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

Primary Purpose

Myelodysplasia

Status

Recruiting

Phase

Phase 2

Locations

France

Study Type

Interventional

Intervention

Deferasirox

About this trial

This is an interventional treatment trial for Myelodysplasia focused on measuring efficacy, safety

Eligibility Criteria

18 Years - 100 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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

Exclusion Criteria:

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
5q- deletion to karyotype
Patient eligible for allograft
Patient participating in another interventional clinical study or exclusion period from another study
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.

Sites / Locations

CHU de GRENOBLE ALPESRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Deferasirox

Arm Description

efficacy of 3.5mg/kg/day

Outcomes

Primary Outcome Measures

Percentage of patients without transfusion-dependence at 12 months.

Secondary Outcome Measures

Full Information

NCT ID

NCT03387475

First Posted

December 22, 2017

Last Updated

May 19, 2022

Sponsor

University Hospital, Grenoble

Collaborators

Novartis

1. Study Identification

Unique Protocol Identification Number

NCT03387475

Brief Title

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

Acronym

LODEFI

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)

Study Type

Interventional

2. Study Status

Record Verification Date

September 2021

Overall Recruitment Status

Recruiting

Study Start Date

February 20, 2018 (Actual)

Primary Completion Date

December 31, 2024 (Anticipated)

Study Completion Date

December 31, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

University Hospital, Grenoble

Collaborators

Novartis

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Myelodysplasia

Keywords

efficacy, safety

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

39 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Deferasirox

Arm Type

Experimental

Arm Description

efficacy of 3.5mg/kg/day

Intervention Type

Drug

Intervention Name(s)

Deferasirox

Other Intervention Name(s)

exjade

Intervention Description

iron chelation

Primary Outcome Measure Information:

Title

Percentage of patients without transfusion-dependence at 12 months.

Description

Percentage of patients without transfusion-dependence at 12 months.

Time Frame

12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

100 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Exclusion Criteria: 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 5q- deletion to karyotype Patient eligible for allograft Patient participating in another interventional clinical study or exclusion period from another study 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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Sophie Park

Phone

0476762777

Spark@chu-grenoble.fr

First Name & Middle Initial & Last Name or Official Title & Degree

Valérie Rolland

Phone

0476765096

VRolland-neyret@chu-grenoble.fr

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Sophie Park

Organizational Affiliation

University Hospital, Grenoble

Official's Role

Principal Investigator

Facility Information:

Facility Name

CHU de GRENOBLE ALPES

City

Grenoble

ZIP/Postal Code

38043

Country

France

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Sophie PARK

Phone

04 76 76 27 77

SPark@chu-grenoble.fr

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

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Learn more about this trial

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

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