Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| KD025-215 | Other Identifier | Kadmon |
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Sponsor decision due to slow enrollment and strategic consideration; not driven by any safety concerns.
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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.
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:
Safety was be assessed throughout the study and will include:.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Belumosudil | Experimental | Participants received belumosudil 200 mg tablet orally PO, BID for 52 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Belumosudil | Drug | 10 participants with dcSSc received belumosudil 200 mg PO BID for 52 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
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:
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.
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.
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, Los Angeles Medical Center_Site number 104 | Los Angeles | California | 90095 | United States | ||
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.
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A total of 10 participants were enrolled and treated with belumosudil in the study.
The study was conducted at 6 active sites. A total of 11 participants were screened between 03 Mar 2021 and 23 Nov 2021, of which 1 participant was screen failure due to not meeting eligibility criteria.
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| ID | Title | Description |
|---|---|---|
| FG000 | Belumosudil | Participants received belumosudil 200 milligrams (mg) tablet orally (PO), twice a day (BID) for 52 weeks. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Analysis was performed on modified intent to treat (mITT) population which included all participants who received at least 1 dose of belumosudil 200 mg.
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| ID | Title | Description |
|---|---|---|
| BG000 | Belumosudil | Participants received belumosudil 200 mg tablet PO, BID for 52 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Analysis was performed on mITT population. Here, overall number of participants analyzed signifies participants with available data for this outcome measure. | Posted | Mean | Standard Deviation | score on a scale | Week 24 |
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)
Reported AEs and deaths were treatment emergent AEs that developed, worsened, or became serious during treatment emergent period (time from first dose of study drug up to 28 days after the last dose of study drug). Analysis was performed on safety population.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Belumosudil | Participants received belumosudil 200 mg tablet orally PO, BID for 52 weeks. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dehydration | Metabolism and nutrition disorders | MedDRA 23.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sinus Bradycardia | Cardiac disorders | MedDRA 23.1 | Systematic Assessment |
Due to slow enrollment and strategic consideration, Sponsor decided to terminate the study and the termination was not driven by any safety concerns.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Sanofi (Kadmon, a Sanofi Company) | 800-633-1610 | 6# | Contact-US@sanofi.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 1, 2021 | May 8, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 20, 2023 | May 8, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D045743 | Scleroderma, Diffuse |
| D012871 | Skin Diseases |
| D012595 | Scleroderma, Systemic |
| ID | Term |
|---|---|
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
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| ID | Term |
|---|---|
| C000718240 | belumosudil |
| C000619755 | KD025 |
Not provided
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Belumosudil 200 mg PO BID
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| 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. | 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. | 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. | 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. | 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. | 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. | 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). | 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. | 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. | 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. | 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. | 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) |
| Yale University School of Medicine_Site number 140 |
| New Haven |
| Connecticut |
| 06519 |
| United States |
| Northwestern University_Site number 124 | Chicago | Illinois | 60611 | United States |
| Columbia University Medical Center_Site number 086 | New York | New York | 10032 | United States |
| University of Utah_Site number 048 | Salt Lake City | Utah | 84132 | United States |
| Virginia Mason Medical Center_Site number 145 | Seattle | Washington | 98101 | United States |
| Withdrawal by Subject |
|
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| ID |
|---|
| Title |
|---|
| Description |
|---|
| OG000 | Belumosudil | Participants received belumosudil 200 mg tablet orally PO, BID for 52 weeks. |
|
|
| Secondary | 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. | Analysis was performed on mITT population. Here, "number analyzed" signifies participants with available data for each specified category. | Posted | Mean | Standard Deviation | score on a scale | Week 8, 16, 36 and 52 |
|
|
|
| Secondary | 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. | Analysis was performed on mITT population. Here," overall number of participants analyzed" signifies participants with available data for this outcome measure. | Posted | Mean | Standard Deviation | score on a scale | Week 24 |
|
|
|
| Secondary | 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. | Analysis was performed on mITT population. Here," overall number of participants analyzed" signifies participants with available data for this outcome measure. | Posted | Mean | Standard Deviation | liters | Week 24 |
|
|
|
| Secondary | 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. | Analysis was performed on mITT population. Here," overall number of participants analyzed" signifies participants with available data for this outcome measure. | Posted | Mean | Standard Deviation | score on a scale | Week 24 |
|
|
|
| Secondary | 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. | Analysis was performed on mITT population. Here," overall number of participants analyzed" signifies participants with available data for this outcome measure. | Posted | Mean | Standard Deviation | score on a scale | Week 24 |
|
|
|
| Secondary | 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. | Analysis was performed on mITT population. Here," overall number of participants analyzed" signifies participants with available data for this outcome measure. | Posted | Mean | Standard Deviation | score on a scale | Week 24 |
|
|
|
| Secondary | 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. | Analysis was performed on mITT population. Here, "number analyzed" signifies participants with available data for each specified category. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Week 8, 16, 36 and 52 |
|
|
|
| Secondary | 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). | Analysis was performed on the mITT population. Here, "number analyzed" signifies participants with available data for each specified category. | Posted | Mean | Standard Deviation | liters | Baseline, 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 | 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. | Analysis was performed on the mITT population. Here, "number analyzed" signifies participants with available data for each specified category. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Week 8, 16, 36 and 52 |
|
|
|
| Secondary | 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. | Analysis was performed on the mITT population. Here, "number analyzed" signifies participants with available data for each specified category. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Week 8, 16, 36 and 52 |
|
|
|
| Secondary | 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. | Analysis was performed on the mITT population. Here, "number analyzed" signifies participants with available data for each specified category. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Week 8, 16, 36 and 52 |
|
|
|
| Secondary | 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. | Analysis was performed on safety population which included all participants who received at least 1 dose of belumosudil 200 mg. | Posted | Count of Participants | Participants | 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) |
|
|
|
| 1 |
| 10 |
| 4 |
| 10 |
| 10 |
| 10 |
| Intraductal Proliferative Breast Lesion | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.1 | Systematic Assessment |
|
| Syncope | Nervous system disorders | MedDRA 23.1 | Systematic Assessment |
|
| Acute Kidney Injury | Renal and urinary disorders | MedDRA 23.1 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA 23.1 | Systematic Assessment |
|
| Shock | Vascular disorders | MedDRA 23.1 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 23.1 | Systematic Assessment |
|
| Ear Pain | Ear and labyrinth disorders | MedDRA 23.1 | Systematic Assessment |
|
| Dry Eye | Eye disorders | MedDRA 23.1 | Systematic Assessment |
|
| Photophobia | Eye disorders | MedDRA 23.1 | Systematic Assessment |
|
| Vision Blurred | Eye disorders | MedDRA 23.1 | Systematic Assessment |
|
| Abdominal Discomfort | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Abdominal Distension | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Abdominal Pain | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Gastrooesophageal Reflux Disease | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Oral Pain | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Stomatitis | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Tooth Discolouration | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 23.1 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 23.1 | Systematic Assessment |
|
| Covid-19 | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
|
| Coronavirus Infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
|
| Gastrointestinal Bacterial Overgrowth | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
|
| Helicobacter Gastritis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
|
| Helicobacter Infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
|
| Oral Candidiasis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
|
| Postoperative Wound Infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
|
| Rash Pustular | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
|
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Urinary Tract Infection | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Procedural Pain | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
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| Road Traffic Accident | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
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| Urine Analysis Abnormal | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Weight Decreased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Decreased Appetite | Metabolism and nutrition disorders | MedDRA 23.1 | Systematic Assessment |
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| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 23.1 | Systematic Assessment |
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| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 23.1 | Systematic Assessment |
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| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 23.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Muscular Weakness | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Systemic Scleroderma | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Temporomandibular Joint Syndrome | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA 23.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 23.1 | Systematic Assessment |
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| Memory Impairment | Nervous system disorders | MedDRA 23.1 | Systematic Assessment |
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| Peripheral Sensory Neuropathy | Nervous system disorders | MedDRA 23.1 | Systematic Assessment |
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| Abnormal Dreams | Psychiatric disorders | MedDRA 23.1 | Systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA 23.1 | Systematic Assessment |
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| Irritability | Psychiatric disorders | MedDRA 23.1 | Systematic Assessment |
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| Breast Mass | Reproductive system and breast disorders | MedDRA 23.1 | Systematic Assessment |
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| Vaginal Haemorrhage | Reproductive system and breast disorders | MedDRA 23.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Angioedema | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Hypertrichosis | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Skin Tightness | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Skin Ulcer | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA 23.1 | Systematic Assessment |
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| Raynaud's Phenomenon | Vascular disorders | MedDRA 23.1 | Systematic Assessment |
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The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
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