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

• Status: Recruiting
• Trial end date: 2022-02-19
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University Hospital, Grenoble
• Collaborator: Novartis
• Treatments: Deferasirox

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.