Diffuse Cutaneous Systemic SclerosisPhase 2dcSSCNCT04680975
Efficacy and Safety of Belumosudil in Subjects With Diffuse Cutaneous Systemic Sclerosis
A Phase 2 study testing Belumosudil in people with Diffuse Cutaneous Systemic Sclerosis, run by Kadmon, a Sanofi Company at 6 sites in United States.
Trial summary
This was a phase 2, open-label, single-cohort, multicenter trial of belumosudil in participants with Diffuse Cutaneous Systemic Sclerosis (dcSSc). An estimated total of 12 to 15 participants would receive belumosudil 200 milligrams (mg) administered orally (PO) twice daily (BID) for 52 weeks. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Diffuse Cutaneous Systemic Sclerosis
- What is being tested?
- Belumosudil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 6 sitesUnited States
- Study dates
- 3 Mar 2021to 19 Dec 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Male and female participants greater than or equal to (>=) 18 years old with the diagnosis of dcSSc according to the 2013 American College of Rheumatology and European League Against Rheumatism.
- Had disease duration (defined as interval from first non Raynaud disease manifestation) of less than or equal to (<=) 6 years.
- Had mRSS of >=15 but <=40.
- Had active disease as determined by the Principal Investigator within the 6 months prior to screening.
- Adequate organ and bone marrow functions evaluated during the 28 days prior to enrollment as follows:
- Absolute neutrophil count >= 1.5*10^9/L
Show all 15Show fewer
- Platelet count >= 100*10^9/L
- Total bilirubin <= 1.0*upper limit of normal (ULN)
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST), and serum creatinine <= 1.5*ULN.
- Female participants of childbearing potential had a negative pregnancy test at screening. Females of childbearing potential were defined as sexually mature women without prior hysterectomy or who had any evidence of menses in the past 12 months. However, women who had been amenorrheic for 12 or more months were still considered to be of childbearing potential if the amenorrhea was possibly due to prior chemotherapy, anti-estrogens, or ovarian suppression.
- Women of childbearing potential (i.e., menstruating women) must had a negative urine pregnancy test (positive urine tests were to be confirmed by serum test) documented within the 24-hour period prior to the first dose of study drug.
- Sexually active women of childbearing potential enrolled in the study agreed to use two forms of accepted methods of contraception during the course of the study and for 3 months after their last dose of study drug. Effective birth control includes (i) intrauterine device plus 1 barrier method; (ii) on stable doses of hormonal contraception for at least 3 months (e.g., oral, injectable, implant, transdermal) plus 1 barrier method; or (iii) 2 barrier methods. Effective barrier methods are male or female condoms, diaphragms, and spermicides (creams or gels that contain a chemical to kill sperm), or a vasectomized partner.
- For male participants who were sexually active and who were partners of premenopausal women: agreement to use 2 forms of contraception as in criterion number 6b above during the treatment period and for at least 3 months after the last dose of study drug.
- Male participants must not donate sperm for 3 months after last dose of study drug.
- Able to provide written informed consent prior to the performance of any study-specific procedures.
Who cannot join
- Participants had corrected QT interval using Fridericia's formula (QTcF) greater than 450 milliseconds.
- Ongoing use or current use of concomitant medication known to have the potential for QTc prolongation.
- Female participant who was pregnant or breastfed.
- Participated in another study with an investigational drug within 28 days of study entry (for studies involving biologics, within 3 half-lives of the biologic).
- History or other evidence of severe illness or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study.
- Chronic heart failure with New York Heart Association Classes II, III, or IV.
Show all 13Show fewer
- Acute or chronic liver disease (e.g., cirrhosis).
- Positive human immunodeficiency virus (HIV) test.
- Active hepatitis C virus (HCV), hepatitis B virus (HBV), or positive whole blood tuberculin test.
- Diagnosed with any malignancy within 3 years of enrollment, with the exception of basal cell or completely resected squamous cell carcinoma of the skin, resected in situ cervical malignancy, resected breast ductal carcinoma in situ, or low risk prostate cancer after curative resection.
- Had previous exposure to belumosudil or known allergy/sensitivity to belumosudil, or any other Rho-associated Protein Kinase-2 (ROCK2) inhibitor.
- Scleroderma renal crisis within 4 months prior to enrollment.
- Forced vital capacity <= 50% Predicted.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Northwestern University_Site number 124
Chicago, Illinois, 60611
University of California, Los Angeles Medical Center_Site number 104
Los Angeles, California, 90095
Yale University School of Medicine_Site number 140
New Haven, Connecticut, 06519
Columbia University Medical Center_Site number 086
New York, New York, 10032
University of Utah_Site number 048
Salt Lake City, Utah, 84132
Virginia Mason Medical Center_Site number 145
Seattle, Washington, 98101
About this study
Official title: A Phase 2, Open-label Multicenter Study to Evaluate the Efficacy and Safety of Belumosudil in Subjects With Diffuse Cutaneous Systemic Sclerosis (dcSSc)
This was a phase 2, open-label, single-cohort, multicenter trial of belumosudil in participants with Diffuse Cutaneous Systemic Sclerosis (dcSSc). An estimated total of 12 to 15 participants would receive belumosudil 200 milligrams (mg) administered orally (PO) twice daily (BID) for 52 weeks. The primary analysis was at 24 weeks.
Read the detailed description
The primary objective of this phase 2, open-label, single-cohort, multicenter trial was to evaluate the efficacy of belumosudil 200 mg BID using the Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) after 24 weeks of therapy. The duration of the study was approximately 14 months (4 weeks for screening, 52 weeks of dosing period, and 4 weeks of Follow-up)
Participants who had signed an Institutional Review Board/Independent Ethics Committee-(IRB/IEC)-approved informed consent form (ICF) and met all of the inclusion/exclusion criteria were enrolled. A total of 10 participants at 6 sites received belumosudil 200 mg in tablet form administered PO BID for 52 weeks. The total duration of the study is approximately 14 months: a 4-week screening period, a 52-week treatment period, and a 4-week follow-up.
The primary endpoint was analyzed using Week 24 data.
Efficacy was assessed throughout the 52-week dosing period using:
- Composite Response Index in Systemic Sclerosis (CRISS)
- Modified Rodnan Skin Score (mRSS)
- Pulmonary Function Tests (PFTs)
- Physician Global Assessment
- Patient Global Assessment
Safety was be assessed throughout the study and will include:.
- Physical examinations (PEs)
- Vital sign measurements
- Weight measurements
- Blood sample collection for hematology and chemistry; urinalysis
- Electrocardiograms (ECGs)
- Adverse event (AE) assessments
- Concomitant medication assessments
- Pregnancy testing for females of childbearing potential.
Reasons for discontinuation of treatment because of adverse events were documented. Careful monitoring of all adverse events was carried out. Dosing could be reduced 1 dose level. If the dose was not tolerated, then the participant was discontinued from the study. If there is an interruption of dosing, after 14 days the participant was discontinued from the study.
Participants were given a study drug diary to record the details of each dose of belumosudil 200 mg. Diaries were dispense/collected on each visit. Compliance with dosing was confirmed using participant diaries, which was examined at each visit by site staff to determine if dosing was as instructed per protocol and follow-up.
A 4-Week Safety Follow-up Visit occurred 28 days (± 3 days) after the last dose of study drug.
- Conditions
- Diffuse Cutaneous Systemic Sclerosis
- Being tested
- Belumosudil
- Keywords
- skin diseases, systemic sclerosis, phase 2, Scleroderma Health Assessment Questionnaire Disability Index, Modified Rodnan Skin Score, Combined Response Index in dcSSc (CRISS), diffuse cutaneous systemic sclerosis
- Medical subject headings
- Scleroderma, Diffuse, Skin Diseases, Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BelumosudilDrug
Also called KD025
10 participants with dcSSc received belumosudil 200 mg PO BID for 52 weeks
| Study group | Role | What they receive |
|---|---|---|
| Belumosudil | Experimental | Participants received belumosudil 200 mg tablet orally PO, BID for 52 weeks. Receives: Drug: Belumosudil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Week 24
CRISS components included the following domains: modified Rodnan skin score (mRSS), forced vital capacity (FVC) percent predicted, physician global assessment, patient global assessment, and scleroderma health assessment questionnaire disability-index (SHAQ-DI). An algorithm determines the predicted probability of improvement from Baseline by incorporating change in the mRSS, FVC percent predicted, physician and patient global assessments, and SHAQ-DI. The outcome is a continuous variable between 0.0 and 1.0 (0 to 100%). A higher score indicated greater probability of improvement. Participants are not considered improved if they develop new onset of renal crisis, new onset or worsening of lung fibrosis, new onset of pulmonary arterial hypertension, new onset of left ventricular failure during the trial. CRISS score greater than 60% is considered the minimally important difference.
Measured: Week 24
Secondary outcomes (12)
Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Weeks 8, 16, 36, and 52
CRISS components included the following domains: mRSS, FVC percent predicted, physician global assessment, patient global assessment, and SHAQ-DI. An algorithm determines the predicted probability of improvement from Baseline by incorporating change in the mRSS, FVC percent predicted, physician and patient global assessments, and SHAQ-DI. The outcome is a continuous variable between 0.0 and 1.0 (0 to 100%). A higher score indicated greater probability of improvement. Participants are not considered improved if they develop new onset of renal crisis, new onset or worsening of lung fibrosis, new onset of pulmonary arterial hypertension, new onset of left ventricular failure during the trial. CRISS score greater than 60% is considered the minimally important difference.
Measured: Week 8, 16, 36 and 52
Modified Rodnan Skin Score (mRSS) at Week 24
The mRSS is an accepted clinical measure of the skin thickness. The investigator assessed the thickening of the skin using the modified Rodnan Skin Score through simple palpation on 17 different skin sites in the fingers, hands, forearms, arms, feet, legs, and thighs (bilaterally) and face, chest, and abdomen (singly). Each skin site was rated on a 0 to 3 scale; where 0 = normal skin, 1 = mild thickness, 2 = moderate thickness, and 3 = severe thickness and unable to pinch. Individual skin scores in the 17 body areas were summed and defined as the total mRSS which ranged from 0 (no thickening) to 51 (severe thickening), where higher score indicated more severity of skin thickening/worst outcome.
Measured: Week 24
Forced Vital Capacity (FVC) Level at Week 24
FVC was the total amount of air (in liters) exhaled from the lungs during the lung function test measured by spirometer which assessed the change in lung function related to the disease status of an underlying interstitial lung disease.
Measured: Week 24
Physician Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Week 24
The Physician Global Assessment (reported by the physician) quantified the participant's overall health during the last week based on a VAS which ranged from 0 (extremely poor) to 100 (excellent). Higher score indicated better outcome.
Measured: Week 24
Patient Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Week 24
The Patient Global Assessment (reported by participant) quantified the participant's overall health during the last week based on a VAS which ranged from 0 (extremely poor) to 100 (excellent). Higher score indicated better outcome.
Measured: Week 24
Scleroderma Health Assessment Questionnaire-Disability Index (SHAQ-DI) Total Score at Week 24
SHAQ-DI included the general health assessment questionnaire-disability index (HAD-DI) assessment and 6 scleroderma-specific VAS items to explore the impact of participant's disease. The general HAD-DI assessment included 8 domains addressing scleroderma related manifestations that contribute to disability. It is a quality of life measure. For each question, level of difficulty was scored from 0 to 3, where 0=no difficulty, 1=some difficulty, 2=much difficulty, 3=unable to do. Some domains in the SHAQ are visual analog scales that are measured first and then changed to a 0-3 scale. SHAQ-DI total score was computed as the sum of domain scores divided by the number of domains answered and it ranged from 0 (no difficulty) to 3 (maximum difficulty), where higher score indicated greater disability/worse functionality.
Measured: Week 24
Change From Baseline in Modified Rodnan Skin Thickness Score (mRSS) at Weeks 8, 16, 36, and 52
The mRSS is an accepted clinical measure of the skin thickness. The investigator assessed the thickening of the skin using the modified Rodnan Skin Score through simple palpation on 17 different skin sites in the fingers, hands, forearms, arms, feet, legs, and thighs (bilaterally) and face, chest, and abdomen (singly). Each skin site was rated on a 0 to 3 scale; where 0 = normal skin, 1 = mild thickness, 2 = moderate thickness, and 3 = severe thickness and unable to pinch. Individual skin scores in the 17 body areas were summed and defined as the total mRSS which ranged from 0 (no thickening) to 51 (severe thickening), where higher score indicated more severity of skin thickening/worst outcome. A negative change from baseline demonstrates improvement.
Measured: Baseline, Week 8, 16, 36 and 52
Change From Baseline in Forced Vital Capacity (FVC) Level at Weeks 8, 16, 36, and 52
FVC was the total amount of air (in liters) exhaled from the lungs during the lung function test measured by spirometer which assessed the change in lung function related to the disease status of an underlying interstitial lung disease. Change from Baseline was calculated by subtracting Baseline value from specified values at each reported week (Week 8, 16, 36 and 52).
Measured: Baseline, Week 8, 16, 36 and 52
Change From Baseline in Physician Global Assessment (Reported by the Physician) Quantified of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Weeks 8, 16, 36, and 52
The Physician Global Assessment (reported by the physician) quantified the participant's overall health during the last week based on a VAS which ranged from 0 (extremely poor) to 100 (excellent). Higher score indicated better outcome. A positive change from Baseline demonstrated improvement.
Measured: Baseline, Week 8, 16, 36 and 52
Change From Baseline in Patient Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Weeks 8, 16, 36, and 52
The Patient Global Assessment (reported by participant) quantified the participant's overall health during the last week based on a VAS which ranged from 0 (extremely poor) to 100 (excellent). Higher score indicated better outcome. A positive change from Baseline demonstrated improvement.
Measured: Baseline, Week 8, 16, 36 and 52
Change From Baseline in Scleroderma Health Assessment Questionnaire-Disability Index (SHAQ-DI) Total Score at Weeks 8, 16, 36, and 52
SHAQ-DI VAS included the general HAD-DI assessment and 6 scleroderma-specific VAS items to explore the impact of participant's disease. The general HAD-DI assessment included 8 domains addressing scleroderma related manifestations that contribute to disability. It is a quality of life measure. Each activity category consisted of 2 to 3 items. For each question, level of difficulty was scored from 0 to 3, where 0=no difficulty, 1=some difficulty, 2=much difficulty, 3=unable to do. Some domains in the SHAQ are visual analog scales that are measured first and then changed to a 0-3 scale. SHAQ-DI total score was computed as the sum of domain scores divided by the number of domains answered and it ranged from 0 (no difficulty) to 3 (maximum difficulty), where higher score indicated greater disability/worse functionality.
Measured: Baseline, Week 8, 16, 36 and 52
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Adverse event (AE): any untoward medical occurrence in a participant who received investigational medicinal product (IMP) without regard to possibility of causal relationship with the treatment. TEAEs: AEs that developed/worsened or became serious during treatment-emergent period (time of first dose administration of study medication up to 28 days after last dose). Serious AE (SAE): any untoward medical occurrence resulted in any of following outcomes: death, life-threatening, required initial/prolonged in-patient hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event.
Measured: From first dose administration (i.e., Day 1) of study medication up to 28 days after last dose (i.e., up to 57.5 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2020
- Study start3 Mar 2021
- Primary completion10 May 2022
- Study completion19 Dec 2022
Why it stopped: Sponsor decision due to slow enrollment and strategic consideration; not driven by any safety concerns.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Diffuse Cutaneous Systemic SclerosisStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Belumosudil 200 mg PO BID
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kadmon, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04680975
- Sponsor's protocol ID
- ACT17634
- Other IDs
- KD025-215
- Acronym
- dcSSC
- Status last verified
- May 2023
- First submitted
- 11 Dec 2020
- Last updated
- 5 Jun 2023
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized, and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org.
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-label Multicenter Study to Evaluate the Efficacy and Safety of Belumosudil in Subjects With Diffuse Cutaneous Systemic Sclerosis (dcSSc)
- ClinicalTrials.gov ID
- NCT04680975
- Acronym / sponsor ID
- dcSSC / ACT17634
- Conditions
- Diffuse Cutaneous Systemic Sclerosis
- Keywords
- skin diseases, systemic sclerosis, phase 2, Scleroderma Health Assessment Questionnaire Disability Index, Modified Rodnan Skin Score, Combined Response Index in dcSSc (CRISS), diffuse cutaneous systemic sclerosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision due to slow enrollment and strategic consideration; not driven by any safety concerns.
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Kadmon, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 3 Mar 2021 (actual)
- Primary completion
- 10 May 2022 (actual)
- Study completion
- 19 Dec 2022 (actual)
- First posted
- 23 Dec 2020 (actual)
- Results
- Posted 5 Jun 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a phase 2, open-label, single-cohort, multicenter trial of belumosudil in participants with Diffuse Cutaneous Systemic Sclerosis (dcSSc). An estimated total of 12 to 15 participants would receive belumosudil 200 milligrams (mg) administered orally (PO) twice daily (BID) for 52 weeks. The primary analysis was at 24 weeks.
Detailed description
The primary objective of this phase 2, open-label, single-cohort, multicenter trial was to evaluate the efficacy of belumosudil 200 mg BID using the Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) after 24 weeks of therapy. The duration of the study was approximately 14 months (4 weeks for screening, 52 weeks of dosing period, and 4 weeks of Follow-up)
Participants who had signed an Institutional Review Board/Independent Ethics Committee-(IRB/IEC)-approved informed consent form (ICF) and met all of the inclusion/exclusion criteria were enrolled. A total of 10 participants at 6 sites received belumosudil 200 mg in tablet form administered PO BID for 52 weeks. The total duration of the study is approximately 14 months: a 4-week screening period, a 52-week treatment period, and a 4-week follow-up.
The primary endpoint was analyzed using Week 24 data.
Efficacy was assessed throughout the 52-week dosing period using:
- Composite Response Index in Systemic Sclerosis (CRISS)
- Modified Rodnan Skin Score (mRSS)
- Pulmonary Function Tests (PFTs)
- Physician Global Assessment
- Patient Global Assessment
Safety was be assessed throughout the study and will include:.
- Physical examinations (PEs)
- Vital sign measurements
- Weight measurements
- Blood sample collection for hematology and chemistry; urinalysis
- Electrocardiograms (ECGs)
- Adverse event (AE) assessments
- Concomitant medication assessments
- Pregnancy testing for females of childbearing potential.
Reasons for discontinuation of treatment because of adverse events were documented. Careful monitoring of all adverse events was carried out. Dosing could be reduced 1 dose level. If the dose was not tolerated, then the participant was discontinued from the study. If there is an interruption of dosing, after 14 days the participant was discontinued from the study.
Participants were given a study drug diary to record the details of each dose of belumosudil 200 mg. Diaries were dispense/collected on each visit. Compliance with dosing was confirmed using participant diaries, which was examined at each visit by site staff to determine if dosing was as instructed per protocol and follow-up.
A 4-Week Safety Follow-up Visit occurred 28 days (± 3 days) after the last dose of study drug.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Belumosudil 200 mg PO BID
| Arm | Type | Receives |
|---|---|---|
| Belumosudil | Experimental | Drug: BelumosudilParticipants received belumosudil 200 mg tablet orally PO, BID for 52 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Male and female participants greater than or equal to (>=) 18 years old with the diagnosis of dcSSc according to the 2013 American College of Rheumatology and European League Against Rheumatism.
- Had disease duration (defined as interval from first non Raynaud disease manifestation) of less than or equal to (<=) 6 years.
- Had mRSS of >=15 but <=40.
- Had active disease as determined by the Principal Investigator within the 6 months prior to screening.
- Adequate organ and bone marrow functions evaluated during the 28 days prior to enrollment as follows:
Key exclusion criteria (13 listed)
- Participants had corrected QT interval using Fridericia's formula (QTcF) greater than 450 milliseconds.
- Ongoing use or current use of concomitant medication known to have the potential for QTc prolongation.
- Female participant who was pregnant or breastfed.
- Participated in another study with an investigational drug within 28 days of study entry (for studies involving biologics, within 3 half-lives of the biologic).
- History or other evidence of severe illness or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study.
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 |
|---|---|---|
| BelumosudilAlso: KD02510 participants with dcSSc received belumosudil 200 mg PO BID for 52 weeks | Drug | Belumosudil |
Primary outcome
Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Week 24
Time frame: Week 24
CRISS components included the following domains: modified Rodnan skin score (mRSS), forced vital capacity (FVC) percent predicted, physician global assessment, patient global assessment, and scleroderma health assessment questionnaire disability-index (SHAQ-DI). An algorithm determines the predicted probability of improvement from Baseline by incorporating change in the mRSS, FVC percent predicted, physician and patient global assessments, and SHAQ-DI. The outcome is a continuous variable between 0.0 and 1.0 (0 to 100%). A higher score indicated greater probability of improvement. Participants are not considered improved if they develop new onset of renal crisis, new onset or worsening of lung fibrosis, new onset of pulmonary arterial hypertension, new onset of left ventricular failure during the trial. CRISS score greater than 60% is considered the minimally important difference.
12 secondary outcomes
Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Weeks 8, 16, 36, and 52
Time frame: Week 8, 16, 36 and 52
Modified Rodnan Skin Score (mRSS) at Week 24
Time frame: Week 24
Forced Vital Capacity (FVC) Level at Week 24
Time frame: Week 24
Physician Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Week 24
Time frame: Week 24
Patient Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Week 24
Time frame: Week 24
Scleroderma Health Assessment Questionnaire-Disability Index (SHAQ-DI) Total Score at Week 24
Time frame: Week 24
Change From Baseline in Modified Rodnan Skin Thickness Score (mRSS) at Weeks 8, 16, 36, and 52
Time frame: Baseline, Week 8, 16, 36 and 52
Change From Baseline in Forced Vital Capacity (FVC) Level at Weeks 8, 16, 36, and 52
Time frame: Baseline, Week 8, 16, 36 and 52
Change From Baseline in Physician Global Assessment (Reported by the Physician) Quantified of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Weeks 8, 16, 36, and 52
Time frame: Baseline, Week 8, 16, 36 and 52
Change From Baseline in Patient Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Weeks 8, 16, 36, and 52
Time frame: Baseline, Week 8, 16, 36 and 52
Change From Baseline in Scleroderma Health Assessment Questionnaire-Disability Index (SHAQ-DI) Total Score at Weeks 8, 16, 36, and 52
Time frame: Baseline, Week 8, 16, 36 and 52
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Time frame: From first dose administration (i.e., Day 1) of study medication up to 28 days after last dose (i.e., up to 57.5 weeks)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized, and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org.
Related trials for Diffuse Cutaneous Systemic Sclerosis
RecruitingPhase 1
A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease
SLE (Systemic Lupus), Lupus Erythematosus, Systemic
RecruitingPhase 1/2
Controlling Hyperactive Immunity With Long-lived Lymphocytes
Diffuse Cutaneous Systemic Sclerosis, Rheumatoid Arthritis (RA)
Active, not recruitingPhase 2
Belimumab and Rituximab Combination Therapy for the Treatment of Diffuse Cutaneous Systemic Sclerosis
Systemic Sclerosis
Active, not recruitingPhase 2
A Clinical Study to Evaluate Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis
Diffuse Cutaneous Systemic Sclerosis
RecruitingPhase 2
EncompaSSc: Evaluation of MTX-474 in Participants With Diffuse Cutaneous Systemic Sclerosis (dcSSc)
Diffuse Cutaneous Systemic Sclerosis
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.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Belumosudil in people with Diffuse Cutaneous Systemic Sclerosis, run by Kadmon, a Sanofi Company at 6 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 15 inclusion and 13 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: stopped early, as of the sponsor's last update on 5 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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?
Yes. Results are posted in the registry (first posted 5 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Week 24 (time frame: Week 24). 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 5 Jun 2023.