Fibrotic and atrophic cutaneous lesions in localized scleroderma diseases. MedDRA version: 20.0 Level: HLT Classification code 10039711 Term: Scleroderma and associated disorders System Organ Class: 100000004870
Conditions
Interventions
Trade Name: PLACENTEX ¿5.625 mg/3 ml soluzione iniettabile¿
Product Name: PLACENTEX ¿ Polydeoxyribonucleotide 5.625 mg/3 ml
Pharmaceutical Form: Solution for injection
INN or Proposed INN: POLYDEOXYR
Sponsors
MASTELLI SRL
Eligibility
Sex/Gender
All
Inclusion criteria
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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 21 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 24
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy on clinical improvement of cutaneous lesions following treatment with PLACENTEX ¿ Polydeoxyribonucleotide 5.625 mg/3 ml for parenteral use.;Secondary Objective: 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.;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.;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). | — |
Secondary
| Measure | Time frame |
|---|---|
| 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.;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). | — |
Countries
Italy
Contacts
Public ContactDirezione Medica
Mastelli Srl
Outcome results
None listed