Ignite Creation Date:
2024-05-06 @ 10:54 AM
Last Modification Date:
2024-10-26 @ 12:37 PM
Study NCT ID:
NCT03388255
Status:
TERMINATED
Last Update Posted:
2021-09-16
First Post:
2017-12-21
Brief Title:
Efficacy and Safety of PLACENTEX im in Patients With Scleroderma Diseases
Sponsor:
Mastelli Srl
Organization:
Mastelli Srl
Study Overview
Official Title:
A Phase IV Single-arm Open-label Clinical Trial to Evaluate the Efficacy and Safety of PLACENTEX Polydeoxyribonucleotide im in Patients With Fibrotic and Atrophic Cutaneous Lesions in Scleroderma Diseases
Status:
TERMINATED
Status Verified Date:
2021-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
the clinical center has too much difficulty recruiting as a rare condition
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a phase IV single-arm open-label clinical trial to evaluate the efficacy and safety of PLACENTEX Polydeoxyribonucleotide im 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-outs3 months of treatment with PLACENTEX Polydeoxyribonucleotide one vial per day for intra-muscular administration
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-outs3 months of treatment with PLACENTEX Polydeoxyribonucleotide one vial per day for intra-muscular administration
Detailed Description:
This is a phase IV single-arm open-label clinical trial to evaluate the efficacy and safety of PLACENTEX Polydeoxyribonucleotide 5625 mg3 ml for parenteral use im 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 5625 mg3 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 Investigatorsite coordinator will assess the patients 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 5625 mg3 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 andor systemic immunosuppressive therapy within 1 month prior to screening visit are therefore not allowed Additionally the patient will be requested not to take localsystemic corticosteroids or localsystemic immunosuppressive therapy during the study period
In case of non-response to the treatment with PLACENTEX or in case of worsening of the disease the patient will be withdrawn from the study and an alternative treatment will be considered by Investigator on a case-by-case basis
During the study period the following assessments will be performed
Photography of target cutaneous lesion to record size and location
Tele-thermography of target cutaneous lesion to record the characteristics of heat loss and store TT images of the lesion
Ultrasound evaluation of target cutaneous lesion to record the subcutaneous measuring parameters indicative of the disease thickness of subcutaneous adipose layer etc
Clinical evaluation of patients cutaneous lesions A score LOSCAT Score from 0 none to 3 high will be calculated with regard to the presence of atrophy and sclerosis
A biopsy sample will be collected from target cutaneous lesion for subsequent histological analysis A score from 0 none to 3 high will be calculated with regard to the presence of epidermal dermal hypodermic and skin appendages atrophy as well as dermal and hypodermic sclerosis
A self-administered Dermatology Life Quality Index DLQI from patient to collect information with particular regard to quality of life difficulty in walking loss of function
Evaluation of the target cutaneous lesion through a SkinFibrometer to measure the degree of induration of skin
Vital signs will be measured
Concomitant medications and medical history will be recorded
Local laboratory assessments will be performed in order to confirm the safety of the drug treatment as absence of inflammation or clinical worsening following treatment with drug according to Investigators judgment When abnormal laboratory values or test results constitute an adverse event ie induces clinical signssymptoms or requires therapy they must be recorded on the Adverse Events e-CRF page
Vital signs heart rate and blood pressure will be completed at screening visit Blood pressure and heart rate will be recorded in a sitting position after resting for 5 minutes instead Physical examination will be performed at all scheduled visits Significant findings present prior to the start of the study medication treatment must be included in the relevant medical history or current medical history condition in the e-CRF
Significant findings after the start of the study which meet the definition of an adverse event must be recorded in appropriate place in the e-CRF and evaluated by the Investigators
In the study any event occurring after that patient has signed the Informed Consent should be considered and recorded as an AE Adverse events especially those for which the relationship to the study drug is possible probable or remote should be followed up until they have stabilizedThe Sponsor via PhV responsabile is responsible for reporting all applicable SAEs and SUSARs to Regulatory authorities Investigators and IECs as applicable in accordance with national regulations in the countries where the study is conducted
All analyses will be performed using SAS v 92 Statistical Analysis System - New Cary USA or later in Microsoft Windows 7 Professional environment
For patients with missing values will be applied the Last Observation Carried Forward LOCF approach All variables will be summarized using appropriate descriptive statistics mean standard deviation median and range for continuous variables and frequencies and percentages for categorical variables followed by the calculation of 95 Confidence interval if pertinent and applicable A two-tailed alpha level of 005 will be used for each statistical test All treated patients will be included in the Intention-To-Treat population ITT
The patients enrolled will be evaluated at study site at screening V1 then after 3 months of treatment with PLACENTEX Polydeoxyribonucleotide 5625 mg3 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 Investigatorsite coordinator will assess the patients 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 5625 mg3 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 andor systemic immunosuppressive therapy within 1 month prior to screening visit are therefore not allowed Additionally the patient will be requested not to take localsystemic corticosteroids or localsystemic immunosuppressive therapy during the study period
In case of non-response to the treatment with PLACENTEX or in case of worsening of the disease the patient will be withdrawn from the study and an alternative treatment will be considered by Investigator on a case-by-case basis
During the study period the following assessments will be performed
Photography of target cutaneous lesion to record size and location
Tele-thermography of target cutaneous lesion to record the characteristics of heat loss and store TT images of the lesion
Ultrasound evaluation of target cutaneous lesion to record the subcutaneous measuring parameters indicative of the disease thickness of subcutaneous adipose layer etc
Clinical evaluation of patients cutaneous lesions A score LOSCAT Score from 0 none to 3 high will be calculated with regard to the presence of atrophy and sclerosis
A biopsy sample will be collected from target cutaneous lesion for subsequent histological analysis A score from 0 none to 3 high will be calculated with regard to the presence of epidermal dermal hypodermic and skin appendages atrophy as well as dermal and hypodermic sclerosis
A self-administered Dermatology Life Quality Index DLQI from patient to collect information with particular regard to quality of life difficulty in walking loss of function
Evaluation of the target cutaneous lesion through a SkinFibrometer to measure the degree of induration of skin
Vital signs will be measured
Concomitant medications and medical history will be recorded
Local laboratory assessments will be performed in order to confirm the safety of the drug treatment as absence of inflammation or clinical worsening following treatment with drug according to Investigators judgment When abnormal laboratory values or test results constitute an adverse event ie induces clinical signssymptoms or requires therapy they must be recorded on the Adverse Events e-CRF page
Vital signs heart rate and blood pressure will be completed at screening visit Blood pressure and heart rate will be recorded in a sitting position after resting for 5 minutes instead Physical examination will be performed at all scheduled visits Significant findings present prior to the start of the study medication treatment must be included in the relevant medical history or current medical history condition in the e-CRF
Significant findings after the start of the study which meet the definition of an adverse event must be recorded in appropriate place in the e-CRF and evaluated by the Investigators
In the study any event occurring after that patient has signed the Informed Consent should be considered and recorded as an AE Adverse events especially those for which the relationship to the study drug is possible probable or remote should be followed up until they have stabilizedThe Sponsor via PhV responsabile is responsible for reporting all applicable SAEs and SUSARs to Regulatory authorities Investigators and IECs as applicable in accordance with national regulations in the countries where the study is conducted
All analyses will be performed using SAS v 92 Statistical Analysis System - New Cary USA or later in Microsoft Windows 7 Professional environment
For patients with missing values will be applied the Last Observation Carried Forward LOCF approach All variables will be summarized using appropriate descriptive statistics mean standard deviation median and range for continuous variables and frequencies and percentages for categorical variables followed by the calculation of 95 Confidence interval if pertinent and applicable A two-tailed alpha level of 005 will be used for each statistical test All treated patients will be included in the Intention-To-Treat population ITT
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None