Trial Outcomes & Findings for Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis (NCT NCT01670565)
NCT ID: NCT01670565
Last Updated: 2022-05-24
Results Overview
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
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
Baseline and at 52 weeks
Results posted on
2022-05-24
Participant Flow
Participant milestones
| Measure |
Mycophenolate Mofetil + Belimumab
Patients in this arm will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice titrated to 2 grams of MMF per day, followed by 10 mg/kg belimumab (Benlysta). Belimumab Infusion will be administered 14 times over a period of 48 weeks. MMF will be administered through 48 weeks.
Belimumab: Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic sclerosis.
|
Mycophenolate Mofetil + Saline (Placebo)
Patients in this arm will FIRST receive MMF alone followed by normal saline infusion that appears identical to the belimumab infusion. Saline Infusion will be administered 14 times over a period of 48 weeks. MMF will be administered through 48 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
10
|
|
Overall Study
COMPLETED
|
9
|
9
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
MRSS values are given for treated patients (n=9 per group) and not for all patients randomized (n=10 per group)
Baseline characteristics by cohort
| Measure |
Mycophenolate Mofetil + Belimumab
n=10 Participants
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After the patient has been titrated to 2 grams of MMF per day, the patient will receive EITHER a 10 mg/kg belimumab (Benlysta) intravenous infusion OR placebo (saline) infusion. This medication and infusion will be covered by the study.
Belimumab: Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic sclerosis.
|
Mycophenolate Mofetil + Saline (Placebo)
n=10 Participants
In order to observe the difference between belimumab/MMF compared to MMF alone, half of the patients will receive a normal saline infusion that appears identical to the belimumab infusion.
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
7 Participants
n=10 Participants
|
7 Participants
n=10 Participants
|
14 Participants
n=20 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=10 Participants
|
3 Participants
n=10 Participants
|
6 Participants
n=20 Participants
|
|
Age, Continuous
|
53 years
STANDARD_DEVIATION 12.1 • n=10 Participants
|
56.7 years
STANDARD_DEVIATION 10.26 • n=10 Participants
|
54.85 years
STANDARD_DEVIATION 11.18 • n=20 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=10 Participants
|
8 Participants
n=10 Participants
|
15 Participants
n=20 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=10 Participants
|
2 Participants
n=10 Participants
|
5 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=10 Participants
|
3 Participants
n=10 Participants
|
4 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=10 Participants
|
7 Participants
n=10 Participants
|
16 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=10 Participants
|
9 Participants
n=10 Participants
|
16 Participants
n=20 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=10 Participants
|
1 Participants
n=10 Participants
|
4 Participants
n=20 Participants
|
|
Region of Enrollment
United States
|
10 Participants
n=10 Participants
|
10 Participants
n=10 Participants
|
20 Participants
n=20 Participants
|
|
Interstitial Lung Disease (ILD)
|
1 Participants
n=10 Participants
|
2 Participants
n=10 Participants
|
3 Participants
n=20 Participants
|
|
Anti-Scl-70
|
2 Participants
n=10 Participants
|
3 Participants
n=10 Participants
|
5 Participants
n=20 Participants
|
|
Anti- RNA Pol III
|
7 Participants
n=10 Participants
|
3 Participants
n=10 Participants
|
10 Participants
n=20 Participants
|
|
Modified Rodnan Skin Score (MRSS) at baseline
|
27 units on a scale
n=9 Participants • MRSS values are given for treated patients (n=9 per group) and not for all patients randomized (n=10 per group)
|
28 units on a scale
n=9 Participants • MRSS values are given for treated patients (n=9 per group) and not for all patients randomized (n=10 per group)
|
27 units on a scale
n=18 Participants • MRSS values are given for treated patients (n=9 per group) and not for all patients randomized (n=10 per group)
|
|
Forced Vital Capacity (FVC)
|
88 % predicted
n=9 Participants • Values for treated patients such as FVC% predicted and DLCO% predicted are given for treated patients (n=9 per group) and not for all patients randomized (n=10).
|
95 % predicted
n=9 Participants • Values for treated patients such as FVC% predicted and DLCO% predicted are given for treated patients (n=9 per group) and not for all patients randomized (n=10).
|
94.5 % predicted
n=18 Participants • Values for treated patients such as FVC% predicted and DLCO% predicted are given for treated patients (n=9 per group) and not for all patients randomized (n=10).
|
|
Diffusing capacity of the lungs for carbon monoxide
|
85 % predicted
n=9 Participants • Values for treated patients such as FVC% predicted and DLCO% predicted are given for treated patients (n=9 per group) and not for all patients randomized (n=10).
|
81 % predicted
n=9 Participants • Values for treated patients such as FVC% predicted and DLCO% predicted are given for treated patients (n=9 per group) and not for all patients randomized (n=10).
|
83 % predicted
n=18 Participants • Values for treated patients such as FVC% predicted and DLCO% predicted are given for treated patients (n=9 per group) and not for all patients randomized (n=10).
|
PRIMARY outcome
Timeframe: Baseline and at 52 weeksChange 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
Outcome measures
| Measure |
Mycophenolate Mofetil + Belimumab
n=10 Participants
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a 10 mg/kg belimumab (Benlysta) intravenous infusion infusion.This medication and infusion will of course be covered by the study.
Belimumab: Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic
|
Mycophenolate Mofetil + Saline (Placebo)
n=10 Participants
In order to observe the difference between belimumab/MMF compared to MMF alone, half of the patients will receive a normal saline infusion that appears identical to the belimumab infusion.
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a placebo (saline) infusion.This infusion will of course be covered by the study.
.
|
|---|---|---|
|
Change in Modified Rodnan Skin Score (MRSS)
|
-10 units on a scale
Interval -13.0 to -9.0
|
-3.0 units on a scale
Interval -15.0 to -1.0
|
PRIMARY outcome
Timeframe: At 52 weeksThe 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.
Outcome measures
| Measure |
Mycophenolate Mofetil + Belimumab
n=10 Participants
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a 10 mg/kg belimumab (Benlysta) intravenous infusion infusion.This medication and infusion will of course be covered by the study.
Belimumab: Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic
|
Mycophenolate Mofetil + Saline (Placebo)
n=10 Participants
In order to observe the difference between belimumab/MMF compared to MMF alone, half of the patients will receive a normal saline infusion that appears identical to the belimumab infusion.
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a placebo (saline) infusion.This infusion will of course be covered by the study.
.
|
|---|---|---|
|
Number of Adverse Events and Serious Adverse Events
Serious AEs
|
0 number of AEs
|
3 number of AEs
|
|
Number of Adverse Events and Serious Adverse Events
Total number of AEs
|
53 number of AEs
|
56 number of AEs
|
|
Number of Adverse Events and Serious Adverse Events
Total number of infectious AEs
|
18 number of AEs
|
16 number of AEs
|
PRIMARY outcome
Timeframe: Baseline and Week 52Forced 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.
Outcome measures
| Measure |
Mycophenolate Mofetil + Belimumab
n=9 Participants
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a 10 mg/kg belimumab (Benlysta) intravenous infusion infusion.This medication and infusion will of course be covered by the study.
Belimumab: Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic
|
Mycophenolate Mofetil + Saline (Placebo)
n=9 Participants
In order to observe the difference between belimumab/MMF compared to MMF alone, half of the patients will receive a normal saline infusion that appears identical to the belimumab infusion.
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a placebo (saline) infusion.This infusion will of course be covered by the study.
.
|
|---|---|---|
|
Change in Forced Vital Capacity (FVC)
|
5.00 % predicted
Interval 0.0 to 8.0
|
-2.00 % predicted
Interval -6.0 to 4.0
|
PRIMARY outcome
Timeframe: Baseline and Week 52Diffusing 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.
Outcome measures
| Measure |
Mycophenolate Mofetil + Belimumab
n=9 Participants
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a 10 mg/kg belimumab (Benlysta) intravenous infusion infusion.This medication and infusion will of course be covered by the study.
Belimumab: Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic
|
Mycophenolate Mofetil + Saline (Placebo)
n=9 Participants
In order to observe the difference between belimumab/MMF compared to MMF alone, half of the patients will receive a normal saline infusion that appears identical to the belimumab infusion.
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a placebo (saline) infusion.This infusion will of course be covered by the study.
.
|
|---|---|---|
|
Change in Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)
|
2.00 % predicted
Interval -7.0 to 7.0
|
0.00 % predicted
Interval -6.0 to 7.0
|
SECONDARY outcome
Timeframe: Baseline and Week 52The 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.
Outcome measures
| Measure |
Mycophenolate Mofetil + Belimumab
n=9 Participants
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a 10 mg/kg belimumab (Benlysta) intravenous infusion infusion.This medication and infusion will of course be covered by the study.
Belimumab: Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic
|
Mycophenolate Mofetil + Saline (Placebo)
n=9 Participants
In order to observe the difference between belimumab/MMF compared to MMF alone, half of the patients will receive a normal saline infusion that appears identical to the belimumab infusion.
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a placebo (saline) infusion.This infusion will of course be covered by the study.
.
|
|---|---|---|
|
Change in Scleroderma Health Assessment Questionnaire Disability Index (SHAQ DI)
|
-0.25 score on a scale
Interval -0.38 to -0.25
|
0.00 score on a scale
Interval -0.13 to 0.13
|
SECONDARY outcome
Timeframe: Baseline and at 52 weeksThe 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.
Outcome measures
| Measure |
Mycophenolate Mofetil + Belimumab
n=9 Participants
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a 10 mg/kg belimumab (Benlysta) intravenous infusion infusion.This medication and infusion will of course be covered by the study.
Belimumab: Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic
|
Mycophenolate Mofetil + Saline (Placebo)
n=9 Participants
In order to observe the difference between belimumab/MMF compared to MMF alone, half of the patients will receive a normal saline infusion that appears identical to the belimumab infusion.
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a placebo (saline) infusion.This infusion will of course be covered by the study.
.
|
|---|---|---|
|
Change in in Short Form-36 (SF-36) Questionnaire:Mental Component Summary
|
7.50 score on a scale
Interval 2.5 to 18.5
|
3.00 score on a scale
Interval 0.0 to 10.0
|
SECONDARY outcome
Timeframe: Baseline and Week 52The 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.
Outcome measures
| Measure |
Mycophenolate Mofetil + Belimumab
n=9 Participants
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a 10 mg/kg belimumab (Benlysta) intravenous infusion infusion.This medication and infusion will of course be covered by the study.
Belimumab: Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic
|
Mycophenolate Mofetil + Saline (Placebo)
n=9 Participants
In order to observe the difference between belimumab/MMF compared to MMF alone, half of the patients will receive a normal saline infusion that appears identical to the belimumab infusion.
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a placebo (saline) infusion.This infusion will of course be covered by the study.
.
|
|---|---|---|
|
Change in Short Form-36 (SF-36) Questionnaire: Physical Component Summary
|
8.00 scores on a scale, 0-100
Interval -3.5 to 19.0
|
-3.00 scores on a scale, 0-100
Interval -3.0 to 27.0
|
Adverse Events
Mycophenolate Mofetil + Belimumab
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Mycophenolate Mofetil + Saline (Placebo)
Serious events: 2 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Mycophenolate Mofetil + Belimumab
n=10 participants at risk
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, the patient will receive a 10 mg/kg belimumab (Benlysta) intravenous infusion. This medication and infusion will be covered by the study.
Belimumab: Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic sclerosis.
|
Mycophenolate Mofetil + Saline (Placebo)
n=10 participants at risk
In order to observe the difference between belimumab/MMF compared to MMF alone, half of the patients will receive a normal saline infusion that appears identical to the belimumab infusion.
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a placebo (saline) infusion.This infusion will of course be covered by the study.
|
|---|---|---|
|
Psychiatric disorders
Anxiety Attack
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Cardiac disorders
Chest Pain
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 2 • 12 months
|
Other adverse events
| Measure |
Mycophenolate Mofetil + Belimumab
n=10 participants at risk
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, the patient will receive a 10 mg/kg belimumab (Benlysta) intravenous infusion. This medication and infusion will be covered by the study.
Belimumab: Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic sclerosis.
|
Mycophenolate Mofetil + Saline (Placebo)
n=10 participants at risk
In order to observe the difference between belimumab/MMF compared to MMF alone, half of the patients will receive a normal saline infusion that appears identical to the belimumab infusion.
All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After being titrated to 2 grams of MMF per day, patients in this experimental group will receive a placebo (saline) infusion.This infusion will of course be covered by the study.
|
|---|---|---|
|
Renal and urinary disorders
Urinary Tract Infection
|
20.0%
2/10 • Number of events 3 • 12 months
|
30.0%
3/10 • Number of events 6 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Infection
|
50.0%
5/10 • Number of events 5 • 12 months
|
40.0%
4/10 • Number of events 4 • 12 months
|
|
Metabolism and nutrition disorders
Vomiting
|
30.0%
3/10 • Number of events 3 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Psychiatric disorders
Depression
|
0.00%
0/10 • 12 months
|
20.0%
2/10 • Number of events 3 • 12 months
|
|
Metabolism and nutrition disorders
Nausea
|
10.0%
1/10 • Number of events 2 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Infections and infestations
Zoster
|
10.0%
1/10 • Number of events 1 • 12 months
|
10.0%
1/10 • Number of events 2 • 12 months
|
|
Gastrointestinal disorders
Diarrhea
|
10.0%
1/10 • Number of events 1 • 12 months
|
20.0%
2/10 • Number of events 2 • 12 months
|
|
Metabolism and nutrition disorders
Anemia
|
10.0%
1/10 • Number of events 1 • 12 months
|
20.0%
2/10 • Number of events 2 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
10.0%
1/10 • Number of events 1 • 12 months
|
20.0%
2/10 • Number of events 2 • 12 months
|
|
Immune system disorders
Lymphocyte count decreased
|
10.0%
1/10 • Number of events 1 • 12 months
|
20.0%
2/10 • Number of events 2 • 12 months
|
|
Infections and infestations
Lip infection (HSV-1)
|
10.0%
1/10 • Number of events 2 • 12 months
|
0.00%
0/10 • 12 months
|
|
Nervous system disorders
Dizziness
|
20.0%
2/10 • Number of events 2 • 12 months
|
0.00%
0/10 • 12 months
|
|
General disorders
Fatigue
|
20.0%
2/10 • Number of events 2 • 12 months
|
0.00%
0/10 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
20.0%
2/10 • Number of events 2 • 12 months
|
0.00%
0/10 • 12 months
|
|
Skin and subcutaneous tissue disorders
Rash Acneiform
|
0.00%
0/10 • 12 months
|
20.0%
2/10 • Number of events 2 • 12 months
|
|
Nervous system disorders
Headache
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 2 • 12 months
|
|
General disorders
Allergic Reaction (Infusion)
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
General disorders
ALT Increased
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
General disorders
AST Increased
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Renal and urinary disorders
Asymptomatic Bacteriuria
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Eye disorders
Blurred Vision
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Eye disorders
Conjunctival Hemorrhage
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Renal and urinary disorders
Cystitis noninfective
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Gastrointestinal disorders
Gastroesophageal Refluex Disease
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Generalized Muscle Weakness
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Blood and lymphatic system disorders
Hemoglobin A1c Increased
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Immune system disorders
IgM Decreased
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Infections and infestations
Infections and Infestations -Other (H Pylori, Stomach)
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Infections and infestations
Infections and Infestations -Other (Viral Syndrome)
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Infections and infestations
Laryngeal Inflammation
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Meniscal Tear
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Acroosteolysis
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Calcinosis
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
General disorders
Oral Hemorrhage
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Ear and labyrinth disorders
Otitis Media
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Nodule Benign
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Infections and infestations
Salivary Gland Infection
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Reproductive system and breast disorders
Testicular Disorder (hydrocele)
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
General disorders
Tooth Mobility
|
10.0%
1/10 • Number of events 1 • 12 months
|
0.00%
0/10 • 12 months
|
|
Psychiatric disorders
Anxiety (Acute)
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Vascular disorders
Aortic Valve Disease (progression)
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Eye disorders
Cataract
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Cardiac disorders
Chest-Pain Cardiac
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Skin and subcutaneous tissue disorders
Contact Dermatitis
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Blood and lymphatic system disorders
CPK Increased
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Fracture (Shoulder)
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Renal and urinary disorders
Hermaturia
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Blood and lymphatic system disorders
Hyperglycemia
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Reproductive system and breast disorders
Irregular Menstruation
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Irritable Bowel
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Infections and infestations
Lung Infection
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Nervous system disorders
Memory Impairment-acute
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Nervous system disorders
Neuralgia
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Skin and subcutaneous tissue disorders
Skin Ulceration (Leg)
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Infections and infestations
Soft Tissue Infection (wart)
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous Cell Carcinoma
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
General disorders
Syncope
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Nervous system disorders
Tremor
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Reproductive system and breast disorders
Vaginal Infection
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
|
Metabolism and nutrition disorders
Weight Loss
|
0.00%
0/10 • 12 months
|
10.0%
1/10 • Number of events 1 • 12 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place