Darbepoetin for Preterm Infants

(Darbe Trial)

Recruiting at 15 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: NICHD Neonatal Research Network

Must not be taking: Epo, Darbe

Disqualifiers: Congenital anomalies, Seizures, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 22

Duration: 9 weeks

650 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether participants must stop taking their current medications.

What data supports the effectiveness of the drug Darbepoetin for preterm infants?

Research shows that Darbepoetin, a drug similar to erythropoietin, has been effective in reducing the need for blood transfusions and may help protect the brain in preterm infants, potentially improving their development. ^{1 2 3 4 5}

Is Darbepoetin safe for use in preterm infants?

Research shows that Darbepoetin has been studied for safety in preterm infants, with trials indicating it is generally safe when used for conditions like anemia and brain protection. It has been used to reduce the need for blood transfusions and may help protect the brain, with no major safety concerns reported in these studies. ^{1 2 3 4 6}

How is the drug Darbepoetin unique for preterm infants?

Darbepoetin is unique for preterm infants because it is a long-acting erythropoiesis-stimulating agent (ESA) that not only helps reduce the need for blood transfusions by promoting red blood cell production but also shows promise in protecting the brain and improving neurodevelopmental outcomes. ^{1 2 3 4 7}

What is the purpose of this trial?

Study Hypothesis: Preterm infants administered weekly Darbe during the neonatal period will have improved neurocognitive outcome at 22-26 months compared to placebo

Research Team

Robin Ohls, MD

Principal Investigator

University of Utah

Eligibility Criteria

This trial is for preterm infants born between 23 and nearly 29 weeks of gestation, who are less than one day old. It's not for babies with genetic or heart defects, brain anomalies, high blood count, seizures, clotting disorders, very high blood pressure without medication support, or if they're already getting similar treatments.

Inclusion Criteria

23 0/7-28 6/7 weeks gestation

My baby is less than 24 hours old.

My baby was born prematurely.

Exclusion Criteria

I have a bleeding or blood disorder.

Hematocrit > 60%

Congenital thrombotic disease

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Preterm infants receive weekly Darbepoetin or placebo during the neonatal period

9 weeks

Weekly visits for dosing

Follow-up

Participants are monitored for safety and effectiveness after treatment

22-26 months

Visits at 22-26 months corrected age

Extended follow-up

Subjects will be seen for follow-up at 4-5 years and 6-7 years corrected age to assess long-term outcomes

Long-term

Treatment Details

Interventions

• Darbepoetin
• Placebo

Trial Overview The study tests whether giving Darbepoetin (Darbe) to premature babies weekly can improve their brain development and cognitive skills by the time they're about two years old. They'll compare results from babies given Darbe against those given a placebo.

Participant Groups

Group I: Darbepoetin Experimental Treatment 1 Intervention

Darbepoetin 10 micrograms/kg/once every week (IV or SC)

Group II: Placebo Placebo Group 1 Intervention

Equal volume normal saline for IV administration, or sham dosing

Who Is Running the Clinical Trial?

NICHD Neonatal Research Network

Lead Sponsor

Trials

62

Recruited

209,000+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials

3,987

Recruited

47,860,000+

Findings from Research

The study assessed the safety and feasibility of administering a single dose of Darbepoetin alpha (Darbe) to 21 neonates with mild neonatal encephalopathy, and found no adverse events reported in either the Darbe or placebo groups.

Both groups showed average developmental scores on the Bayley Scales of Infant and Toddler Development, indicating that while the treatment was safe, further large-scale trials are needed to evaluate the efficacy of Darbe in improving outcomes for these infants.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Darbepoetin as a neuroprotective agent in mild neonatal encephalopathy: a randomized, placebo-controlled, feasibility trial. DuPont, TL., Baserga, M., Lowe, J., et al.[2023]

In a study involving 90 preterm neonates (45 treated with darbepoetin alfa and 45 controls), the administration of darbepoetin alfa did not significantly affect neutrophil counts or the rates of neutropenia compared to the control group.

There were no differences in the occurrence of neutropenia (ANC ≤1000/μL) or severe neutropenia (ANC ≤500/μL) between the darbe-treated and control neonates, indicating that darbepoetin alfa does not impact neutrophil levels in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Characterization of Neutropenia in Preterm Neonates Following Administration of Darbepoetin Alfa. Andrews, N., Friedman, S., Dunham, M., et al.[2023]

Darbepoetin, a long-acting erythropoiesis-stimulating agent (ESA), has shown significant effectiveness in promoting red blood cell production in both term and preterm infants, potentially reducing the need for blood transfusions.

In addition to its erythropoietic effects, darbepoetin may also offer neuroprotective benefits, helping to prevent and treat brain injuries in infants, which highlights its dual role in neonatal care.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Darbepoetin Administration in Term and Preterm Neonates. Patel, S., Ohls, RK.[2018]

Darbepoetin alfa (DA) was safely administered to 3 neonates with Rhesus isoimmunization to treat late-onset anemia, with no adverse effects reported.

The treatment with DA reduced the need for packed red blood cell transfusions, indicating its efficacy in managing anemia in these infants.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Darbepoetin Alfa for Late-onset Anemia in Neonates with Rhesus Hemolytic Disease. Rv, S., S, R.[2023]

References

1. United States pubmed.ncbi.nlm.nih.gov

Darbepoetin as a neuroprotective agent in mild neonatal encephalopathy: a randomized, placebo-controlled, feasibility trial. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Cognitive outcomes of preterm infants randomized to darbepoetin, erythropoietin, or placebo. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Characterization of Neutropenia in Preterm Neonates Following Administration of Darbepoetin Alfa. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Darbepoetin Administration in Term and Preterm Neonates. [2018]

5. United States pubmed.ncbi.nlm.nih.gov

Recombinant human erythropoietin improves neurological outcomes in very preterm infants. [2018]

6. United States pubmed.ncbi.nlm.nih.gov

Darbepoetin administration to neonates undergoing cooling for encephalopathy: a safety and pharmacokinetic trial. [2018]

7. India pubmed.ncbi.nlm.nih.gov

Darbepoetin Alfa for Late-onset Anemia in Neonates with Rhesus Hemolytic Disease. [2023]