Thalidomide at Low Dose for the Treatment of Patient With Myelodysplastic Syndromes - THAL-SMD-200

Sponsor

Groupe Francophone des Myelodysplasies (Other)

Overall Status

Completed

CT.gov ID

NCT00455910

Collaborator

(none)

112

Enrollment

Locations

49.9

Duration (Months)

5.6

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Myelodysplastic Syndromes	Drug: Thalidomide	Phase 2

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.

Study Design

Study Type:

Interventional

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Thalidomide for the Treatment of Cytopenias of Patients With Low Risk Myelodysplastic Syndromes

Study Start Date :

Jan 1, 2003

Actual Study Completion Date :

Mar 1, 2007

Outcome Measures

Primary Outcome Measures

Efficacy evaluated at week 12 according to the IWG criterias []

Secondary Outcome Measures

Safety []

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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

Exclusion Criteria:

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.

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	CHU d'Angers	Angers	France	49 033
2	CH d'Avignon	Avignon	France	84 000
3	CH de la Cote Basque	Bayonne	France	64 100
4	Hopital Avicenne	Bobigny	France	93009
5	CHU de Brest - Hopital Morvan	Brest	France	29 609
6	CHU Dijon	Dijon	France	21 000
7	CHU Albert Michallon	Grenoble	France	38 043
8	CHRU de Lille - Hopital C. Huriez	Lille	France	59037
9	CHU de Limoges	Limoges	France	87 042
10	Institut Paoli Calmette	Marseille	France	13009
11	CHU de Nantes	Nantes	France	44 093
12	CHU de Nice - Hopital de l'Archet 1	Nice	France	06 202
13	Hotel Dieu	Paris	France	75 004
14	Hopital Saint Antoine	Paris	France	75 012
15	Hopital Cochin	Paris	France	75014
16	Hopital Necker	Paris	France	75015
17	CH Joffre	Perpignan	France	66 046
18	Centre Henry Becquerel	Rouen	France	76 038
19	CHU Purpan	Toulouse	France	31059
20	CHU Nancy-Brabois	Vandoeuvre les Nancy	France	54511