Myelodysplastic SyndromesPhase 2NCT00455910
Thalidomide at Low Dose for the Treatment of Patient With Myelodysplastic Syndromes - THAL-SMD-200
A Phase 2 study testing Thalidomide in people with Myelodysplastic Syndromes, run by Groupe Francophone des Myelodysplasies at 20 sites in France.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Thalidomide
- How many people
- 112 participants
- Where
- 20 sitesFrance
- Study dates
- Jan 2003to Mar 2007
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 13Show fewer
- 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.
Trial locations and contacts
20 sites in 1 country.
20 sites in 1 country
France20 sites
CHU d'Angers
Angers, 49 033
CH d'Avignon
Avignon, 84 000
CH de la Cote Basque
Bayonne, 64 100
Hopital Avicenne
Bobigny, 93009
CHU de Brest - Hopital Morvan
Brest, 29 609
CHU Dijon
Dijon, 21 000
CHU Albert Michallon
Grenoble, 38 043
CHRU de Lille - Hopital C. Huriez
Lille, 59037
CHU de Limoges
Limoges, 87 042
Institut Paoli Calmette
Marseille, 13009
CHU de Nantes
Nantes, 44 093
CHU de Nice - Hopital de l'Archet 1
Nice, 06 202
Hotel Dieu
Paris, 75 004
Hopital Saint Antoine
Paris, 75 012
Hopital Cochin
Paris, 75014
Hopital Necker
Paris, 75015
CH Joffre
Perpignan, 66 046
Centre Henry Becquerel
Rouen, 76 038
CHU Purpan
Toulouse, 31059
CHU Nancy-Brabois
Vandœuvre-lès-Nancy, 54511
About this study
Official title: Thalidomide for the Treatment of Cytopenias of Patients With Low Risk Myelodysplastic Syndromes
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.
Read the 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.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Thalidomide
- Keywords
- Low risk myelodysplastic syndromes, MDS, Bone marrow diseases, Thalidomide, Cytopenias, Anemia
- Medical subject headings
- Myelodysplastic Syndromes, Bone Marrow Diseases, Cytopenia, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ThalidomideDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy evaluated at week 12 according to the IWG criterias
Secondary outcomes (1)
Safety
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Apr 2007 (estimated)
- Study startJan 2003
- Study completionMar 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 112 participants
Sponsor and investigators
- Lead sponsor
- Groupe Francophone des Myelodysplasies
- Sponsor type
- University, hospital or other
- Investigators
- Didier Bouscary, MD, Ph-D, Principal investigator, Groupe Francophone des Myelodysplasies
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00455910
- Sponsor's protocol ID
- 020895
- Other IDs
- CCPPRB Cochin 2402-1-1928
- Status last verified
- Apr 2007
- First submitted
- 3 Apr 2007
- Last updated
- 4 Apr 2007
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 16351636
Study at a glance
From the registry record, last updated by the sponsor on 4 Apr 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Thalidomide for the Treatment of Cytopenias of Patients With Low Risk Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT00455910
- Acronym / sponsor ID
- 020895
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Low risk myelodysplastic syndromes, MDS, Bone marrow diseases, Thalidomide, Cytopenias, Anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 112 participants
- Lead sponsor
- Groupe Francophone des MyelodysplasiesUniversity, hospital or other
- Study start
- Jan 2003
- Study completion
- Mar 2007 (actual)
- First posted
- 4 Apr 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Thalidomide | Drug | – |
Primary outcome
Efficacy evaluated at week 12 according to the IWG criterias
1 secondary outcome
Safety
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Apr 2007.
- Background: PubMed 16351636Bouscary 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.
- website of French Group of MyelodysplasiaLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Didier Bouscary, MD, Ph-D principal investigator
Groupe Francophone des Myelodysplasies
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Thalidomide in people with Myelodysplastic Syndromes, run by Groupe Francophone des Myelodysplasies at 20 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Apr 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy evaluated at week 12 according to the IWG criterias. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00455910, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Apr 2007.