Efficacy and Safety of PLACENTEX ® i.m. in Patients With Scleroderma Diseases
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|ClinicalTrials.gov Identifier: NCT03388255|
Recruitment Status : Recruiting
First Posted : January 2, 2018
Last Update Posted : January 3, 2018
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).
|Condition or disease||Intervention/treatment||Phase|
|Scleroderma Disease||Drug: Polydeoxyribonucleotides||Phase 4|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||45 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||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|
|Actual Study Start Date :||November 8, 2016|
|Estimated Primary Completion Date :||March 8, 2018|
|Estimated Study Completion Date :||September 8, 2018|
PLACENTEX: Polydeoxyribonucleotides 5.625 mg/3 ml for parenteral use i.m.
The study period consists of the following phases:
Polydeoxyribonucleotide 5.625 mg/3 ml for parenteral use i.m.
The study period consists of the following phases:
Other Name: PLACENTEX
- Localized Scleroderma Cutaneous Assessment Tool - LOSCAT [ Time Frame: 6 months ]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.
- tele-thermographic profile [ Time Frame: 6 months ]Changes in the tele-thermographic profile of target cutaneous lesion following treatment with drug, according to Investigator's judgment
- ultrasound profile [ Time Frame: 6 months ]Changes in the ultrasound profile of target cutaneous lesion following treatment with drug, according to Investigator's judgment.
- histology improvement [ Time Frame: 3 months ]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 judgment.
- Dermatology Life Quality Index (DLQI) [ Time Frame: 6 months ]
It is a simple 10-question validated questionnaire. The aim of the questionnaire is to measure how much patient's skin problem has affected patient's life. The scoring of each question is as follows:
Very much scored 3 A lot scored 2 A little scored 1 Not at all scored 0 Not relevant scored 0 Question 7, 'prevented work or studying' scored 3 The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimumof 0. The higher the score, the more quality of life is impaired.
HOW TO INTERPRET MEANING OF DLQI SCORES 0 - 1 no effect at all on patient's life 2 - 5 small effect on patient's life 6 - 10 moderate effect on patient's life 11 - 20 very large effect on patient's life 21 - 30 extremely large effect on patient's life
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03388255
|Contact: Giulia Cattarini, Dremail@example.com|
|IRCCS Ca' Granda Ospedale Maggiore Policlinico||Recruiting|
|Milan, Italy, 21122|
|Contact: Simona Muratori, Dr +390255035186|
|Principal Investigator: Simona Muratori, Dr|
|Principal Investigator:||Simona Muratori, Dr||IRCCS Ca' Granda Osp. Maggiore Policlinico|