Clinical trial NCT00613171: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Systemic Sclerosis, Scleroderma
Keywords: Systemic sclerosis, scleroderma, fibrosis, STI571, imatinib
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2008-02-12
Enrollment: 27
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


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

Primary outcomes:
measure: Change From Baseline in Modified Rodnan Skin Score (MRSS) at Each Time Point of Analysis
timeFrame: Baseline, Weeks 2, 4, 6, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and Week 48/End of Study (EOS)
measure: Number of Participants With Adverse Events (AE's) and Serious Adverse Events (SAE's)
timeFrame: Baseline to Week 48/EOS
Secondary outcomes:
measure: Number of Participants With Non-response, Partial Response, Complete Response, and Remission Assessed by MRSS Values
timeFrame: Weeks 2, 4, 6, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and Week 48/End of Study (EOS)
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Novartis Investigator Site
state: Illinois
city: Baltimore
country: United States
facility: Novartis Investigator Site
state: Maryland
city: Boston
country: United States
facility: Novartis Investigator Site
state: Massachusetts
city: Erlangen
country: Germany
facility: Novartis Investigator Site
city: Florence
country: Italy
facility: Novartis Investigator Site
city: Zurich
country: Switzerland
facility: Novartis Investigator Site
city: London
country: United Kingdom
facility: Novartis Investigator Site
Study officials:
affiliation: Novartis investigator site
name: NOVARTIS
role: PRINCIPAL_INVESTIGATOR
References: