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Efficacy and Tolerability of STI571 (Imatinib Mesylate) for the Treatment of Fibrosis in Participants With Systemic Sclerosis

Primary Purpose

Systemic Sclerosis, Scleroderma

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
STI571
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Systemic Sclerosis, Scleroderma focused on measuring Systemic sclerosis, scleroderma, fibrosis, STI571, imatinib

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Male and female participants who are equal to or older than 18 years of age and who have early diffuse cutaneous systemic sclerosis (Disease duration < 18 months from the first non-Raynaud's symptom)
  • Participants with a modified Rodnans Skin Score (MRSS) of at least 20 in the absence of trunk involvement or a MRSS of at least 16 in patients with trunk involvement
  • Female patients of childbearing potential practicing two acceptable forms of contraception

Exclusion Criteria:

  • SSc patients with a MRSS greater than 35
  • Concurrent connective tissue diseases other than systemic sclerosis
  • Significant pre-existing heart, liver, lungs, digestive system, blood and other diseases, cancer
  • Conditions that might mimic the potential side effects of STI571 (blood conditions, liver damage, chronic diarrhea, edema)
  • Concurrent medical therapies (or during last 6 weeks before first dosing) that may potentially influence outcome of the study
  • Allergic to the study medication
  • Pregnancy
  • Breast feeding

Sites / Locations

  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ST1571

Arm Description

Participants received ST1571 100 mg tablets, orally, once daily. Initiated at an oral dose of 200 mg/day for 4 weeks then titrated up to 400 mg/day for 2 weeks followed by 600 mg/day until Week 24, if well tolerated.

Outcomes

Primary Outcome Measures

Change From Baseline in Modified Rodnan Skin Score (MRSS) at Each Time Point of Analysis
The efficacy of oral STI571 in participants with systemic sclerosis is defined by an improvement in MRSS. Skin thickness was assessed clinically in each of 17 body areas and scored using a 0-3 scale, where 0= normal, 1= mild thickness, 2= moderate thickness, and 3= severe thickness (maximum score 51). A higher score indicates greater severity of the disease.
Number of Participants With Adverse Events (AE's) and Serious Adverse Events (SAE's)
An AE is the appearance or worsening of any undesirable sign, symptom, or medical condition occurring after starting the study drug even if the event is not considered to be related to the study drug. An SAE is defined as an event that is fatal or life-threatening, results in persistent or significant disability/incapacity, constitutes a congenital anomaly/birth defect, requires inpatient hospitalization or prolongation of existing hospitalization, is medically significant, i.e., defined as an event that jeopardizes the patient or may require medical or surgical intervention to prevent one of the outcomes listed above.

Secondary Outcome Measures

Number of Participants With Non-response, Partial Response, Complete Response, and Remission Assessed by MRSS Values
The following MRSS categories were calculated for up to Week 48: Non-response: a reduction in MRSS <25%, Partial response: a reduction in MRSS between 25-<50%, Complete response: a reduction in MRSS between 50-<80%, Remission: a reduction in MRSS ≥80%.

Full Information

First Posted
January 25, 2008
Last Updated
July 5, 2021
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT00613171
Brief Title
Efficacy and Tolerability of STI571 (Imatinib Mesylate) for the Treatment of Fibrosis in Participants With Systemic Sclerosis
Official Title
A Multi-centre, Open-label, Proof of Concept (PoC) Study to Evaluate the Efficacy and Tolerability of STI571 for the Treatment of Fibrosis in Patients With Systemic Sclerosis
Study Type
Interventional

2. Study Status

Record Verification Date
July 2021
Overall Recruitment Status
Completed
Study Start Date
January 2, 2008 (Actual)
Primary Completion Date
January 13, 2010 (Actual)
Study Completion Date
January 13, 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
This study investigates the efficacy and safety of STI571 for the treatment of fibrosis in participants with systemic sclerosis. Other purposes of the study were to investigate whether STI571 is effective in improving lung functions and other test results called biomarkers. Whether STI571 is well-absorbed in systemic sclerosis participants' gut was also investigated by testing the drug level in the blood (pharmacokinetics).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Systemic Sclerosis, Scleroderma
Keywords
Systemic sclerosis, scleroderma, fibrosis, STI571, imatinib

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
27 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ST1571
Arm Type
Experimental
Arm Description
Participants received ST1571 100 mg tablets, orally, once daily. Initiated at an oral dose of 200 mg/day for 4 weeks then titrated up to 400 mg/day for 2 weeks followed by 600 mg/day until Week 24, if well tolerated.
Intervention Type
Drug
Intervention Name(s)
STI571
Other Intervention Name(s)
Gleevec, Glivec
Intervention Description
STI571 tablets taken orally once a day
Primary Outcome Measure Information:
Title
Change From Baseline in Modified Rodnan Skin Score (MRSS) at Each Time Point of Analysis
Description
The efficacy of oral STI571 in participants with systemic sclerosis is defined by an improvement in MRSS. Skin thickness was assessed clinically in each of 17 body areas and scored using a 0-3 scale, where 0= normal, 1= mild thickness, 2= moderate thickness, and 3= severe thickness (maximum score 51). A higher score indicates greater severity of the disease.
Time Frame
Baseline, Weeks 2, 4, 6, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and Week 48/End of Study (EOS)
Title
Number of Participants With Adverse Events (AE's) and Serious Adverse Events (SAE's)
Description
An AE is the appearance or worsening of any undesirable sign, symptom, or medical condition occurring after starting the study drug even if the event is not considered to be related to the study drug. An SAE is defined as an event that is fatal or life-threatening, results in persistent or significant disability/incapacity, constitutes a congenital anomaly/birth defect, requires inpatient hospitalization or prolongation of existing hospitalization, is medically significant, i.e., defined as an event that jeopardizes the patient or may require medical or surgical intervention to prevent one of the outcomes listed above.
Time Frame
Baseline to Week 48/EOS
Secondary Outcome Measure Information:
Title
Number of Participants With Non-response, Partial Response, Complete Response, and Remission Assessed by MRSS Values
Description
The following MRSS categories were calculated for up to Week 48: Non-response: a reduction in MRSS <25%, Partial response: a reduction in MRSS between 25-<50%, Complete response: a reduction in MRSS between 50-<80%, Remission: a reduction in MRSS ≥80%.
Time Frame
Weeks 2, 4, 6, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and Week 48/End of Study (EOS)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male and female participants who are equal to or older than 18 years of age and who have early diffuse cutaneous systemic sclerosis (Disease duration < 18 months from the first non-Raynaud's symptom) Participants with a modified Rodnans Skin Score (MRSS) of at least 20 in the absence of trunk involvement or a MRSS of at least 16 in patients with trunk involvement Female patients of childbearing potential practicing two acceptable forms of contraception Exclusion Criteria: SSc patients with a MRSS greater than 35 Concurrent connective tissue diseases other than systemic sclerosis Significant pre-existing heart, liver, lungs, digestive system, blood and other diseases, cancer Conditions that might mimic the potential side effects of STI571 (blood conditions, liver damage, chronic diarrhea, edema) Concurrent medical therapies (or during last 6 weeks before first dosing) that may potentially influence outcome of the study Allergic to the study medication Pregnancy Breast feeding
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
NOVARTIS
Organizational Affiliation
Novartis investigator site
Official's Role
Principal Investigator
Facility Information:
Facility Name
Novartis Investigator Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Novartis Investigator Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21224
Country
United States
Facility Name
Novartis Investigator Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Novartis Investigator Site
City
Erlangen
Country
Germany
Facility Name
Novartis Investigator Site
City
Florence
Country
Italy
Facility Name
Novartis Investigator Site
City
Zurich
Country
Switzerland
Facility Name
Novartis Investigator Site
City
London
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

Efficacy and Tolerability of STI571 (Imatinib Mesylate) for the Treatment of Fibrosis in Participants With Systemic Sclerosis

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