90) / Visita di Controllo (giorno 180). ; Visita di Screening (giorno 0) al basale / Visita di Fine Trattamento (giorno 90) / Visita di Controllo (giorno 180). |
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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 | No |
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) | 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 | 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 | 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.8.2.4 | 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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | No |
E.8.5.1 | Number of sites anticipated in the EEA | 1 |
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 | 11 |
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 | 11 |
E.8.9.2 | In all countries concerned by the trial days | 0 |