Efficacy and Safety of PLACENTEX ® i.m. in Patients With Scleroderma Diseases

NCT03388255 | Terminated Phase 4

• 1 other identifier: PDRN-01-16
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

This is a phase IV, single-arm, open-label clinical trial to evaluate the efficacy and safety of PLACENTEX ® Polydeoxyribonucleotide i.m. in patients with fibrotic and atrophic cutaneous lesions in scleroderma diseases during the inactive stage of the disease (experiencing dystrophic outcomes of the disease with no inflammatory component at the time of enrolment). The patients enrolled will be evaluated at study site at screening (V1), then after 3 months of treatment with PLACENTEX ® Polydeoxyribonucleotide (one vial per day for intra-muscular administration) (V2). After completion of the study treatment period, the patients will be followed for an additional period of 3 months without study medication, after which the patient will visit the site for the last visit (V3). 1 investigational site. 45 patients enrolled (included drop-outs).3 months of treatment with PLACENTEX ® Polydeoxyribonucleotide (one vial per day for intra-muscular administration).

Conditions

Scleroderma Disease

Keywords

Polydeoxyribonucletide; Placentex; PDRN

Outcome Measures

Primary Outcomes (1)

• Localized Scleroderma Cutaneous Assessment Tool - LOSCAT

  The LoSCAT assesses 18 cutaneous anatomic sites, capturing both disease activity (mLoSSI) and damage (LoSDI) parameters. This score will take into consideration all the cutaneous lesions resulting from localized scleroderma diseases during the inactive stage of the disease.Scores for each site are based on the most severe score for each parameter. In order to minimize inter-subject variability, all skin changes are compared with the contralateral or ipsilateral skin area.

  6 months

Secondary Outcomes (4)

• tele-thermographic profile

  6 months

• ultrasound profile

  6 months

• histology improvement

  3 months

• Dermatology Life Quality Index (DLQI)

  6 months

Study Arms (1)

Polydeoxyribonucleotides

EXPERIMENTAL

PLACENTEX: Polydeoxyribonucleotides 5.625 mg/3 ml for parenteral use i.m. The study period consists of the following phases: * Treatment period: 3 months (daily i.m. treatment with PLACENTEX ® Polydeoxyribonucleotide 5.625 mg/3 ml for parenteral use, one vial per day for intra-muscular administration). * Follow up period: 3 months after end of active treatment, without study medication.

Drug: Polydeoxyribonucleotides

Interventions

Polydeoxyribonucleotides DRUG

Polydeoxyribonucleotide 5.625 mg/3 ml for parenteral use i.m. The study period consists of the following phases: * Treatment period: 3 months (daily i.m. treatment with PLACENTEX ® Polydeoxyribonucleotide 5.625 mg/3 ml for parenteral use, one vial per day for intra-muscular administration). * Follow up period: 3 months after end of active treatment, without study medication.

Also known as: PLACENTEX

Polydeoxyribonucleotides

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female age \> 18 years.
• Patients diagnosed with localized scleroderma diseases during inactive stage with fibrotic and atrophic cutaneous lesions confirmed histologically.
• Understanding the nature of the study and Signature of the written informed consent.
• Negative pregnancy test at study entry for females of child bearing potential.
• 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.

You may not qualify if:

• Patients under treatment with steroid therapy and/or systemic immunosuppressive therapy within 1 month prior to screening.
• Patients with ongoing infectious processes at the level of target lesions.
• Women who are pregnant or breast feeding.
• Know allergy or hypersensitivity to the active principle of the investigational drug or to one of its excipients.
• 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.

Sponsors & Collaborators

• Mastelli S.r.l: lead
• Sintesi Research Srl: collaborator

Study Sites (1)

IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 21122, Italy

Location

MeSH Terms

Conditions

Scleroderma, Diffuse

Interventions

Polydeoxyribonucleotides

Condition Hierarchy (Ancestors)

Scleroderma, Systemic; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Polynucleotides; Nucleotides; Nucleic Acids, Nucleotides, and Nucleosides

Study Officials

• Simona Muratori, Dr

  IRCCS Ca' Granda Osp. Maggiore Policlinico

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2017
• First Posted: January 2, 2018
• Study Start: November 8, 2016
• Primary Completion: October 30, 2019
• Study Completion: October 31, 2019
• Last Updated: September 16, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)