EPO-4-Rhesus Study

NCT ID: NCT03104426

Last Updated: 2019-10-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2/PHASE3

Total Enrollment

42 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-10-31

Study Completion Date

2020-08-31

Brief Summary

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Up to 80% of infants with hemolytic disease due to maternal alloimmunization, treated with IUT, require at least one top-up transfusion for late anemia during the first 3 months of life. Erythropoietin deficiency is also considered as a possible contributing factor to late anemia and therefore we will assess the role of EPO (darbepoetin alfa) in the treatment of these infants.

Detailed Description

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The mainstay of antenatal treatment of fetal anemia due to red cell alloimmunization is (serial) IUT. The mainstay of postnatal treatment in HDN is (1) intensive phototherapy and exchange transfusion to treat hyperbilirubinemia and prevent kernicterus, and (2) top-up transfusions to treat anemia. Up to 80% of infants with HDN treated with IUT require at least one top-up transfusion for late anemia during the first 3 months of life.

Several risk factors for late anemia have been reported, including serial IUT (due to bone marrow suppression), severity of HDN, reduced use of exchange transfusions during the neonatal period and reduced survival of transfused red blood cells. Finally, erythropoietin deficiency is also considered as a possible contributing factor to late anemia.

EPO has been increasingly used in neonates to prevent or reduce neonatal anemia without short or long-term adverse effects. Several small studies and casuistic reports suggest that neonates with HDN may benefit from treatment with EPO to reduce the risk of delayed anemia and subsequent top-up transfusions. However, other authors found that EPO may be less effective than expected. Due to the lack of evidence, routine use of EPO is currently not recommended. To determine a role for EPO in this group of patients, a well-designed randomized controlled clinical trial of sufficient sample size is required. Potentially, EPO stabilizes the hemoglobin levels of these infants and thus prevents top-up transfusions and extra admissions, creating a more stable and natural postnatal course for patients with HDN.

Conditions

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Erythroblastosis, Fetal Erythroblastosis Fetalis, Rh Disease Erythroblastosis Fetalis Due to RH Antibodies Erythroblastosis Fetalis Due to Isoimmunization

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

RCT with unblinded treatment allocation 1:1 ratio. Either treatment with darbepoetin alfa or "standard care". No placebo.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Darbepoetin alfa group

Group treated with darbepoetin alfa (Aranesp) 10microg/kg once a week for a period of 8 weeks.

Group Type ACTIVE_COMPARATOR

Darbepoetin Alfa

Intervention Type DRUG

Darbepoetin alfa dosage 10microg/kg once a week for 8 weeks

Control group

"Standard care" which involves close monitoring of hemoglobin levels and if necessary, top-up red cell transfusion.

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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Darbepoetin Alfa

Darbepoetin alfa dosage 10microg/kg once a week for 8 weeks

Intervention Type DRUG

Other Intervention Names

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Aranesp

Eligibility Criteria

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Inclusion Criteria

* all (near)-term neonates (gestational age ≥ 35 weeks) admitted to the Leiden University Medical Center (LUMC) with HDFN, treated with IUT.

Exclusion Criteria

* none.
Maximum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Leiden University Medical Center

OTHER

Sponsor Role collaborator

Sanquin-LUMC J.J van Rood Center for Clinical Transfusion Research

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Masja de Haas, MD PhD

Role: PRINCIPAL_INVESTIGATOR

Sanquin Research

Locations

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Leiden University Medical Center

Leiden, , Netherlands

Site Status RECRUITING

Countries

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Netherlands

Central Contacts

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Isabelle MC Ree, MD

Role: CONTACT

+31715262814

Enrico Lopriore, MD PhD

Role: CONTACT

+31715262965

Facility Contacts

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Enrico Lopriore, MD Phd

Role: primary

References

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Ree IMC, de Haas M, van Geloven N, Juul SE, de Winter D, Verweij EJT, Oepkes D, van der Bom JG, Lopriore E. Darbepoetin alfa to reduce transfusion episodes in infants with haemolytic disease of the fetus and newborn who are treated with intrauterine transfusions in the Netherlands: an open-label, single-centre, phase 2, randomised, controlled trial. Lancet Haematol. 2023 Dec;10(12):e976-e984. doi: 10.1016/S2352-3026(23)00285-5.

Reference Type DERIVED
PMID: 38030319 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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P17.102

Identifier Type: -

Identifier Source: org_study_id

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