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

Myelodysplastic Syndromes Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00455910
• Other study ID #: 020895
• Secondary ID: CCPPRB Cochin 24
• Status: Completed
• Phase: Phase 2
• First received: April 3, 2007
• Last updated: April 3, 2007
• Start date: January 2003
• Est. completion date: March 2007

Study information

• Verified date: April 2007
• Source: Groupe Francophone des Myelodysplasies
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 112
• Est. completion date: March 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Myelodysplastic Syndromes
• Preleukemia
• Syndrome

Intervention

Drug:
• Thalidomide

Locations

Country	Name	City	State
France	CHU d'Angers	Angers
France	CH d'Avignon	Avignon
France	CH de la Cote Basque	Bayonne
France	Hopital Avicenne	Bobigny
France	CHU de Brest - Hopital Morvan	Brest
France	CHU Dijon	Dijon
France	CHU Albert Michallon	Grenoble
France	CHRU de Lille - Hopital C. Huriez	Lille
France	CHU de Limoges	Limoges
France	Institut Paoli Calmette	Marseille
France	CHU de Nantes	Nantes
France	CHU de Nice - Hopital de l'Archet 1	Nice
France	Hopital Cochin	Paris
France	Hopital Necker	Paris
France	Hopital Saint Antoine	Paris
France	Hotel Dieu	Paris
France	CH Joffre	Perpignan
France	Centre Henry Becquerel	Rouen
France	CHU Purpan	Toulouse
France	CHU Nancy-Brabois	Vandoeuvre les Nancy

Sponsors (1)

Lead Sponsor	Collaborator
Groupe Francophone des Myelodysplasies

Country where clinical trial is conducted

France, 

References & Publications (1)

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 Français des Myélodysplasies (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 Français des Myélodysplasies. Br J Haematol. 2005 Dec;131(5):609-18. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Efficacy evaluated at week 12 according to the IWG criterias
Secondary	Safety

External Links

•   website of French Group of Myelodysplasia