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A Study of Combined Deferasirox, Vitamin D and Azacytidine in High Risk MDS (GFM-EXVD-AZA)

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2015 by Groupe Francophone des Myelodysplasies
Information provided by (Responsible Party):
Groupe Francophone des Myelodysplasies Identifier:
First received: October 24, 2012
Last updated: October 27, 2015
Last verified: October 2015

Determinate safety and response rate of the association Deferasirox -Vitamine D - Azacitidine in treatment of high risk MDS

Deferasirox Exjade:

The dose of Deferasirox will be assigned according to the ferritin level. Dose escalation is scheduled during the phase I, with 5 additional patients per group.

The maximal tolerated dose of Deferasirox will be required for the phase II of the study.

The first dose will be assigned according to the ferritin level of the patient at time of inclusion:

5 mg/kg/d if the ferritin is >300ng/ml and < 1000ng/ml in Group 1 10 mg/kg/d if the ferritin is ≥1000ng/ml) in Group 2

Group 1 : Ferritin 300 to 1000ng /ml:

  • cohort 1 : 5 mg/kg/d
  • cohort 2 : 10mg/kg/d
  • cohort 3 : 15 mg/kg/d

Group 2 : Ferritin > 1000ng /ml:

  • cohort 1 : 10 mg/kg/d
  • cohort 2 : 15mg/kg/d
  • cohort 3 : 20 mg/kg/d

    5 patients will be treated by cohort. In absence of toxicity (extra-hematological toxicity grade 3 or 4 or hematological grade 4), 5 additional patients will be included in the next cohort.

Deferasirox will be administrated once daily during all the study period. Uvedose will be administrated once weekly during all the study period (100.000 UI P.O).

Azacitidine will be administrated sc at 75 mg/m²/d, during 7 days, J1 to J7 of each cycles(One cycle is 28 days)

During phase I and II, Deferasirox will always be associated with Vitamin D and Azacitidine

Patients will be received 6 cycles of treatment (except if progression, unacceptable toxicity or withdrawn of patients occured) After 3 and 6 cycles, an evaluation will be done to evaluate the efficacy of the treatment.

No dose modification of deferasirox will be done after 3 cycles of treatment except in case of progression). After 6 cycles, patients with CR, PR, marrow CR or HI will be treated with the same dose of Deferasirox until progression .

Condition Intervention Phase
Drug: Deferasirox, Vitamin D and Azacitidine
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase I-II Study of Association of Deferasirox, Vitamin D and Azacytidine as Treatment of High Risk MDS (IPSS Int-2 and High)

Resource links provided by NLM:

Further study details as provided by Groupe Francophone des Myelodysplasies:

Primary Outcome Measures:
  • To determine the maximal tolerated dose(MTD [ Time Frame: 6 month of treatment ]
    patient will be evaluable after at least one cycle. Treatment will be administrated during 6 month and responders will be treated until progression or death

Estimated Enrollment: 50
Study Start Date: February 2013
Estimated Study Completion Date: February 2018
Estimated Primary Completion Date: February 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Group 1

Patients will be included in 2 groups according to the ferritin level at time of inclusion.

Patients with the ferritin level >300ng/ml and < 1000ng/ml, will be included in Group 1.

5 patients in each cohort: Cohort 1: Deferasirox: 5mg/kg/d Cohort 2: Deferasirox: 10mg/kg/d Cohort 3: Deferasirox: 15mg/kg/d

Drug: Deferasirox, Vitamin D and Azacitidine
Other Names:
  • Exjade
  • Uvedose
  • Vidaza
Experimental: Group 2

Patients will be included in 2 groups according to the ferritin level at time of inclusion.

Patients with the ferritin level > 1000ng/ml, will be included in Group 2.

5 patients in each cohort: Cohort 1: Deferasirox: 10mg/kg/d Cohort 2: Deferasirox: 15mg/kg/d Cohort 3: Deferasirox: 20mg/kg/d

Drug: Deferasirox, Vitamin D and Azacitidine
Other Names:
  • Exjade
  • Uvedose
  • Vidaza

Detailed Description:

Deferasirox will be administrated once daily in the morning on an empty stomach, 30 minutes before meal.

Deferasirox will be stopped if the ferritin level is under 100 ng/ml,and could be restarted is the ferritin level increase to 200 ng/ml

Uvedose dose could be adjusted according to the phosphocalcic metabolism parameters and the plasma Vitamin D3 level.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • High risk MDS, according to OMS classification
  • High risk CMML (WBC < 13 G/L)
  • AREBT of the FAB classification with less than 30% of blastes
  • IPSS>=1.5 (int-2 and high risk)
  • Age >=18y
  • Performance status<=2 (ECOG)
  • Bilirubin and transaminase < 1.5 x ULN
  • Normal renal function
  • Patient not eligible for Allogeneic stem cell transplant
  • Male and female patients must use an effective contraceptive method during the study and for a minimum of 3 months after study treatment.
  • Agree the need for the use of a condom if engaged in sexual activity with a pregnant woman or a woman of childbearing potential. during the entire period of treatment, even if disruption of treatment and during 3 months after end of treatment
  • Male patient: Agree not to conceive during treatment and study drug therapy (including doses interruptions) and for 3 months after the end of the study drug therapy
  • Agree not to donate semen during study drug therapy and for one week after end of study drug therapy.
  • Agree to learn about the procedures for preservation of sperm,before starting treatment
  • Patient be able to adhere to the study visit schedule and other protocol requirements

Exclusion Criteria:

  • Active infection or uncontrolled disease
  • Use of cytotoxic chemotherapeutic agents or experimental agents(agents that are not commercially available) for the treatment of MDS within 28 days. In case of used of cytotoxic chemotherapeutic agents or hypomethylating agent a wash out of 3 mont is required.
  • Active Cancer or Cancer within one year before inclusion
  • Previous calcic urinary lithiasis
  • Previous hyperparathyroid primitive disease or uncontrolled
  • Hypercalcemia, hyperphosphoremia, hypervitaminosis D
  • Patient already include in another experimental study
  • Active infection by HIV, hepatite B or C
  • Pregnant or lactating females
  • Patient not able (medical/psychiatric) to understand and sign the written consent
  • Patients with a ferritin level less than 300ng/ml
  • Patient eligible for an Allogeneic stem cell transplant
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01718366

Contact: Felipe Suarez, MD 033144495286
Contact: Fatiha Chermat, CRA 0171207059

Chu Amiens Not yet recruiting
Amiens, France
Contact: Berengere gruson, MD    0322455914      
Principal Investigator: Berengere Gruson, MD         
Centre Hospitalier de La Cote Basque Recruiting
Bayonne, France, 64100
Contact: Anne Banos, MD    033559443832   
Principal Investigator: Anne Banos, MD         
Hôpital Avicenne Recruiting
Bobigny, France, 93009
Contact: Thorsten Braun, MD    33148957051   
Principal Investigator: Thorsten Braun, MD         
Centre Hospitalier de Boulogne sur Mer Not yet recruiting
Boulogne sur Mer, France
Contact: Bachra Chouffi    0321998202      
Principal Investigator: Bachra Chouffi, MD         
CHU Le Mans Recruiting
Le MANS, France
Contact: Kamel Laribi, MD         
Hôpital Saint Vincent de Paul Recruiting
Lille, France, 59020
Contact: Christian Rose, MD    33320874532   
Principal Investigator: Christian Rose, MD         
Institut Paoli Calmettes Active, not recruiting
Marseille, France, 13009
Centre hospitalier de Meaux Not yet recruiting
Meaux, France, 77100
Contact: Loic Fouillard, MD         
Contact: Loic Fouillard, MD    0164653876      
Principal Investigator: Loic Fouillard, MD         
CHU Brabois Active, not recruiting
Nancy, France, 54511
CHU Nantes Active, not recruiting
Nantes, France, 44093
Centre Catherine de Sienne Recruiting
Nantes, France
Contact: Jacques Delaunay, MD         
Principal Investigator: Jacques Delaunay, MD         
Hôpital saint Louis Recruiting
Paris, France, 75010
Contact: Pierre Fenaux, MD    033170207022   
Principal Investigator: Pierre Fenaux, MD         
Hôpital cochin Recruiting
Paris, France, 75679
Contact: François Dreyfus, MD    33158411996   
Principal Investigator: François Dreyfus, MD         
Hôpital Necker Recruiting
Paris, France, 75743
Contact: Olivier Hermine, MD    33144495283   
Principal Investigator: Olivier Hermine, MD         
Sub-Investigator: Felipe Suarez, MD         
Centre Hospitalier Joffre-Perpignan Not yet recruiting
Perpignan, France
Contact: Laurence Sanhes, MD    0468616448      
Principal Investigator: Laurence Sanhes, MD         
IUCT Oncopole Toulouse Not yet recruiting
Toulouse, France
Contact: Odile Beyne-Rauzy    0531156355      
Principal Investigator: Odile Beyne-Rauzy, MD         
Centre Hospitalier Valence Not yet recruiting
Valence, France
Contact: Liu Jixing, MD    0475757566      
Principal Investigator: Jixing Liu, MD         
Sponsors and Collaborators
Groupe Francophone des Myelodysplasies
Principal Investigator: Olivier Hermine, MD Necker Hospital (Paris)
Study Director: Pierre Fenaux, MD Saint Louis Hospital (Paris)
Study Chair: Felipe Suarez, MD Necker Hospital (Paris)
  More Information

Responsible Party: Groupe Francophone des Myelodysplasies Identifier: NCT01718366     History of Changes
Other Study ID Numbers: GFM-EXVD-AZA-2011-005623-41
Study First Received: October 24, 2012
Last Updated: October 27, 2015

Additional relevant MeSH terms:
Vitamin D
Growth Substances
Physiological Effects of Drugs
Bone Density Conservation Agents
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Antimetabolites, Antineoplastic
Antineoplastic Agents
Enzyme Inhibitors processed this record on May 25, 2017