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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03387475
Recruitment Status : Recruiting
First Posted : January 2, 2018
Last Update Posted : May 7, 2018
Sponsor:
Collaborator:
Novartis
Information provided by (Responsible Party):
University Hospital, Grenoble

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

Condition or disease Intervention/treatment Phase
Myelodysplasia Drug: Deferasirox Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 39 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : February 20, 2018
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : June 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Deferasirox

Arm Intervention/treatment
Experimental: Deferasirox
efficacy of 3.5mg/kg/day
Drug: Deferasirox
iron chelation
Other Name: exjade




Primary Outcome Measures :
  1. Percentage of patients without transfusion-dependence at 12 months. [ Time Frame: 12 months ]
    Percentage of patients without transfusion-dependence at 12 months.



Information from the National Library of Medicine

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, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 100 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03387475


Contacts
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Contact: Sophie Park 0476762777 Spark@chu-grenoble.fr
Contact: Valérie Rolland 0476765096 VRolland-neyret@chu-grenoble.fr

Locations
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France
CHU de GRENOBLE ALPES Recruiting
Grenoble, France, 38043
Contact: Sophie PARK    04 76 76 27 77    SPark@chu-grenoble.fr   
Sponsors and Collaborators
University Hospital, Grenoble
Novartis
Investigators
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Principal Investigator: Sophie Park University Hospital, Grenoble

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Responsible Party: University Hospital, Grenoble
ClinicalTrials.gov Identifier: NCT03387475    
Other Study ID Numbers: 38RC17.064
First Posted: January 2, 2018    Key Record Dates
Last Update Posted: May 7, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: e crf

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by University Hospital, Grenoble:
efficacy
safety
Additional relevant MeSH terms:
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Preleukemia
Myelodysplastic Syndromes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action