CompletedPhase 2

Efficacy and Safety of Imatinib in Scleroderma

France28 participantsStarted 2007-12

Plain-language summary

In vitro studies have shown that imatinib 1mM inhibits strongly the growth of cutaneous fibroblasts. The hypothesis is that imatinib inhibits PDGFR which is known to be a potential target for the molecule, as recently also proposed after the discovery of autoantibodies activating the PDGF receptors. Recent data indicate that TGFb is also a potential target of imatinib. Cutaneous scleroderma is characterized by progressive cutaneous fibrosis caused by hyperactive dermal fibroblasts. Since no established treatment for skin sclerosis in scleroderma is currently available. This study will test the safety and efficacy of imatinib in the treatment of patients with scleroderma and severe cutaneous involvement.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * More than 18 years old * Documented diagnostic of scleroderma (systemic or cutaneous) * Severe cutaneous sclerodermia or systemic sclerodermia with m-Rodnan score \> 20/51 * Ejection fraction of more than 45 per cent at cardiac ultrasound pre-inclusion study * Woman with efficient contraceptive method during trail treatment and during 3 month after the end of trial treatment * All female patients with reproductive potential must have a negative pregnancy test (serum or urine) within the 7 days prior to enrolment * Affiliated or profit patient of a social security system * Signed informed consent Exclusion Criteria: * new systemic treatment, potentially interfering with disease progression, beginning 3 months prior the start trial treatment * Patient with isolated cutaneous scleroderma treated with a drug potentially interfering with the course of the disease 4 weeks before starting the trial (Systemic corticosteroids, methotrexate, cyclophosphamide, bosentan) * Scleroderma " en coup de sabre " * Severe organ failure or anomaly of blood chemistry/hematology (bilirubin, SGOT, SGPT, creatinine \> 1,5 ´ upper normal limit, polymorphonuclear granulocytes less than 1\*10\*9/l or platelets less than 50\*10\*9/l), * Ongoing cancer * Ejection fraction ≤ 45 per cent at cardiac ultrasound pre inclusion study * myocardial infarction of less than 6 mois at pre inclusion visit * Non controlled chronic illness (diabetes, chronic kidney failure, chronic hepatitis, HI…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Compare the efficacy of imatinib mesylate vs placebo based on the percent variation of modified Rodnan score (0-51) between inclusion and 6-month visits.

Timeframe: 6 month

Trial details

NCT IDNCT00479934

SponsorUniversity Hospital, Bordeaux

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2010-06

View on ClinicalTrials.gov More Scleroderma, Localized trials