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IL1-TRAP, Rilonacept, in Systemic Sclerosis

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ClinicalTrials.gov Identifier: NCT01538719
Recruitment Status : Completed
First Posted : February 24, 2012
Results First Posted : May 1, 2018
Last Update Posted : May 1, 2018
Sponsor:
Collaborator:
Regeneron Pharmaceuticals
Information provided by (Responsible Party):
Robert Simms, Boston University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Scleroderma
Systemic Sclerosis
Diffuse Scleroderma
Diffuse Systemic Sclerosis
Interventions Drug: Rilonacept
Other: Placebo
Enrollment 24
Recruitment Details  
Pre-assignment Details 2 subjects withdrew consent and 3 found to be ineligible, leaving 19 for randomization.
Arm/Group Title Placebo Rilonacept
Hide Arm/Group Description

2:1 randomization

Placebo: Patients randomized to placebo will receive saline subcutaneously (SQ) on day 0 and each week for 5 additional weeks

2:1 randomization

Rilonacept: Patients randomized to active study drug will receive Rilonacept 320 mg subcutaneously (SQ) on day 0 and 160 mg SQ each week for 5 additional weeks

Period Title: Overall Study
Started 7 12
Completed 5 12
Not Completed 2 0
Reason Not Completed
Lost to Follow-up             2             0
Arm/Group Title Placebo Rilonacept Total
Hide Arm/Group Description

2:1 randomization

Placebo: Patients randomized to placebo will receive saline subcutaneously (SQ) on day 0 and each week for 5 additional weeks

2:1 randomization

Rilonacept: Patients randomized to active study drug will receive Rilonacept 320 mg subcutaneously (SQ) on day 0 and 160 mg SQ each week for 5 additional weeks

Total of all reporting groups
Overall Number of Baseline Participants 7 12 19
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 7 participants 12 participants 19 participants
49.86  (11.10) 51.33  (7.33) 50.79  (8.63)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 12 participants 19 participants
Female
2
  28.6%
7
  58.3%
9
  47.4%
Male
5
  71.4%
5
  41.7%
10
  52.6%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 7 participants 12 participants 19 participants
7
 100.0%
12
 100.0%
19
 100.0%
1.Primary Outcome
Title Change in 2- Gene Biomarker
Hide Description To investigate the effect of rilonacept on 2-gene biomarker expression in skin after treatment with rilonacept compared to pre-treatment 2-gene biomarker expression. These were measured at visit 3 (Day 42) and visit 1 (Day 0). This was calculated using a previously validated equation (MRSS = −27.6844 + [4.46(baseline THBS1)] + [5.31(ΔMS4A4A) + 4.96(ΔTHBS1)]). In this equation the expression of two genes (THBS1 and MS4A4) in collected samples are measured via nanostring, and then the expression levels of each gene are inserted into the equation in order to obtain the 2- gene biomarker score. A high biomarker score is equivalent to a high skin score, suggesting a higher severity of the disease.
Time Frame Visit 3 (Day 42) - Visit 1 (Day 0)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Analysis run on 4 available samples in placebo arm.
Arm/Group Title Placebo Rilonacept
Hide Arm/Group Description:

2:1 randomization

Placebo: Patients randomized to placebo will receive saline subcutaneously (SQ) on day 0 and each week for 5 additional weeks

2:1 randomization

Rilonacept: Patients randomized to active study drug will receive Rilonacept 320 mg subcutaneously (SQ) on day 0 and 160 mg SQ each week for 5 additional weeks

Overall Number of Participants Analyzed 4 12
Mean (95% Confidence Interval)
Unit of Measure: 2- gene biomarkers score
-2.67
(-5.92 to 0.57)
0.42
(-2.97 to 3.82)
2.Secondary Outcome
Title Change in Modified Rodnan Skin Score
Hide Description Change in Modified Rodnan Skin Score over time. The fully validated modified version of the Rodnan skin thickness score was used. On this scale, a total of 17 skin sites are evaluated, including the face, upper arms, forearms, dorsum of the hands, fingers, chest, abdomen, thighs, forearms and feet. The total score can range from 0 to 51, with higher scores indicating greater severity of skin thickening and involvement (MRSS-51). Each of the 17 skin sites are scored from 0 to 3, where the following criteria apply: 0, normal skin; 1, thickened skin; 2, thickened and unable to pinch; and 3, thickened and unable to move. Scores from visit 3 (Day 42) and visit 1 (Day 0) were compared.
Time Frame Visit 3 (Day 42) - Visit 1 (Day 0)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Rilonacept
Hide Arm/Group Description:

2:1 randomization

Placebo: Patients randomized to placebo will receive saline subcutaneously (SQ) on day 0 and each week for 5 additional weeks

2:1 randomization

Rilonacept: Patients randomized to active study drug will receive Rilonacept 320 mg subcutaneously (SQ) on day 0 and 160 mg SQ each week for 5 additional weeks

Overall Number of Participants Analyzed 7 12
Mean (Full Range)
Unit of Measure: Units on the Modified Rodnan Skin Score
-0.4
(-5 to 9)
-0.6667
(-5 to 14)
Time Frame Adverse events were collected from screening (Day -28) up to study completion (visit 4 / Day 84)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Rilonacept
Hide Arm/Group Description

2:1 randomization

Placebo: Patients randomized to placebo will receive saline subcutaneously (SQ) on day 0 and each week for 5 additional weeks

2:1 randomization

Rilonacept: Patients randomized to active study drug will receive Rilonacept 320 mg subcutaneously (SQ) on day 0 and 160 mg SQ each week for 5 additional weeks

All-Cause Mortality
Placebo Rilonacept
Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)   0/12 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Rilonacept
Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)   0/12 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Placebo Rilonacept
Affected / at Risk (%) Affected / at Risk (%)
Total   2/7 (28.57%)   10/12 (83.33%) 
Gastrointestinal disorders     
Diarrhea   0/7 (0.00%)  1/12 (8.33%) 
General disorders     
Malaise   1/7 (14.29%)  0/12 (0.00%) 
Fatigue   0/7 (0.00%)  2/12 (16.67%) 
Skin and subcutaneous tissue disorders     
Pain   1/7 (14.29%)  0/12 (0.00%) 
Allergic Reaction on Abdomen   0/7 (0.00%)  1/12 (8.33%) 
Digital Ulcer Worsening   0/7 (0.00%)  1/12 (8.33%) 
Abscess on Back   0/7 (0.00%)  1/12 (8.33%) 
Left Breast Swelling   0/7 (0.00%)  1/12 (8.33%) 
Injection Site Reaction   0/7 (0.00%)  2/12 (16.67%) 
Infection   0/7 (0.00%)  1/12 (8.33%) 
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Eric A Stratton, Sr. Clinical Research Manager
Organization: Boston University School of Medicine
Phone: 6174142507
EMail: eas@bu.edu
Responsible Party: Robert Simms, Boston University
ClinicalTrials.gov Identifier: NCT01538719     History of Changes
Other Study ID Numbers: H31332
First Submitted: February 21, 2012
First Posted: February 24, 2012
Results First Submitted: January 5, 2018
Results First Posted: May 1, 2018
Last Update Posted: May 1, 2018