Clinical Trials /

Evaluating Low-dose Deferasirox (DFX) in Patients With Low-risk MDS Resistant or Relapsing After ESA Agents

NCT03387475

Description:

Patients with low-risk MDS verifying the eligibility criteria may be included in the study.

Related Conditions:
  • Chronic Myelomonocytic Leukemia-1
  • Myelodysplastic Syndromes
  • Refractory Anemia
  • Refractory anemia with ring sideroblasts
Recruiting Status:

Recruiting

Phase:

Phase 2

Trial Eligibility

Document

Title

  • Brief Title: Evaluating Low-dose Deferasirox (DFX) in Patients With Low-risk MDS Resistant or Relapsing After ESA Agents
  • Official Title: Phase II Trial Evaluating Low-dose Deferasirox (DFX) in Patients With Low-risk (MDS) Myelodysplastic Syndrome Resistant or Relapsing After ESA Agents (LODEFI)

Clinical Trial IDs

  • ORG STUDY ID: 38RC17.064
  • NCT ID: NCT03387475

Conditions

  • Myelodysplasia

Interventions

DrugSynonymsArms
DeferasiroxexjadeDeferasirox

Purpose

Patients with low-risk MDS verifying the eligibility criteria may be included in the study.

Detailed Description

      Patients will receive the number of DFX tablets (Exjade, cp film-coated, 90mg or 360mg), in
      relation to their body weight to be closer to 3.5mg / kg / d for 12 months.

      At one month and six months of treatment, the residual plasma levels of DFX will be measured
      in patients (sent to the Bordeaux laboratory of Biochemistry, Pr Molimard) to allow the
      dosage to be adjusted (plasma objective of 3 μM).

      At inclusion (J1) and at 6 months of treatment, a dosage of NTBI and hepcidine will be
      performed.

      If patients become transfusion-dependent (≥) 2 packed red cells (RBP) per 2-month period
      evaluated over 6 months, they will stop low-dose DFX and go out of protocol to receive DFX at
      20mg / kg (according to EMEA authorization) for iron chelation.
    

Trial Arms

NameTypeDescriptionInterventions
DeferasiroxExperimentalefficacy of 3.5mg/kg/day
  • Deferasirox

Eligibility Criteria

        Inclusion Criteria:

          1. Patients with MDS according to WHO 2008 criteria (refractory anemia with multilineage
             dysplasia, RA (refractory anemia), refractory anemia with ringed sideroblasts (RARS)
             including CMML-1 (chronic myelomonocytic leukemia) type 1 with <10% blasts)

          2. low risk (IPSS-R very low, low and intermediate)

          3. in primary or secondary failure after erythropoiesis stimulating agents (ESAs),
             (either epoetins (≥60,000 units / week), or darbepoetin (≥250 μg / week), administered
             for at least 12 weeks, as defined by the IWG criteria 2006 (no erythroid response at
             12 weeks, or more than 15g / l decrease in Hb after response to ESAs) PS: Patients
             with low transfusion of less than (<) 4RBP assessed over 4 months (RBP administered
             for patients with Hb ≤ 9g / dl) will be accepted)

          4. age ≥ 18 years

          5. ECOG ≤2

          6. informed consent dated and signed

          7. affiliated to a social security scheme

          8. Women and men of childbearing potential must have effective contraception throughout
             the duration of the study and up to 4 days after the last administration of
             deferasirox

        Exclusion Criteria:

          1. Transfusion dependent patient (≥) 2 red blood cells (RBP) per 2-month period evaluated
             over 4 months between M-4 and M0

          2. Patients with high-risk MDS (based on IPSS-R) and patients with other hematologic and
             non-haematological malignancies who should not benefit from chelation therapy due to
             rapid progression of their disease

          3. Ferritin <200 ng / ml

          4. Iron overload: ferritin> 1000 ng / ml

          5. Creatinine clearance according to MDRD ≤60 ml / min

          6. 5q- deletion to karyotype

          7. Patient eligible for allograft

          8. Patient participating in another interventional clinical study or exclusion period
             from another study

          9. History of cancer treated or untreated for less than 5 years, whether or not there are
             signs of relapse or metastases, with the exception of basocellular cancers.

         10. Persons referred to in Articles L1121-5 to L1121-8 of the CSP: pregnant woman,
             parturient, mother who is breastfeeding, person deprived of liberty by judicial or
             administrative decision, person subject to a legal protection measure, can not not be
             included in clinical trials.
      
Maximum Eligible Age:100 Years
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Percentage of patients without transfusion-dependence at 12 months.
Time Frame:12 months
Safety Issue:
Description:Percentage of patients without transfusion-dependence at 12 months.

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:University Hospital, Grenoble

Trial Keywords

  • efficacy
  • safety

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