A RANDOMIZED TRIAL TESTING EARLY VERSUS LATE ONSET OF EPO ALFA TREATMENT IN LOWER RISK MDS WITH NON RBC TRANSFUSION DEPENDENT ANEMIA AND WITHOUT DEL 5Q

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-000812-41-FR

Enrollment

124

Registered

2017-10-23

Start date

2017-12-29

Completion date

Unknown

Last updated

2024-09-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Adult subject (18 years of age older) with low or intermediate-1 IPSS risk MDS with transfusions independent anemia MedDRA version: 20.0 Level: HLT Classification code 10028536 Term: Myelodysplastic syndromes System Organ Class: 100000004851

Interventions

Trade Name: EPREX Product Name: Eprex Pharmaceutical Form: Injection INN or Proposed INN: EPOETIN ALFA CAS Number: 113427-24-0 Current Sponsor code: eprex Other descriptive name: Eprex Concentration u

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Age = 18 years at the time of signing the informed consent form 2. Must understand and voluntarily sign the informed consent form 3. Diagnosis of MDS according to WHO 2016 criteria, and low or int 1 classical IPSS, including CMML with WBC =65 years) yes F.1.3.1 Number of subjects for this age range 124

Exclusion criteria

Exclusion criteria: 1. Higher risk MDS (IPSS intermediate-2 or high) 2. Del 5q 3. Baseline Hemoglobin level > 10.5 g/dl or 9g/dl 5. Transfusion threshold less than 1 g/dl below baseline Hb level 6. RBC transfusion dependence. Patients may have received only one transfusion series for MDS prior to inclusion 7. CMML , if >10 % BM blasts or WBC>13.000/mm3 8. Uncontrolled hypertension 9. Uncontrolled cardiovascular disease including angina pectoris or cardiac failure 10. Renal failure: Creatinine clearance<40ml/min (using MDRD formula) 11. Pregnancy (positive bettaHCG) or nursing

Design outcomes

Primary

Measure	Time frame
Main Objective: Randomly compare, in non RBC transfusion dependent lower risk MDS with anemia, the time to RBC transfusion dependence in patients with early onset of EPO ALFA (at inclusion) and patients with delayed onset of EPO ALFA ( at the threshold chosen for RBC transfusions) ;Secondary Objective: Compare in those 2 randomized groups: 1. erythroid response (according to IWG 2006 criteria) after 12 weeks of EPO ALFA treatment 2. response duration to EPO ALFA 3. progression to higher risk MDS and /or AML 4. the incidence of cardiovascular events 5. QoL (assessed based on usual scales) 6.Overall survival. 7.We will also analyse biological correlates and compare in those 2 groups the ex vivo effect of EPO ALFA on erythropoiesis, in particular on the disease clonal architecture by repeated next generation sequencing (NGS) analysis of somatic mutations. One hypothesis is that by beginning EPO ALFA early in the disease course, no or less mutated erythropoiesis may be amplified by EPO ALFA at the expense of more mutated clones, thereby potentially delaying the disease course ;Primary end point(s): Comparison of the time to RBC transfusion dependence between patients with early onset of EPO ALFA (at inclusion ) and patients with delayed onset of EPO ALFA (at the threshold chosen for RBC transfusion) ;Timepoint(s) of evaluation of this end point: through the study	—

Measure

Time frame

Main Objective: Randomly compare, in non RBC transfusion dependent lower risk MDS with anemia, the time to RBC transfusion dependence in patients with early onset of EPO ALFA (at inclusion) and patients with delayed onset of EPO ALFA ( at the threshold chosen for RBC transfusions) ;Secondary Objective: Compare in those 2 randomized groups: 1. erythroid response (according to IWG 2006 criteria) after 12 weeks of EPO ALFA treatment 2. response duration to EPO ALFA 3. progression to higher risk MDS and /or AML 4. the incidence of cardiovascular events 5. QoL (assessed based on usual scales) 6.Overall survival. 7.We will also analyse biological correlates and compare in those 2 groups the ex vivo effect of EPO ALFA on erythropoiesis, in particular on the disease clonal architecture by repeated next generation sequencing (NGS) analysis of somatic mutations. One hypothesis is that by beginning EPO ALFA early in the disease course, no or less mutated erythropoiesis may be amplified by EPO ALFA at the expense of more mutated clones, thereby potentially delaying the disease course ;Primary end point(s): Comparison of the time to RBC transfusion dependence between patients with early onset of EPO ALFA (at inclusion ) and patients with delayed onset of EPO ALFA (at the threshold chosen for RBC transfusion) ;Timepoint(s) of evaluation of this end point: through the study

—

Secondary

Measure	Time frame
Secondary end point(s): 1. IWG 2006 erythroid response and duration 2. QoL, 3. OS 4. molecular biology corrélations ;Timepoint(s) of evaluation of this end point: through the study	—

Countries

France

Contacts

Public ContactFatiha Chermat

GFM-Groupe Francophone des Myélodysplasies

fatiha.chermat-ext@aphp.fr33171207059

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026