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
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 发起方
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Localized Scleroderma Cutaneous Assessment Tool - LOSCAT
研究概览
简要总结
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).
详细描述
This is a phase IV, single-arm, open-label clinical trial to evaluate the efficacy and safety of PLACENTEX ® Polydeoxyribonucleotide 5.625 mg/3 ml for parenteral use 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). A total of n. 45 patients (including drop-outs, who will not be replaced) will be enrolled in this study.
The patients enrolled will be evaluated at study site at screening (V1), then after 3 months of treatment with PLACENTEX ® Polydeoxyribonucleotide 5.625 mg/3 ml for parenteral use (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, in which the patient will visit the site for the last visit (V3).
The IMP units will be dispensed to the patient at the study site at visit 1 (Screening Visit). Patients will be instructed to store IMP at room temperature and to return all used empty secondary packages and unused IMP units at visit 2 (End of Treatment Visit), at this time the Investigator/site coordinator will assess the patient's compliance with the prescribed regimen for the study medication. Compliance with the dosing regimen will be determined by performing IMP accountability of returned boxes of the IMP used and IMP unused units at visit 2 (End of Treatment Visit). Accountability records will be maintained during the study.
In order to prevent any interference in the evaluation of PLACENTEX ® Polydeoxyribonucleotide 5.625 mg/3 ml by prior therapies (corticosteroids, immune-suppressive agents) a wash-out period of 1 month has been planned before starting the investigational treatment. Such period will allow also to be definitely sure of the stability of inactive stage of the disease.
Treatment with steroid therapy and/or systemic immunosuppressive therapy within 1 month prior to screening visit are therefore not allowed. Additionally, the patient will be requested not to take local/systemic corticosteroids or local/systemic immunosuppressive therapy during the study period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Polydeoxyribonucleotides
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.
干预措施: Polydeoxyribonucleotides (Drug)
结局指标
主要结局
Localized Scleroderma Cutaneous Assessment Tool - LOSCAT
时间窗: 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.
次要结局
- tele-thermographic profile(6 months)
- ultrasound profile(6 months)
- Dermatology Life Quality Index (DLQI)(6 months)
- histology improvement(3 months)