Trial Outcomes & Findings for Erythropoietin to Prevent Unnecessary Transfusions In Patients With Cyanotic CHD - A Prospective Control Trial (NCT NCT02564796)
NCT ID: NCT02564796
Last Updated: 2021-12-02
Results Overview
Prophylactic erythropoietin can prevent and/or decrease the amount of blood transfusions needed prior to surgery. We seek to investigate if erythropoietin makes a clinically significant difference in the number of transfusions given to these patients and the morbidity associated with it during the period in which the subjects will be active in the study (from baseline to 14 weeks post initial injection). The primary aim will be assessed when all subjects have completed week 14 or discontinue early.
TERMINATED
PHASE2
4 participants
First 4 months of life
2021-12-02
Participant Flow
4 Patients who had been screened/consented for the study did not reach the minimum hematocrit threshold for enrollment and were therefore screen failures.
Participant milestones
| Measure |
Control
Group II (non-treatment group): Patients in the treatment group will not receive any extra intervention outside of standard of care. They will receive iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization). They will be followed for 14 weeks.
Iron: Patients in the treatment group will not receive any extra intervention outside of standard of care. They will receive iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization). They will be followed for 14 weeks.
|
Epoetin Alfa and Iron Supplements
Group I (treatment group): Patients in the treatment group will receive weekly EPO injections and iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization) They will be followed for 14 weeks.
Epoetin Alfa and Iron: Patients in the treatment group will receive weekly EPO injections and iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization) They will be followed for 14 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
2
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
2
|
Reasons for withdrawal
| Measure |
Control
Group II (non-treatment group): Patients in the treatment group will not receive any extra intervention outside of standard of care. They will receive iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization). They will be followed for 14 weeks.
Iron: Patients in the treatment group will not receive any extra intervention outside of standard of care. They will receive iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization). They will be followed for 14 weeks.
|
Epoetin Alfa and Iron Supplements
Group I (treatment group): Patients in the treatment group will receive weekly EPO injections and iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization) They will be followed for 14 weeks.
Epoetin Alfa and Iron: Patients in the treatment group will receive weekly EPO injections and iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization) They will be followed for 14 weeks.
|
|---|---|---|
|
Overall Study
Physician Decision
|
2
|
2
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Control
n=2 Participants
Group II (non-treatment group): Patients in the treatment group will not receive any extra intervention outside of standard of care. They will receive iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization). They will be followed for 14 weeks.
Iron: Patients in the treatment group will not receive any extra intervention outside of standard of care. They will receive iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization). They will be followed for 14 weeks.
|
Epoetin Alfa and Iron Supplements
n=2 Participants
Group I (treatment group): Patients in the treatment group will receive weekly EPO injections and iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization) They will be followed for 14 weeks.
Epoetin Alfa and Iron: Patients in the treatment group will receive weekly EPO injections and iron supplementation for 6 weeks starting before 8 weeks of age, 1 week after their first procedure (surgery or heart catheterization) They will be followed for 14 weeks.
|
Total
n=4 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=2 Participants
|
2 Participants
n=2 Participants
|
4 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
1 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=2 Participants
|
2 Participants
n=2 Participants
|
3 Participants
n=4 Participants
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Region of Enrollment
United States
|
2 Participants
n=2 Participants
|
2 Participants
n=2 Participants
|
4 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: First 4 months of lifePopulation: The principal investigator discussed the study's future with the sub-investigators on the study and came to the conclusion to close the study due to low enrollment while also considering the financial costs to continue to run the study.
Prophylactic erythropoietin can prevent and/or decrease the amount of blood transfusions needed prior to surgery. We seek to investigate if erythropoietin makes a clinically significant difference in the number of transfusions given to these patients and the morbidity associated with it during the period in which the subjects will be active in the study (from baseline to 14 weeks post initial injection). The primary aim will be assessed when all subjects have completed week 14 or discontinue early.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: First 4 months of lifePopulation: The principal investigator discussed the study's future with the sub-investigators on the study and came to the conclusion to close the study due to low enrollment while also considering the financial costs to continue to run the study.
Often, cyanotic congenital heart defect neonates have prolonged initial hospital stays due to the inability to maintain acceptable oxygen saturations, and transition to adequate oral intake for appropriate weight gain. If the hospital stay is found to be shortened after starting erythropoietin, this may be of clinical and financial significance.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: First 4 months of lifePopulation: The principal investigator discussed the study's future with the sub-investigators on the study and came to the conclusion to close the study due to low enrollment while also considering the financial costs to continue to run the study.
The number of hospital readmissions (related to failure to thrive or cyanosis) may imply the overall clinical stability of a patient. Because these infants are at high risk for mortality at home, there are multiple reasons why they may be admitted to the hospital including clinically significant anemia which requires blood transfusions, poor weight gain, difficulty feeding, inadequate oxygen saturations, and illnesses. Each admission is stressful to the patient and their families. Having a normal hemoglobin level may have a role in preventing several of these factors, especially regarding failure to thrive or cyanosis.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: First 4 months of lifePopulation: The principal investigator discussed the study's future with the sub-investigators on the study and came to the conclusion to close the study due to low enrollment while also considering the financial costs to continue to run the study.
The secondary outcome of weight gain is appropriate in the setting of infants as this variable has been used to monitor the ability to thrive and meet the body's metabolic demands. It is well established in pediatrics that the neonate and infant should gain 15-30 grams per day for optimal growth. Infants who are cyanotic already have a deficiency in meeting their metabolic demands due to a reduced oxygen carrying capacity. This is further complicated in the instance of anemia. Thus, infants may have an increased ability to optimize weight gain in the setting of normal, stable hemoglobin levels which may be achieved with erythropoietin.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: First 4 months of lifePopulation: The principal investigator discussed the study's future with the sub-investigators on the study and came to the conclusion to close the study due to low enrollment while also considering the financial costs to continue to run the study.
If the hospital stay is found to be shortened after starting erythropoietin, this may be of clinical and financial significance.
Outcome measures
Outcome data not reported
Adverse Events
Control
Epoetin Alfa and Iron Supplements
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place