Systemic Sclerosis, Scleroderma Clinical Trial
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
| Verified date | July 2021 |
| Source | Novartis |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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).
| Status | Completed |
| Enrollment | 27 |
| Est. completion date | January 13, 2010 |
| Est. primary completion date | January 13, 2010 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility | 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 |
| Country | Name | City | State |
|---|---|---|---|
| Germany | Novartis Investigator Site | Erlangen | |
| Italy | Novartis Investigator Site | Florence | |
| Switzerland | Novartis Investigator Site | Zurich | |
| United Kingdom | Novartis Investigator Site | London | |
| United States | Novartis Investigator Site | Baltimore | Maryland |
| United States | Novartis Investigator Site | Boston | Massachusetts |
| United States | Novartis Investigator Site | Chicago | Illinois |
| Lead Sponsor | Collaborator |
|---|---|
| Novartis Pharmaceuticals |
United States, Germany, Italy, Switzerland, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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) | |
| Primary | 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 | 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%. | Weeks 2, 4, 6, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and Week 48/End of Study (EOS) |