Trial Outcomes & Findings for Darbepoetin Administration to Preterm Infants (NCT NCT00334737)

NCT ID: NCT00334737

Last Updated: 2019-02-04

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 102 participants
• Primary outcome timeframe: From birth to 36 weeks gestational age
• Results posted on: 2019-02-04

Participant Flow

Participant milestones

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control Sham injection sham injection: sham injection
Hospital Phase STARTED	34	34	34
Hospital Phase COMPLETED	32	32	30
Hospital Phase NOT COMPLETED	2	2	4
Follow up Phase STARTED	32	32	30
Follow up Phase COMPLETED	27	29	24
Follow up Phase NOT COMPLETED	5	3	6

Reasons for withdrawal

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control Sham injection sham injection: sham injection
Hospital Phase Death	1	2	3
Hospital Phase Withdrawal by Subject	0	0	1
Hospital Phase Did not receive study drug	1	0	0
Follow up Phase Lost to Follow-up	5	3	6

Baseline Characteristics

Darbepoetin Administration to Preterm Infants

Baseline characteristics by cohort

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=27 Participants Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=29 Participants Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=24 Participants Sham injection sham injection: sham injection	Total n=80 Participants Total of all reporting groups
Age, Continuous	20 months n=5 Participants	21 months n=7 Participants	20 months n=5 Participants	21 months n=4 Participants
Sex: Female, Male Female	17 Participants n=5 Participants	10 Participants n=7 Participants	9 Participants n=5 Participants	36 Participants n=4 Participants
Sex: Female, Male Male	10 Participants n=5 Participants	19 Participants n=7 Participants	15 Participants n=5 Participants	44 Participants n=4 Participants
Region of Enrollment United States	27 participants n=5 Participants	29 participants n=7 Participants	24 participants n=5 Participants	80 participants n=4 Participants
gestation	28.3 weeks n=5 Participants	27.7 weeks n=7 Participants	28.1 weeks n=5 Participants	28 weeks n=4 Participants
birthweight	980 grams n=5 Participants	930 grams n=7 Participants	1005 grams n=5 Participants	975 grams n=4 Participants

PRIMARY outcome

Timeframe: From birth to 36 weeks gestational age

Outcome measures

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=33 Participants Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=33 Participants Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=33 Participants Sham injection sham injection: sham injection
Number of Transfusions During Hospitalization	1.2 number of transfusions Standard Deviation 2.4	1.2 number of transfusions Standard Deviation 1.6	2.4 number of transfusions Standard Deviation 2.9

PRIMARY outcome

Timeframe: 18-22 months

Bayley Scale of Infant Development Composite Cognitive Score. The total composite score is reported, ranging from the lowest score of 55 to the highest score of 145. Lower values specify worse outcome.

Outcome measures

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=27 Participants Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=29 Participants Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=24 Participants Sham injection sham injection: sham injection
Composite Cognitive Score at 18-22 Months Corrected Age	96.2 BSID III compositie cognitive score Standard Deviation 7.2	97.9 BSID III compositie cognitive score Standard Deviation 14.3	88.7 BSID III compositie cognitive score Standard Deviation 13.5

SECONDARY outcome

Timeframe: From birth to 36 weeks gestational age

Outcome measures

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=32 Participants Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=32 Participants Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=30 Participants Sham injection sham injection: sham injection
Hematocrit	0.35 L/L Standard Deviation 0.09	0.36 L/L Standard Deviation 0.08	0.29 L/L Standard Deviation 0.09

SECONDARY outcome

Timeframe: at day 60 of study

absolute retic count measured at the end of study

Outcome measures

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=32 Participants Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=32 Participants Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=30 Participants Sham injection sham injection: sham injection
Reticulocyte Count	295 1000 cells per microliter Standard Error 50	298 1000 cells per microliter Standard Error 47	130 1000 cells per microliter Standard Error 70

SECONDARY outcome

Timeframe: From birth to 36 weeks gestational age

Outcome measures

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=32 Participants Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=32 Participants Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=30 Participants Sham injection sham injection: sham injection
Volume of Transfusions	30 mL/kg Standard Deviation 58	23 mL/kg Standard Deviation 33	51 mL/kg Standard Deviation 65

SECONDARY outcome

Timeframe: peak from birth to 36 weeks gestational age

Population: One hospital participating in the trial lost their samples. The number analyzed only includes those with samples.

Outcome measures

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=19 Participants Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=16 Participants Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=20 Participants Sham injection sham injection: sham injection
Epo Concentrations	1398.9 mU/mL Standard Deviation 882.7	515.4 mU/mL Standard Deviation 280.3	28.6 mU/mL Standard Deviation 51.1

SECONDARY outcome

Timeframe: 18-22 months

Scores are 0-3, with 3 being the best score.

Outcome measures

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=27 Participants Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=29 Participants Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=24 Participants Sham injection sham injection: sham injection
Object Permanence Scores at 18-22 Months	2.8 units on a scale Standard Deviation 0.4	2.4 units on a scale Standard Deviation 0.9	2.2 units on a scale Standard Deviation 1.0

SECONDARY outcome

Timeframe: 18-22 months

Outcome measures

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=28 Participants Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=30 Participants Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=27 Participants Sham injection sham injection: sham injection
Overall Neurodevelopmental Impairment at 18-22 Months (Visual Impairment, Hearing Impariment, Cerebral Palsy, or Cognitive Score <85/<70 (NDI/Moderate NDI)	4 participants	5 participants	13 participants

SECONDARY outcome

Timeframe: From birth to 36 weeks gestational age

Outcome measures

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=32 Participants Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=32 Participants Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=30 Participants Sham injection sham injection: sham injection
Incidence of Retinopathy of Prematurity Stage 3 or Greater	2 participants	1 participants	2 participants

Adverse Events

Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Placebo/Control

Serious events: 3 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=33 participants at risk Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=33 participants at risk Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=33 participants at risk Sham injection sham injection: sham injection
General disorders death	3.0% 1/33 • Number of events 33 • Initial hospitalization after preterm birth Adverse events collected during subject's initial hospitalization in the NICU	3.0% 1/33 • Number of events 33 • Initial hospitalization after preterm birth Adverse events collected during subject's initial hospitalization in the NICU	9.1% 3/33 • Number of events 33 • Initial hospitalization after preterm birth Adverse events collected during subject's initial hospitalization in the NICU

Other adverse events

Measure	Darbepoetin 10 Mics/kg/Week x 10 Weeks or Until 35 Completed w n=33 participants at risk Darbepoetin 10 mics/kg/week x 10 weeks or until 35 completed weeks darbepoetin alfa: darbepoetin 10 mics/kg once a week SC for 10 weeks or until 35 completed weeks	Epo 400 Units/kg Three Times a Week SC x 10 Weeks or Until 35 n=33 participants at risk Epo 400 units/kg three times a week SC x 10 weeks or until 35 completed weeks erythropoietin: Epo 400 units/kg 3 x weekly SC for 10 weeks or until 35 completed weeks gestation	Placebo/Control n=33 participants at risk Sham injection sham injection: sham injection
Eye disorders ROP>stage 2	6.1% 2/33 • Number of events 2 • Initial hospitalization after preterm birth Adverse events collected during subject's initial hospitalization in the NICU	3.0% 1/33 • Number of events 1 • Initial hospitalization after preterm birth Adverse events collected during subject's initial hospitalization in the NICU	6.1% 2/33 • Number of events 2 • Initial hospitalization after preterm birth Adverse events collected during subject's initial hospitalization in the NICU
Nervous system disorders IVH>grade 2	9.1% 3/33 • Number of events 3 • Initial hospitalization after preterm birth Adverse events collected during subject's initial hospitalization in the NICU	9.1% 3/33 • Number of events 3 • Initial hospitalization after preterm birth Adverse events collected during subject's initial hospitalization in the NICU	21.2% 7/33 • Number of events 7 • Initial hospitalization after preterm birth Adverse events collected during subject's initial hospitalization in the NICU

Additional Information

Dr. Robin K Ohls

University of New Mexico

Phone: 505-272-6753

Email: rohls@salud.unm.edu

Results disclosure agreements

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