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Active, not recruitingNCT03223961EPO-PRETARUpdated Sep 11, 2026

A Trial Testing Early vs Late Onset of EPO Alfa Treatment in Lower Risk MDS

A Phase 3 interventional study of EPREX in Myelodysplastic Syndromes, sponsored by Groupe Francophone des Myelodysplasies. Active, not recruiting at 39 sites in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-11.

Sponsored by Groupe Francophone des Myelodysplasies · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
124
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
01

Study summary

This is an open-label, randomized, multicenter, phase III study

Patients with baseline Hb comprised between 9 and 10.5g/dl will be randomized to receive EPO Alfa 60000 UI/week for at least 12 weeks:

  • Either at diagnosis Or
  • at the Hb threshold chosen for RBC transfusions (must be \< 9g/dl)
Read the detailed description

in this trial we will compare the early introduction of EPO alfa to the delayed introduction in lower risk MDS with non RBC transfusion dependent anemia.

At enrollment patients will be randomised in the 2 arms (early and delayed start of EPO alfa).

Treatment Regimen Epoetin alfa 60000 UI/week for at least 12 weeks

  1. Early onset arm: early onset of EPO ALFA 60000 IU/week , at patient inclusion
  2. Delayed onset arm: late introduction of EPO ALFA 60000 IU/week, whenever the patient reaches the level chosen RBC transfusions (based on age, comorbidities, anticipated tolerance of anemia).
02

Conditions studied

  • Myelodysplastic Syndromes

Keywords

  • myelodysplastic syndrome
  • erythropoetin
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Age ≥ 18 years
  2. MDS according to WHO 2016 criteria, with low or int 1 classical IPSS
  3. Non-RBC transfusion dependent anemia
  4. Hb level between 9 and 10.5g/dl (at the center's lab)
  5. Hb level should be at least 1g/dl higher than the Hb threshold chosen to start RBC transfusions based on age, comorbidities and predicted clinical tolerance of anemia (this transfusion threshold should be chosen between 8 and 9g/dl)
  6. Serum EPO level \<500U/l
  7. No other cause of anemia (including iron deficiency, vitamin B12 or B9 deficiency, hemolysis, hypothyroidism….)
  8. Performance status \<=2

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
  4. Transfusion threshold (based on age , comorbidities…) >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
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
124 participants (estimated)

Study arms

  • Experimental
    Early onset arm

    Intervention: early onset of Eprex60000 IU/week , at patient inclusion

    Drug: EPREX

  • Experimental
    Delayed onset arm

    Intervention: late introduction of Eprex60000 IU/week, whenever the patient reaches the level chosen RBC transfusions (based on age, comorbidities, anticipated tolerance of anemia).

    Drug: EPREX

Interventions

  • DrugEPREX

    60 000 U/week for at least 12 weeks

    Also known as: Epoetin alfa

05

What researchers measure

Primary outcomes

  1. Time to RBC transfusion dependence in non RBC transfusion dependent lower risk MDS patients with anemia with early (at inclusion of the patient) versus delayed onset,( at the threshold chosen for RBC transfusion) of EPO ALFA

    RBC transfusion dependence will be defined by requirement of at least transfusions of 2 PRBC within an interval of less than 8 weeks, given for Hb \<8g/dl or \<9g/dl according to comorbidities and in the absence of other cause of anemia (bleeding, surgery…), taking into account only transfusions given at least 12 weeks after onset of treatment with EPO ALFA.

    Time frame: 12 weeks

Secondary outcomes

  1. Erythroid response (according to IWG 2006 criteria)

    Erythroid response (according to IWG 2006 criteria) after 12 weeks of EPO ALFA treatment

    Time frame: 12 weeks

  2. response duration to EPO ALFA

    response duration to EPO ALFA measured from the date of enrollment until failure

    Time frame: 4 years

  3. Overall survival

    Overall survival measured from the date of enrollment to death or the date of last contact

    Time frame: 4 years

06

Study locations

39 sites
  • Chu Amiens
    Amiens, France
  • CH Angers
    Angers, 49 000, France
  • CH Avignon
    Avignon, 84000, France
  • Centre Hospitalier de La Cote Basque
    Bayonne, 64100, France
  • Hopital Nord Franche-Comté
    Belfort, 90015, France
  • CHU de Besançon
    Besançon, 25030, France
  • Hopital Avicenne
    Bobigny, 9300, France
  • Hôpital Morvan
    Brest, 29609, France
  • CHU de Caen
    Caen, 14033, France
  • CH de Sevigné
    Cesson, 35510, France
  • CH de Cholet
    Cholet, 49325, France
  • CHU Estaing
    Clermont-Ferrand, 63000, France
  • CHSF Gilles de Corbeil
    Corbeil-Essonnes, 91100, France
  • Hôpital Henri-Mondor
    Créteil, 94010, France
  • CHU de Grenoble
    Grenoble, 38043, France
  • Centre Hospitalier du Mans
    Le Mans, 72000, France
  • Clinique Victor Hugo
    Le Mans, 72000, France
  • Hopital Saint-Vincent de Paul
    Lille, 59160, France
  • CHRU Limoges
    Limoges, 87042, France
  • Centre Hospitalier Lyon Sud
    Lyon, 69495, France
  • IPC
    Marseille, 13273, France
  • Centre Hospitalier du Mont de Marsan
    Mont-de-Marsan, 40000, France
  • CHU de Nantes
    Nantes, 44093, France
  • CHU de Nice
    Nice, France
  • Hopital St Louis T4
    Paris, 75475, France
  • Centre Hospitalier Joffre-Perpignan
    Perpignan, 66046, France
  • Sophie Dimicoli-Salazar
    Pessac, 33604, France
  • CH de Périgueux
    Périgueux, 24019, France
  • CHU de Poitiers
    Poitiers, 86021, France
  • CHR d'Annecy
    Pringy, 74374, France
  • CHRU de Reims
    Reims, 51092, France
  • CHU Pontchaillou
    Rennes, 35033, France
  • Centre Henri Becquerel
    Rouen, 76038, France
  • CH Saint Nazaire
    Saint-Nazaire, 44600, France
  • Centre Hospitalier Universitaire de STRASBOURG
    Strasbourg, 67098, France
  • IUCT Oncopole
    Toulouse, 31059, France
  • CH de Troyes
    Troyes, 10003, France
  • CH Valence
    Valence, 26953, France
  • CHU Brabois
    Vandœuvre-lès-Nancy, 54511, France
07

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT03223961
Lead sponsor
Groupe Francophone des Myelodysplasies
Responsible party
Sponsor
First posted
Jul 21, 2017
Start date
Mar 26, 2018
Primary completion
Sep 21, 2023
Completion
Oct 1, 2028 (estimated)
Last update
Sep 11, 2026

Study contacts

Sophie Park, Prof
principal investigator · University Hospital, Grenoble

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is active, not recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.

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