Thalidomide at Low Dose for the Treatment of Patient With Myelodysplastic Syndromes - THAL-SMD-200
- Conditions
- Myelodysplastic Syndromes
- Registration Number
- NCT00455910
- Lead Sponsor
- Groupe Francophone des Myelodysplasies
- Brief Summary
The GFM previously conducted a dose-escalating phase II trial of thalidomide in MDS with a minimum dose of 200mg/d and a maximum dose 800mg/d. Responses were evaluated according to IWG criteria at week 16 and thalidomide continued up to week 56 in responders. 82% patients received at least 8 weeks of treatment and were evaluable. 59% had hematological improvement, mainly on the erythroid lineage (Increase of Hemoglobin). Most responses were observed at low doses and between 4 and 8 weeks.
The objectives of this trial (Thal-SMD-20) are to evaluate the efficacy and tolerance of lower doses thalidomide in low risk MDS patients with transfusion-dependant anemia.
- Detailed Description
Thalidomide:
First part of the trial: 82 patients at 200mg/day given at bedtime x 12 weeks, decreased to 100mg/day if grade 1 or 2 side. Stopped temporally for 1 week if grade 3 or 4 side effects. Then reintroduced at the same dose. If side effects again, definitively stopped.
Responses evaluated at 12 weeks according to IWG criteria for the erythroid lineage
At week 12:
* If no Hematological improvement (HI): increased to 300mg/day for 8 weeks and then eventually to 400mg/day for 8 weeks more, if no HI.
* If Hematological improvement (HI): continued at the same dose.
Second part of the trial: 30 patients treated at 50mg/day x 12 weeks. Responses evaluated at 12 weeks according to IWG criteria for the erythroid lineage
At week 12:
* If no Hematological improvement (HI): increased to 100mg/day for 8 weeks and then eventually to 200mg/day for 8 weeks more, if no HI.
* If Hematological improvement (HI): continued at the same dose.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 112
- Patients ≥18 years, with IPSS Low or Int-1 MDS
- Transfusion dependant anemia above 2 packed red blood cells (PRBC)/month
- ECOG index = 0, 1, 2
- No peripheral neurological disease
- MDS patients with IPSS Int-2 or High
- Patients with less than 2 packed red blood cells (PRBC)/month
- Patients with previous history of venous thrombosis
- Patient treated with EPO +/- G-CSF in the 2 months before inclusion in the protocol
- Patient having received intensive chemotherapy in the 3 months before inclusion in the protocol
- Patient having received Thalidomide in a previous protocol
- Patient presenting an iron, B12 vitamin or folic acid uncorrected deficiency
- Patient with peripheral neurological disease
- Patient not being able to subject itself to a regular clinical and biological follow-up
- Pregnant patient or patient in a period of lactation
- Patient refusing to take a contraceptive treatment through out all the study
- Patient receiving drugs able to interfere with the mechanism of action of Thalidomide
- Patient refusing to sign the informed consent.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method Efficacy evaluated at week 12 according to the IWG criterias
- Secondary Outcome Measures
Name Time Method Safety
Trial Locations
- Locations (20)
CHU Albert Michallon
🇫🇷Grenoble, France
CH d'Avignon
🇫🇷Avignon, France
Hopital Avicenne
🇫🇷Bobigny, France
CHU d'Angers
🇫🇷Angers, France
CHU de Nice - Hopital de l'Archet 1
🇫🇷Nice, France
CHU de Brest - Hopital Morvan
🇫🇷Brest, France
CHU Dijon
🇫🇷Dijon, France
CHRU de Lille - Hopital C. Huriez
🇫🇷Lille, France
CHU de Limoges
🇫🇷Limoges, France
Institut Paoli Calmette
🇫🇷Marseille, France
CHU de Nantes
🇫🇷Nantes, France
Hotel Dieu
🇫🇷Paris, France
Hopital Saint Antoine
🇫🇷Paris, France
Hopital Necker
🇫🇷Paris, France
CH Joffre
🇫🇷Perpignan, France
Centre Henry Becquerel
🇫🇷Rouen, France
CHU Nancy-Brabois
🇫🇷Vandoeuvre les Nancy, France
CH de la Cote Basque
🇫🇷Bayonne, France
Hopital Cochin
🇫🇷Paris, France
CHU Purpan
🇫🇷Toulouse, France