Trial of Topical C-82 in Systemic Sclerosis - A Phase I/II Biomarker and Safety Trial
Primary Purpose
Systemic Scleroderma
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
C-82 Topical Gel, 1%
C-82 Topical Gel, Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Systemic Scleroderma focused on measuring systemic sclerosis, scleroderma
Eligibility Criteria
Inclusion Criteria:
- meet the American College of Rheumatology criteria for systemic sclerosis with diffuse cutaneous involvement (clinical skin involvement proximal to forearms and or knees, not including the face).
- Disease duration of <36 months since the onset of the first SSc manifestation other than Raynaud's phenomenon, or patients with progressive disease based on new or worsening skin disease based on physician assessment.
- local skin score over the bilateral forearm of ≥ 2.
- a MRSS of ≥ 12.
- on stable dose of any immunosuppressive other than cyclophosphamide or high dose steroids (excluded treatments) for at least one month and through the course of study treatment.
- Subjects of child-producing potential must agree to use effective contraception while and for at least 3 months after the last treatment.
Exclusion Criteria:
- Receiving treatment as part of an interventional clinical trial within 4 weeks of screening or 5 half-lives of the investigational drug (whichever is longer).
- Ongoing use of high dose steroids (> 10mg/day prednisone or equivalent) or unstable steroid dose in the past 4 weeks.
- Use of topical creams or gels on the forearm area within the past month and through the course of study treatment.
- UV light therapy for 4 weeks before or during the study period.
- Treatment with cyclophosphamide within the past month and through the course of study treatment.
- Known active bacterial, viral fungal mycobacterial, or other infection
- history of malignancy within the past 2 years.
- Moderate to severe hepatic impairment, .
- Scleroderma renal crisis within 4 months or creatinine greater than 2.0.
- Pregnancy.
- Nursing mothers are to be excluded.
- Gastrointestinal involvement requiring total parenteral nutrition or hospitalization within the past 3 months for pseudo-obstruction
- Moderately severe pulmonary disease with FVC < 40%, or DLCO < 30% predicted or evidence of progressive lung disease as manifest by a decrease in FVC or 10% or more over the previous year.
- Moderately severe cardiac disease with clinically significant heart failure, or unstable angina.
- AST or ALT > 2.5 x Upper Limit of Normal.
- Total bilirubin > 1.5 x upper limit of normal (ULN). Patients with Gilbert's Disease may be included if their total bilirubin is ≤ 3.0 mg/dL.
- significant medical or psychosocial problems that warrant exclusion.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Placebo Comparator
Active Comparator
Arm Label
placebo
Active
Arm Description
C-82 Topical Gel, Placebo
C-82 Topical Gel, 1%
Outcomes
Primary Outcome Measures
frequency and character of adverse events and abnormal clinical tests
change in gene biomarker expression of THBS1 and COMP in skin biopsies over time compared to placebo
Secondary Outcome Measures
Full Information
NCT ID
NCT02349009
First Posted
January 18, 2015
Last Updated
August 16, 2017
Sponsor
Prism Pharma Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT02349009
Brief Title
Trial of Topical C-82 in Systemic Sclerosis - A Phase I/II Biomarker and Safety Trial
Official Title
A Randomized, Double-Blind, Placebo-Controlled Trial of Topical C-82 in Systemic Sclerosis - A Phase I/II Biomarker and Safety Trial
Study Type
Interventional
2. Study Status
Record Verification Date
September 2016
Overall Recruitment Status
Completed
Study Start Date
June 2015 (undefined)
Primary Completion Date
July 2016 (Actual)
Study Completion Date
September 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Prism Pharma Co., Ltd.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
1:1 active treatment: placebo, blinded trial, evaluating the effect of a 4-week treatment period with topical C-82 on skin expression of two gene biomarker surrogates (THBS1 and COMP) for the modified Rodnan skin score (MRSS). Study subjects will be randomized to apply the active study medication daily for 4 weeks to either the right or left forearm and placebo to the contralateral forearm.
Detailed Description
1:1 active treatment: placebo, blinded trial, evaluating the effect of a 4-week treatment period with topical C-82 on skin expression of two gene biomarker surrogates (THBS1 and COMP) for the modified Rodnan skin score (MRSS). Study subjects will be randomized to apply the study medication daily for 4 weeks to either the right or left forearm. All subjects will apply placebo (i.e., a topical gel without C-82) to the contralateral forearm. Both physician and subject will be blinded to treatment arm assignment. Skin biopsies will be taken from the mid-forearm of both arms at baseline and after four weeks of study drug to test for biomarker gene expression; instructions and supplies will be provided to each site. Safety assessments will extend to 4 weeks after the final dose of study drug/placebo.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Systemic Scleroderma
Keywords
systemic sclerosis, scleroderma
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
17 (Actual)
8. Arms, Groups, and Interventions
Arm Title
placebo
Arm Type
Placebo Comparator
Arm Description
C-82 Topical Gel, Placebo
Arm Title
Active
Arm Type
Active Comparator
Arm Description
C-82 Topical Gel, 1%
Intervention Type
Drug
Intervention Name(s)
C-82 Topical Gel, 1%
Intervention Description
active
Intervention Type
Drug
Intervention Name(s)
C-82 Topical Gel, Placebo
Intervention Description
placebo
Primary Outcome Measure Information:
Title
frequency and character of adverse events and abnormal clinical tests
Time Frame
28 days
Title
change in gene biomarker expression of THBS1 and COMP in skin biopsies over time compared to placebo
Time Frame
28 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
meet the American College of Rheumatology criteria for systemic sclerosis with diffuse cutaneous involvement (clinical skin involvement proximal to forearms and or knees, not including the face).
Disease duration of <36 months since the onset of the first SSc manifestation other than Raynaud's phenomenon, or patients with progressive disease based on new or worsening skin disease based on physician assessment.
local skin score over the bilateral forearm of ≥ 2.
a MRSS of ≥ 12.
on stable dose of any immunosuppressive other than cyclophosphamide or high dose steroids (excluded treatments) for at least one month and through the course of study treatment.
Subjects of child-producing potential must agree to use effective contraception while and for at least 3 months after the last treatment.
Exclusion Criteria:
Receiving treatment as part of an interventional clinical trial within 4 weeks of screening or 5 half-lives of the investigational drug (whichever is longer).
Ongoing use of high dose steroids (> 10mg/day prednisone or equivalent) or unstable steroid dose in the past 4 weeks.
Use of topical creams or gels on the forearm area within the past month and through the course of study treatment.
UV light therapy for 4 weeks before or during the study period.
Treatment with cyclophosphamide within the past month and through the course of study treatment.
Known active bacterial, viral fungal mycobacterial, or other infection
history of malignancy within the past 2 years.
Moderate to severe hepatic impairment, .
Scleroderma renal crisis within 4 months or creatinine greater than 2.0.
Pregnancy.
Nursing mothers are to be excluded.
Gastrointestinal involvement requiring total parenteral nutrition or hospitalization within the past 3 months for pseudo-obstruction
Moderately severe pulmonary disease with FVC < 40%, or DLCO < 30% predicted or evidence of progressive lung disease as manifest by a decrease in FVC or 10% or more over the previous year.
Moderately severe cardiac disease with clinically significant heart failure, or unstable angina.
AST or ALT > 2.5 x Upper Limit of Normal.
Total bilirubin > 1.5 x upper limit of normal (ULN). Patients with Gilbert's Disease may be included if their total bilirubin is ≤ 3.0 mg/dL.
significant medical or psychosocial problems that warrant exclusion.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robert Lafyatis, MD
Organizational Affiliation
Boston University
Official's Role
Principal Investigator
Facility Information:
City
Chicago
State/Province
Illinois
Country
United States
City
Boston
State/Province
Massachusetts
Country
United States
City
New York
State/Province
New York
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Trial of Topical C-82 in Systemic Sclerosis - A Phase I/II Biomarker and Safety Trial
We'll reach out to this number within 24 hrs