Trial · NCT04680975

Efficacy and Safety of Belumosudil in Subjects With Diffuse Cutaneous Systemic Sclerosis

NCT04680975, Phase 2, Terminated, Kadmon, a Sanofi Company; results first posted 2023-06-05

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Efficacy and Safety of Belumosudil in Subjects With Diffuse Cutaneous Systemic Sclerosis. Trials, record NCT04680975. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04680975

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Efficacy and Safety of Belumosudil in Subjects With Diffuse Cutaneous Systemic Sclerosis
NCT number: NCT04680975
Status: Terminated
Phases: Phase 2
Enrollment: 10 participants, actual
Lead sponsor: Kadmon, a Sanofi Company
Conditions: Diffuse Cutaneous Systemic Sclerosis
Interventions: Belumosudil
First posted: 2020-12-23
Last updated: 2023-06-05
Results first posted: 2023-06-05
Limitations and caveats: Due to slow enrollment and strategic consideration, Sponsor decided to terminate the study and the termination was not driven by any safety concerns.

Source ClinicalTrials.gov

Registered outcomes13

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Week 24	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.
Secondary	Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Weeks 8, 16, 36, and 52	Week 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.
Secondary	Modified Rodnan Skin Score (mRSS) at Week 24	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.
Secondary	Forced Vital Capacity (FVC) Level at Week 24	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.
Secondary	Physician Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Week 24	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.
Secondary	Patient Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Week 24	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.
Secondary	Scleroderma Health Assessment Questionnaire-Disability Index (SHAQ-DI) Total Score at Week 24	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.
Secondary	Change From Baseline in Modified Rodnan Skin Thickness Score (mRSS) at Weeks 8, 16, 36, and 52	Baseline, Week 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.
Secondary	Change From Baseline in Forced Vital Capacity (FVC) Level at Weeks 8, 16, 36, and 52	Baseline, Week 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).
Secondary	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	Baseline, Week 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.

1 to 10 of 13Source ClinicalTrials.gov

See all 13 registered outcomes

Outcomes13

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Week 24	Week 24	Analysis was performed on mITT population. Here, overall number of participants analyzed signifies participants with available data for this outcome measure.	score on a scale	MEAN
SECONDARY	Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Weeks 8, 16, 36, and 52	Week 8, 16, 36 and 52	Analysis was performed on mITT population. Here, number analyzed signifies participants with available data for each specified category.	score on a scale	MEAN
SECONDARY	Modified Rodnan Skin Score (mRSS) at Week 24	Week 24	Analysis was performed on mITT population. Here, overall number of participants analyzed signifies participants with available data for this outcome measure.	score on a scale	MEAN
SECONDARY	Forced Vital Capacity (FVC) Level at Week 24	Week 24	Analysis was performed on mITT population. Here, overall number of participants analyzed signifies participants with available data for this outcome measure.	liters	MEAN
SECONDARY	Physician Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Week 24	Week 24	Analysis was performed on mITT population. Here, overall number of participants analyzed signifies participants with available data for this outcome measure.	score on a scale	MEAN
SECONDARY	Patient Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Week 24	Week 24	Analysis was performed on mITT population. Here, overall number of participants analyzed signifies participants with available data for this outcome measure.	score on a scale	MEAN
SECONDARY	Scleroderma Health Assessment Questionnaire-Disability Index (SHAQ-DI) Total Score at Week 24	Week 24	Analysis was performed on mITT population. Here, overall number of participants analyzed signifies participants with available data for this outcome measure.	score on a scale	MEAN
SECONDARY	Change From Baseline in Modified Rodnan Skin Thickness Score (mRSS) at Weeks 8, 16, 36, and 52	Baseline, Week 8, 16, 36 and 52	Analysis was performed on mITT population. Here, number analyzed signifies participants with available data for each specified category.	score on a scale	MEAN
SECONDARY	Change From Baseline in Forced Vital Capacity (FVC) Level at Weeks 8, 16, 36, and 52	Baseline, Week 8, 16, 36 and 52	Analysis was performed on the mITT population. Here, number analyzed signifies participants with available data for each specified category.	liters	MEAN
SECONDARY	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	Baseline, Week 8, 16, 36 and 52	Analysis was performed on the mITT population. Here, number analyzed signifies participants with available data for each specified category.	score on a scale	MEAN

1 to 10 of 13Source ClinicalTrials.gov

See all 13 outcomes

Results by group32

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Week 24	Belumosudil		6.65	Standard Deviation 12.926	score on a scale
Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Weeks 8, 16, 36, and 52	Belumosudil	Week 8	13.97	Standard Deviation 24.657	score on a scale
Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Weeks 8, 16, 36, and 52	Belumosudil	Week 16	20.48	Standard Deviation 41.654	score on a scale
Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Weeks 8, 16, 36, and 52	Belumosudil	Week 36	22.38	Standard Deviation 44.715	score on a scale
Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) Score at Weeks 8, 16, 36, and 52	Belumosudil	Week 52	25.49	Standard Deviation 49.674	score on a scale
Modified Rodnan Skin Score (mRSS) at Week 24	Belumosudil		24.2	Standard Deviation 11.16	score on a scale
Forced Vital Capacity (FVC) Level at Week 24	Belumosudil		85.7	Standard Deviation 25.28	liters
Physician Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Week 24	Belumosudil		61.2	Standard Deviation 30.32	score on a scale
Patient Global Assessment of Participant's Overall Health Using Visual Analogue Scale (VAS) Score at Week 24	Belumosudil		51.3	Standard Deviation 34.54	score on a scale
Scleroderma Health Assessment Questionnaire-Disability Index (SHAQ-DI) Total Score at Week 24	Belumosudil		1.750	Standard Deviation 1.072	score on a scale

1 to 10 of 32Source ClinicalTrials.gov

See all 32 results by group

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Belumosudil	10
Overall Study	COMPLETED	Belumosudil	4
Overall Study	NOT COMPLETED	Belumosudil	6

Source ClinicalTrials.gov

Adverse events70

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Sinus Bradycardia	Cardiac disorders	Belumosudil	1	10	1
other	Tachycardia	Cardiac disorders	Belumosudil	1	10	1
other	Ear Pain	Ear and labyrinth disorders	Belumosudil	1	10	1
other	Dry Eye	Eye disorders	Belumosudil	1	10	1
other	Photophobia	Eye disorders	Belumosudil	2	10	2
other	Vision Blurred	Eye disorders	Belumosudil	1	10	1
other	Abdominal Discomfort	Gastrointestinal disorders	Belumosudil	1	10	1
other	Abdominal Distension	Gastrointestinal disorders	Belumosudil	1	10	1
other	Abdominal Pain	Gastrointestinal disorders	Belumosudil	1	10	1
other	Diarrhoea	Gastrointestinal disorders	Belumosudil	6	10	7

1 to 10 of 70Source ClinicalTrials.gov

See all 70 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Belumosudil	1	10
Total, other adverse events	Belumosudil	10	10
Total, serious adverse events	Belumosudil	4	10

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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