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

NCT ID: NCT00455910

Last Updated: 2007-04-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

112 participants

Study Classification

INTERVENTIONAL

Study Start Date

2003-01-31

Study Completion Date

2007-03-31

Brief Summary

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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

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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.

Conditions

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Myelodysplastic Syndromes

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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Thalidomide

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Groupe Francophone des Myelodysplasies

OTHER

Sponsor Role lead

Principal Investigators

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Didier Bouscary, MD, Ph-D

Role: PRINCIPAL_INVESTIGATOR

Groupe Francophone des Myelodysplasies

Locations

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CHU d'Angers

Angers, , France

Site Status

CH d'Avignon

Avignon, , France

Site Status

CH de la Cote Basque

Bayonne, , France

Site Status

Hopital Avicenne

Bobigny, , France

Site Status

CHU de Brest - Hopital Morvan

Brest, , France

Site Status

CHU Dijon

Dijon, , France

Site Status

CHU Albert Michallon

Grenoble, , France

Site Status

CHRU de Lille - Hopital C. Huriez

Lille, , France

Site Status

CHU de Limoges

Limoges, , France

Site Status

Institut Paoli Calmette

Marseille, , France

Site Status

CHU de Nantes

Nantes, , France

Site Status

CHU de Nice - Hopital de l'Archet 1

Nice, , France

Site Status

Hotel Dieu

Paris, , France

Site Status

Hopital Saint Antoine

Paris, , France

Site Status

Hopital Cochin

Paris, , France

Site Status

Hopital Necker

Paris, , France

Site Status

CH Joffre

Perpignan, , France

Site Status

Centre Henry Becquerel

Rouen, , France

Site Status

CHU Purpan

Toulouse, , France

Site Status

CHU Nancy-Brabois

Vandœuvre-lès-Nancy, , France

Site Status

Countries

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France

References

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Bouscary D, Legros L, Tulliez M, Dubois S, Mahe B, Beyne-Rauzy O, Quarre MC, Vassilief D, Varet B, Aouba A, Gardembas M, Giraudier S, Guerci A, Rousselot P, Gaillard F, Moreau A, Rousselet MC, Ifrah N, Fenaux P, Dreyfus F; Groupe Francais des Myelodysplasies (GFM). A non-randomised dose-escalating phase II study of thalidomide for the treatment of patients with low-risk myelodysplastic syndromes: the Thal-SMD-2000 trial of the Groupe Francais des Myelodysplasies. Br J Haematol. 2005 Dec;131(5):609-18. doi: 10.1111/j.1365-2141.2005.05817.x.

Reference Type BACKGROUND
PMID: 16351636 (View on PubMed)

Related Links

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http://www.gfmgroup.org

website of French Group of Myelodysplasia

Other Identifiers

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CCPPRB Cochin 2402-1-1928

Identifier Type: -

Identifier Source: secondary_id

020895

Identifier Type: -

Identifier Source: org_study_id