Erythropoietin to Prevent Unnecessary Transfusions In Patients With Cyanotic CHD - A Prospective Control Trial

NCT02564796 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: 160871
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief Summary

Cyanotic congenital cardiac patients require higher hemoglobin concentrations (red blood cell levels) for optimal oxygen delivery to the body. Prophylactic erythropoietin (EPO) and iron can prevent and/or decrease the amount of blood transfusions needed in this population. We seek to investigate if EPO and iron make a clinically significant difference in the number of transfusions given to these patients and the morbidity associated with it.

Conditions

Cyanotic Congenital Heart Disease; Anemia; Cyanosis; Congenital Heart Disease

Keywords

Erythropoetin; Cyanotic Heart Disease; Transfusion; Congenital Heart Disease

Outcome Measures

Primary Outcomes (1)

• Number of Transfusions Needed

  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.

  First 4 months of life

Secondary Outcomes (4)

• Oxygen Saturation

  First 4 months of life

• Number of Hospitalizations

  First 4 months of life

• Weight Gain

  First 4 months of life

• Time to Initial Discharge

  First 4 months of life

Study Arms (2)

Control

PLACEBO COMPARATOR

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.

Drug: Iron

Epoetin alfa and iron supplements

EXPERIMENTAL

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.

Drug: Epoetin Alfa and Iron

Interventions

Epoetin Alfa and Iron DRUG

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.

Also known as: No other name

Epoetin alfa and iron supplements

Iron DRUG

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.

Also known as: No other name

Control

Eligibility Criteria

• Age: Up to 8 Weeks
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Newborns less than 4 weeks old at diagnosis
• Gestational age \>34 weeks
• Birth weight 2.2-4kg
• Cyanotic heart disease who have had a surgical shunt or a catheterization intervention that is equivalent to a shunt (patent ductus arteriosus stent, right ventricular outflow tract stent).
• Baseline hematocrit to be below \<40%.
• Completes at least 1 injection in the study by 8 weeks of age.

You may not qualify if:

• Infants diagnosed at greater than 4 weeks of age
• Gestation \<34 weeks
• Birth weight \<2.2 kg or \>4kg
• Hematocrit \>40%
• Newborns with acyanotic heart disease
• Infants with significant co-morbidities:
• Renal failure (Creatinine \> 2 standard deviations above age adjusted norm)
• Hepatic failure (elevated AST/ALT levels \> 2 standard deviations above age adjusted norm
• Hemolytic disease
• Hemoglobinopathies (Sickle-cell disease, Thalassemias)

Sponsors & Collaborators

• University of California, San Diego: lead

Study Sites (1)

Rady Children's Hospital

San Diego, California, 92123, United States

Location

Related Publications (4)

• Donato H. Erythropoietin: an update on the therapeutic use in newborn infants and children. Expert Opin Pharmacother. 2005 May;6(5):723-34. doi: 10.1517/14656566.6.5.723.

  PMID: 15934899 BACKGROUND

• Fearon JA, Weinthal J. The use of recombinant erythropoietin in the reduction of blood transfusion rates in craniosynostosis repair in infants and children. Plast Reconstr Surg. 2002 Jun;109(7):2190-6. doi: 10.1097/00006534-200206000-00002.

  PMID: 12045535 BACKGROUND

• Maier RF, Obladen M, Muller-Hansen I, Kattner E, Merz U, Arlettaz R, Groneck P, Hammer H, Kossel H, Verellen G, Stock GJ, Lacaze-Masmonteil T, Claris O, Wagner M, Matis J, Gilberg F; European Multicenter Erythropoietin Beta Study Group. Early treatment with erythropoietin beta ameliorates anemia and reduces transfusion requirements in infants with birth weights below 1000 g. J Pediatr. 2002 Jul;141(1):8-15. doi: 10.1067/mpd.2002.124309.

  PMID: 12091844 BACKGROUND

• Richard S, Brion JP, Couck AM, Flament-Durand J. Accumulation of smooth endoplasmic reticulum in Alzheimer's disease: new morphological evidence of axoplasmic flow disturbances. J Submicrosc Cytol Pathol. 1989 Jul;21(3):461-7.

  PMID: 2477140 BACKGROUND

MeSH Terms

Conditions

Anemia; Cyanosis; Heart Defects, Congenital

Interventions

Epoetin Alfa; Iron

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Erythropoietin; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Metals, Heavy; Elements; Inorganic Chemicals; Transition Elements; Metals

Limitations and Caveats

The principal investigator discussed the study's future with the sub-investigators on the study and came to the conclusion to terminate the study early due to low enrollment while also considering the financial costs to continue to run the study. It would be highly improbable to be able to enroll 54 more patients with the amount of funding left in the grant. No subjects were analyzed.

Results Point of Contact

• Title: Dr. David Werho
• Organization: UC San Diego

dwerho@rchsd.org

(858) 966-5855

Study Officials

• David K Werho, MD

  University of California, San Diego/Rady Children's Hospital San Diego

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Clinical Professor

Study Record Dates

• First Submitted: September 29, 2015
• First Posted: October 1, 2015
• Study Start: November 1, 2016
• Primary Completion: June 1, 2020
• Study Completion: June 1, 2020
• Last Updated: December 2, 2021
• Results First Posted: December 2, 2021

Record last verified: 2021-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)