Systemic SclerodermaPhase 1/2NCT02349009
Trial of Topical C-82 in Systemic Sclerosis - A Phase I/II Biomarker and Safety Trial
A Phase 1/2 study testing C-82 Topical Gel, 1% in people with Systemic Scleroderma, run by Prism Pharma Co., Ltd. at 3 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Systemic Scleroderma
- What is being tested?
- C-82 Topical Gel, 1%
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 17 participants
- Where
- 3 sitesUnited States
- Study dates
- Jun 2015to Sep 2016
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
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- 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.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Study site
Boston, Massachusetts
Study site
Chicago, Illinois
Study site
New York, New York
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Trial of Topical C-82 in Systemic Sclerosis - A Phase I/II Biomarker and Safety Trial
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.
Read the 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.
- Conditions
- Systemic Scleroderma
- Being tested
- C-82 Topical Gel, 1%, C-82 Topical Gel, Placebo
- Keywords
- systemic sclerosis, scleroderma
- Medical subject headings
- Scleroderma, Systemic, Scleroderma, Diffuse
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
C-82 Topical Gel, 1%Drug
active
C-82 Topical Gel, PlaceboDrug
placebo
| Study group | Role | What they receive |
|---|---|---|
| placebo | Placebo comparator | C-82 Topical Gel, Placebo Receives: Drug: C-82 Topical Gel, Placebo |
| Active | Active comparator | C-82 Topical Gel, 1% Receives: Drug: C-82 Topical Gel, 1% |
What is being measured
The primary outcome is the main question the study is designed to answer.
frequency and character of adverse events and abnormal clinical tests
Measured: 28 days
change in gene biomarker expression of THBS1 and COMP in skin biopsies over time compared to placebo
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2015 (estimated)
- Study startJun 2015
- Primary completionJul 2016
- Study completionSep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic SclerodermaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prism Pharma Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Robert Lafyatis, MD, Principal investigator, Boston University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02349009
- Sponsor's protocol ID
- PRI-C82T-3101
- Status last verified
- Sep 2016
- First submitted
- 18 Jan 2015
- Last updated
- 17 Aug 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Trial of Topical C-82 in Systemic Sclerosis - A Phase I/II Biomarker and Safety Trial
- ClinicalTrials.gov ID
- NCT02349009
- Acronym / sponsor ID
- PRI-C82T-3101
- Conditions
- Systemic Scleroderma
- Keywords
- systemic sclerosis, scleroderma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Prism Pharma Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Jun 2015
- Primary completion
- Jul 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 28 Jan 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| placebo | Placebo comparator | Drug: C-82 Topical Gel, PlaceboC-82 Topical Gel, Placebo |
| Active | Active comparator | Drug: C-82 Topical Gel, 1%C-82 Topical Gel, 1% |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (17 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| C-82 Topical Gel, 1%active | Drug | Active |
| C-82 Topical Gel, Placeboplacebo | Drug | placebo |
Primary outcome
frequency and character of adverse events and abnormal clinical tests
Time frame: 28 days
change in gene biomarker expression of THBS1 and COMP in skin biopsies over time compared to placebo
Time frame: 28 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert Lafyatis, MD principal investigator
Boston University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing C-82 Topical Gel, 1% in people with Systemic Scleroderma, run by Prism Pharma Co., Ltd. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
frequency and character of adverse events and abnormal clinical tests (time frame: 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Aug 2017.