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

NCT03387475 | Completed Phase 2 DMC

• 1 other identifier: 38RC17.064
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Myelodysplasia

Keywords

efficacy; safety

Outcome Measures

Primary Outcomes (1)

• Percentage of patients without transfusion-dependence at 12 months.

  Percentage of patients without transfusion-dependence at 12 months.

  12 months

Study Arms (1)

Deferasirox

EXPERIMENTAL

efficacy of 3.5mg/kg/day

Drug: Deferasirox

Interventions

Deferasirox DRUG

iron chelation

Also known as: exjade

Deferasirox

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• low risk (IPSS-R very low, low and intermediate)
• 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)
• age ≥ 18 years
• ECOG ≤2
• informed consent dated and signed
• affiliated to a social security scheme
• 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

You may not qualify if:

• Transfusion dependent patient (≥) 2 red blood cells (RBP) per 2-month period evaluated over 4 months between M-4 and M0
• 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
• Ferritin \<200 ng / ml
• Iron overload: ferritin\> 1000 ng / ml
• Creatinine clearance according to MDRD ≤60 ml / min
• q- deletion to karyotype
• Patient eligible for allograft
• 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.
• 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.

Sponsors & Collaborators

• University Hospital, Grenoble: lead
• Novartis: collaborator

Study Sites (1)

CHU de GRENOBLE ALPES

Grenoble, 38043, France

Location

MeSH Terms

Conditions

Anemia, Refractory, with Excess of Blasts

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Anemia, Refractory; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Sophie Park

  University Hospital, Grenoble

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2017
• First Posted: January 2, 2018
• Study Start: February 20, 2018
• Primary Completion: October 1, 2024
• Study Completion: October 1, 2024
• Last Updated: December 16, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will share

Locations

FR (1)