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

NCT00613171 | Completed Phase 2 Results FDA Drug

• 1 other identifier: CSTI571E2205
• Study Type: interventional
• Target: 27
• Locations: 5 countries
• Sites: 7

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).

Conditions

Systemic Sclerosis, Scleroderma

Keywords

Systemic sclerosis, scleroderma, fibrosis, STI571, imatinib

Outcome Measures

Primary Outcomes (2)

• 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.

  Baseline, Weeks 2, 4, 6, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and Week 48/End of Study (EOS)

• 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.

  Baseline to Week 48/EOS

Secondary Outcomes (1)

• Number of Participants With Non-response, Partial Response, Complete Response, and Remission Assessed by MRSS Values

  Weeks 2, 4, 6, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and Week 48/End of Study (EOS)

Study Arms (1)

ST1571

EXPERIMENTAL

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.

Drug: STI571

Interventions

STI571 DRUG

STI571 tablets taken orally once a day

Also known as: Gleevec, Glivec

ST1571

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (7)

Novartis Investigator Site

Chicago, Illinois, 60611, United States

Location

Novartis Investigator Site

Baltimore, Maryland, 21224, United States

Location

Novartis Investigator Site

Boston, Massachusetts, 02118, United States

Location

Novartis Investigator Site

Erlangen, Germany

Location

Novartis Investigator Site

Florence, Italy

Location

Novartis Investigator Site

Zurich, Switzerland

Location

Novartis Investigator Site

London, United Kingdom

Location

MeSH Terms

Conditions

Scleroderma, Systemic; Scleroderma, Diffuse; Fibrosis

Interventions

Imatinib Mesylate

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Benzamides; Amides; Organic Chemicals; Benzoates; Acids, Carbocyclic; Carboxylic Acids; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Piperazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pyrimidines

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

862-778-8300

Study Officials

• NOVARTIS

  Novartis investigator site

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 25, 2008
• First Posted: February 12, 2008
• Study Start: January 2, 2008
• Primary Completion: January 13, 2010
• Study Completion: January 13, 2010
• Last Updated: July 7, 2021
• Results First Posted: May 21, 2021

Record last verified: 2021-07

Locations

US (3); CH (1); IT (1); GB (1); DE (1)