Trial Outcomes & Findings for Mild Encephalopathy in the Newborn Treated With Darbepoetin (NCT NCT03071861)

NCT ID: NCT03071861

Last Updated: 2023-12-21

Results Overview

The Bayley III and Neuromuscular Assessment were completed between 9-12 months of age. Subjects were abnormal if they had a Bayley III score of less than 70 and/or an abnormal neurological examination.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 28 participants
• Primary outcome timeframe: 9 - 12 months of age
• Results posted on: 2023-12-21

Participant Flow

Participant milestones

Measure	Darbepoetin Alpha IV,10 mcg/kg/dose, Darbepoetin Alpha, one dose at \<24 hours of age Darbepoetin Alfa: Single dose of 10 mcg/kg Darbepoetin Alpha given IV at less than 24 hours of age	Placebo IV, Normal saline (placebo dose), one dose at \<24 hours of age Normal Saline: Single dose of normal saline, IV, given at less than 24 hours of age
Overall Study STARTED	13	15
Overall Study COMPLETED	12	14
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Darbepoetin Alpha n=13 Participants IV,10 mcg/kg/dose, Darbepoetin Alpha, one dose at \<24 hours of age Darbepoetin Alfa: Single dose of 10 mcg/kg Darbepoetin Alpha given IV at less than 24 hours of age	Placebo n=15 Participants IV, Normal saline (placebo dose), one dose at \<24 hours of age Normal Saline: Single dose of normal saline, IV, given at less than 24 hours of age	Total n=28 Participants Total of all reporting groups
Age, Categorical <=18 years	13 Participants n=13 Participants	15 Participants n=15 Participants	28 Participants n=28 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=13 Participants	0 Participants n=15 Participants	0 Participants n=28 Participants
Age, Categorical >=65 years	0 Participants n=13 Participants	0 Participants n=15 Participants	0 Participants n=28 Participants
Age, Continuous	39 weeks STANDARD_DEVIATION 1.9 • n=13 Participants	39 weeks STANDARD_DEVIATION 1.5 • n=15 Participants	39 weeks STANDARD_DEVIATION 1.7 • n=28 Participants
Sex: Female, Male Female	6 Participants n=13 Participants	7 Participants n=15 Participants	13 Participants n=28 Participants
Sex: Female, Male Male	7 Participants n=13 Participants	8 Participants n=15 Participants	15 Participants n=28 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment United States	13 participants n=13 Participants	15 participants n=15 Participants	28 participants n=28 Participants
gestational age less than 36 weeks	0 Participants n=13 Participants	1 Participants n=15 Participants	1 Participants n=28 Participants

PRIMARY outcome

Timeframe: 9 - 12 months of age

Population: one infant was lost to follow-up

The Bayley III and Neuromuscular Assessment were completed between 9-12 months of age. Subjects were abnormal if they had a Bayley III score of less than 70 and/or an abnormal neurological examination.

Outcome measures

Measure	Darbepoetin Alpha n=13 Participants IV,10 mcg/kg/dose, Darbepoetin Alpha, one dose at \<24 hours of age Darbepoetin Alfa: Single dose of 10 mcg/kg Darbepoetin Alpha given IV at less than 24 hours of age	Placebo n=14 Participants IV, Normal saline (placebo dose), one dose at \<24 hours of age Normal Saline: Single dose of normal saline, IV, given at less than 24 hours of age
Normal Neurodevelopment	13 Participants	14 Participants

SECONDARY outcome

Timeframe: 30 days or until hospital discharge whichever comes first

Population: There were NO adverse events reported

Potential adverse events such as (but not limited to) alterations in blood pressure, secondary infections, neutropenia, thrombotic/vascular events, hematologic events (platelets, Hct level, polycythemia), and hepatic/renal function that are outside of normal range for the study population.

Outcome measures

Measure	Darbepoetin Alpha n=13 Participants IV,10 mcg/kg/dose, Darbepoetin Alpha, one dose at \<24 hours of age Darbepoetin Alfa: Single dose of 10 mcg/kg Darbepoetin Alpha given IV at less than 24 hours of age	Placebo n=15 Participants IV, Normal saline (placebo dose), one dose at \<24 hours of age Normal Saline: Single dose of normal saline, IV, given at less than 24 hours of age
Percent of Infants With Adverse Events	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 30 days or until hospital discharge whichever comes first

Population: Patients with seizures after study enrolment

development of clinical or electrographic seizures

Outcome measures

Measure	Darbepoetin Alpha n=13 Participants IV,10 mcg/kg/dose, Darbepoetin Alpha, one dose at \<24 hours of age Darbepoetin Alfa: Single dose of 10 mcg/kg Darbepoetin Alpha given IV at less than 24 hours of age	Placebo n=15 Participants IV, Normal saline (placebo dose), one dose at \<24 hours of age Normal Saline: Single dose of normal saline, IV, given at less than 24 hours of age
Percent of Infants With Seizures	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 30 days or until hospital discharge whichever comes first

Population: Discharge feeding method

Infants who require tube feedings at discharge

Outcome measures

Measure	Darbepoetin Alpha n=13 Participants IV,10 mcg/kg/dose, Darbepoetin Alpha, one dose at \<24 hours of age Darbepoetin Alfa: Single dose of 10 mcg/kg Darbepoetin Alpha given IV at less than 24 hours of age	Placebo n=15 Participants IV, Normal saline (placebo dose), one dose at \<24 hours of age Normal Saline: Single dose of normal saline, IV, given at less than 24 hours of age
Percentage of Infants Who Need Gavage Feeds or Gastrostomy at Discharge Home	0 Participants	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 24 months of age

development of clinical or electrographic seizures

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 9 months of age

Population: 27 infants were followed at 9 months of age. One infant in the placebo arm was lost to follow up.

Growth at <3%

Outcome measures

Measure	Darbepoetin Alpha n=13 Participants IV,10 mcg/kg/dose, Darbepoetin Alpha, one dose at \<24 hours of age Darbepoetin Alfa: Single dose of 10 mcg/kg Darbepoetin Alpha given IV at less than 24 hours of age	Placebo n=14 Participants IV, Normal saline (placebo dose), one dose at \<24 hours of age Normal Saline: Single dose of normal saline, IV, given at less than 24 hours of age
Percent With Failure to Thrive	0 Participants	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 9 months of age

Population: one infant was lost to follow up

Child requires a hearing device

Outcome measures

Measure	Darbepoetin Alpha n=13 Participants IV,10 mcg/kg/dose, Darbepoetin Alpha, one dose at \<24 hours of age Darbepoetin Alfa: Single dose of 10 mcg/kg Darbepoetin Alpha given IV at less than 24 hours of age	Placebo n=14 Participants IV, Normal saline (placebo dose), one dose at \<24 hours of age Normal Saline: Single dose of normal saline, IV, given at less than 24 hours of age
Percent With Hearing Impairment	0 Participants	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 9 months of age

Population: one infant was lost to follow up

requires corrective lenses

Outcome measures

Measure	Darbepoetin Alpha n=13 Participants IV,10 mcg/kg/dose, Darbepoetin Alpha, one dose at \<24 hours of age Darbepoetin Alfa: Single dose of 10 mcg/kg Darbepoetin Alpha given IV at less than 24 hours of age	Placebo n=14 Participants IV, Normal saline (placebo dose), one dose at \<24 hours of age Normal Saline: Single dose of normal saline, IV, given at less than 24 hours of age
Percent With Vision Impairment	0 Participants	0 Participants

Adverse Events

Darbepoetin Alpha

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Tara DuPont

University of Utah

Phone: 8015814178

Email: tara.dupont@hsc.utah.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place