Trial · NCT01670565

Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis

NCT01670565, Phase 2, Completed, Hospital for Special Surgery, New York; results first posted 2022-05-24

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis. Trials, record NCT01670565. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01670565

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Trial

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

Title: Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis
NCT number: NCT01670565
Status: Completed
Phases: Phase 2
Enrollment: 20 participants, actual
Lead sponsor: Hospital for Special Surgery, New York
Conditions: Systemic Sclerosis
Interventions: Belimumab, Mycophenolate Mofetil, Placebo Infusion
First posted: 2012-08-22
Last updated: 2022-05-24
Results first posted: 2022-05-24

Source ClinicalTrials.gov

Registered outcomes7

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

List	Outcome	Time frame	Description
Primary	Change in Modified Rodnan Skin Score (MRSS)	Baseline and at 52 weeks	Change in MRSS is measured by median change (and interquartile range) from Baseline median to week 52 median. The efficacy of the drug will be measured as the change in the Modified Rodnan Skin Score (MRSS) at 52 weeks. The Modified Rodnan Skin Score (MRSS) measures dermal skin thickness through the examination of 17 body areas: fingers, hands, forearms, arms, feet, legs, and thighs (in pairs), and face, chest, and abdomen. The skin score is 0 for uninvolved skin through 3 for severe thickening (hidebound skin). The total skin score is the sum of the skin scores of the individual areas. The minimum score is 0 and the maximum score is 51. A higher score indicates greater severity of disease
Primary	Number of Adverse Events and Serious Adverse Events	At 52 weeks	The safety and tolerability of belimumab in patients with systemic sclerosis will be as assessed by comparing the rates of adverse events (AEs) and serious adverse events (SAEs) between treatment and placebo groups.
Primary	Change in Forced Vital Capacity (FVC)	Baseline and Week 52	Forced vital capacity (FVC) is the amount of air that can be forcibly exhaled from the lungs after taking a deep breath. It is used to determine the severity of lung disease. FVC for a given individual is compared to reference or predicted values. The reference values are based on healthy individuals with normal lung function and indicates values that would be expected for someone of the same sex, age and height. FVC % predicted compares the patients FVC values with the reference values. Results are considered normal if FVC is 80 percent or more of the predicted value. Change in FVC % predicted is measured by median change (and interquartile range) from Baseline median to week 52 median.
Primary	Change in Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)	Baseline and Week 52	Diffusing capacity of the lungs for carbon monoxide (DLCO) measures how much oxygen travels from the alveoli of the lungs to the blood stream. It is used to determine the severity of lung disease. DLCO for a given individual is compared to reference or predicted values. The reference values are based on healthy individuals with normal lung function and indicates values that would be expected for someone of the same sex, age and height. DLCO % predicted compares the patients DLCO values with the reference values. An individuals DLCO result that is at least 80% of the predicted value is considered normal. Change in DLCO % predicted is measured by median change (and interquartile range) from Baseline median to week 52 median.
Secondary	Change in Scleroderma Health Assessment Questionnaire Disability Index (SHAQ DI)	Baseline and Week 52	The Scleroderma Health Assessment Questionnaire (SHAQ) consist of the Health Assessment Questionnaire (HAQ) and 8 other domains addressing scleroderma related manifestations that contribute to disability. It is a quality of life measure. Each question is scored from 0 (without difficulty) to 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. The maximum from each category is added together and divided by the number of categories completed. The total scale range is 0-3. A higher score indicates worse functionality. Change in Scleroderma Health Assessment Questionnaire Disability Index (SHAQ-DI) is measured as median change (and interquartile range) from Baseline median to week 52 median. The reported median change can range from -3 to 3. A negative median change indicates a better outcome.
Secondary	Change in in Short Form-36 (SF-36) Questionnaire:Mental Component Summary	Baseline and at 52 weeks	The Short Form 36 (SF-36) is a validated 36 item questionnaire which measures quality of life across eight domains: physical functioning, role limitations due to physical health, role limitations due to emotional problems, energy/fatigue, emotional well-being, social functioning, pain, general health. The mental component score (MCS) is composed of a subset of the 8 health domains. Each component is directly transformed into a 0 to 100 scale on the assumption that each question carries equal weight. A score of 0 is equal to maximum disability, and a score of 100 is equivalent to no disability. The SF-36 mental component can be obtained by looking at the mean average of all the emotionally relevant items. Change in Short Form 36 mental (SF-36 MC) is measured by median change (and interquartile range) from Baseline to week 52. The median change can range from -100 to 100. A positive median change indicates indicates an improved outcome.
Secondary	Change in Short Form-36 (SF-36) Questionnaire: Physical Component Summary	Baseline and Week 52	The Short Form 36 (SF-36) is a 36 item questionnaire which measures quality of life across eight domains: physical functioning, role limitations due to physical health, role limitations due to emotional problems, energy/fatigue, emotional well-being, social functioning, pain, general health. The physical component score is composed of a subset of the 8 health domains.The SF-36 physical component can be obtained by looking at the mean average of all the physically relevant items. Each component is directly transformed into a 0 to 100 scale on the assumption that each question carries equal weight. A score of 0 is equal to maximum disability, and a score of 100 is equivalent to no disability. Change in Short Form 36 physical component (SF-36 PC) is measured by median change (and interquartile range) from Baseline to week 52. The median change can range from -100 to 100. A positive median change indicates indicates an improved outcome.

Source ClinicalTrials.gov

Outcomes7

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

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Change in Modified Rodnan Skin Score (MRSS)	Baseline and at 52 weeks	units on a scale	MEDIAN
PRIMARY	Number of Adverse Events and Serious Adverse Events	At 52 weeks	number of AEs	NUMBER
PRIMARY	Change in Forced Vital Capacity (FVC)	Baseline and Week 52	% predicted	MEDIAN
PRIMARY	Change in Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)	Baseline and Week 52	% predicted	MEDIAN
SECONDARY	Change in Scleroderma Health Assessment Questionnaire Disability Index (SHAQ DI)	Baseline and Week 52	score on a scale	MEDIAN
SECONDARY	Change in in Short Form-36 (SF-36) Questionnaire:Mental Component Summary	Baseline and at 52 weeks	score on a scale	MEDIAN
SECONDARY	Change in Short Form-36 (SF-36) Questionnaire: Physical Component Summary	Baseline and Week 52	scores on a scale, 0-100	MEDIAN

Source ClinicalTrials.gov

Results by group18

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

Outcome	Group	Category	Value	Spread	Units
Change in Modified Rodnan Skin Score (MRSS)	Mycophenolate Mofetil + Belimumab		-10	Inter-Quartile Range -13 to -9	units on a scale
Change in Modified Rodnan Skin Score (MRSS)	Mycophenolate Mofetil + Saline (Placebo)		-3.0	Inter-Quartile Range -15 to -1	units on a scale
Number of Adverse Events and Serious Adverse Events	Mycophenolate Mofetil + Belimumab	Total number of AEs	53		number of AEs
Number of Adverse Events and Serious Adverse Events	Mycophenolate Mofetil + Saline (Placebo)	Total number of AEs	56		number of AEs
Number of Adverse Events and Serious Adverse Events	Mycophenolate Mofetil + Belimumab	Total number of infectious AEs	18		number of AEs
Number of Adverse Events and Serious Adverse Events	Mycophenolate Mofetil + Saline (Placebo)	Total number of infectious AEs	16		number of AEs
Number of Adverse Events and Serious Adverse Events	Mycophenolate Mofetil + Belimumab	Serious AEs	0		number of AEs
Number of Adverse Events and Serious Adverse Events	Mycophenolate Mofetil + Saline (Placebo)	Serious AEs	3		number of AEs
Change in Forced Vital Capacity (FVC)	Mycophenolate Mofetil + Belimumab		5.00	Inter-Quartile Range 0.00 to 8.00	% predicted
Change in Forced Vital Capacity (FVC)	Mycophenolate Mofetil + Saline (Placebo)		-2.00	Inter-Quartile Range -6.00 to 4.00	% predicted

1 to 10 of 18Source ClinicalTrials.gov

See all 18 results by group

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Mycophenolate Mofetil + Belimumab	10
Overall Study	STARTED	Mycophenolate Mofetil + Saline (Placebo)	10
Overall Study	COMPLETED	Mycophenolate Mofetil + Belimumab	9
Overall Study	COMPLETED	Mycophenolate Mofetil + Saline (Placebo)	9
Overall Study	NOT COMPLETED	Mycophenolate Mofetil + Belimumab	1
Overall Study	NOT COMPLETED	Mycophenolate Mofetil + Saline (Placebo)	1

Source ClinicalTrials.gov

Adverse events136

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	Urinary Tract Infection	Renal and urinary disorders	Mycophenolate Mofetil + Belimumab	2	10	3
other	Urinary Tract Infection	Renal and urinary disorders	Mycophenolate Mofetil + Saline (Placebo)	3	10	6
other	Upper Respiratory Infection	Respiratory, thoracic and mediastinal disorders	Mycophenolate Mofetil + Belimumab	5	10	5
other	Upper Respiratory Infection	Respiratory, thoracic and mediastinal disorders	Mycophenolate Mofetil + Saline (Placebo)	4	10	4
other	Vomiting	Metabolism and nutrition disorders	Mycophenolate Mofetil + Belimumab	3	10	3
other	Vomiting	Metabolism and nutrition disorders	Mycophenolate Mofetil + Saline (Placebo)	1	10	1
other	Depression	Psychiatric disorders	Mycophenolate Mofetil + Belimumab	0	10	0
other	Depression	Psychiatric disorders	Mycophenolate Mofetil + Saline (Placebo)	2	10	3
other	Nausea	Metabolism and nutrition disorders	Mycophenolate Mofetil + Belimumab	1	10	2
other	Nausea	Metabolism and nutrition disorders	Mycophenolate Mofetil + Saline (Placebo)	1	10	1

1 to 10 of 136Source ClinicalTrials.gov

See all 136 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Mycophenolate Mofetil + Belimumab	0	10
Total, all-cause mortality	Mycophenolate Mofetil + Saline (Placebo)	0	10
Total, other adverse events	Mycophenolate Mofetil + Belimumab	9	10
Total, other adverse events	Mycophenolate Mofetil + Saline (Placebo)	9	10
Total, serious adverse events	Mycophenolate Mofetil + Belimumab	0	10
Total, serious adverse events	Mycophenolate Mofetil + Saline (Placebo)	2	10

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
29073351	DERIVED

Source PubMed

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 comparisons2

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

Comparison	Source
Mycophenolate Mofetil + Belimumab vs Mycophenolate Mofetil + Saline (Placebo): deaths from any cause	Trial comparisons
Mycophenolate Mofetil + Belimumab vs Mycophenolate Mofetil + Saline (Placebo): serious adverse events	Trial comparisons

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.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01670565

GET https://conductscience.org/api/trials/v1/NCT01670565/events?page=2

API reference For AI assistants