Pilot Study of the Safety and Efficacy of Oxandrolone in the Prevention and Treatment of Malnutrition in Infants

A Pilot Study of the Safety and Efficacy of Oxandrolone in the Prevention and Treatment of Malnutrition in Infants After Cardiac Surgery

Oxandrolone is an anabolic steroid, marketed in the United States as an adjunctive therapy to combat weight loss resulting from chronic infection, extensive surgery, severe trauma, protein catabolism associated with prolonged administration of corticosteroids, and for the relief of bone pain accompanying osteoporosis. In children, it has been used to prevent and treat growth failure associated with severe burns (≥ 40% of total body surface area), Duchenne muscular dystrophy, Turner's syndrome, constitutional delay of growth and puberty, and chronic wasting in HIV-positive pediatric patients. Other applications in children have included treatment of central idiopathic precocious puberty, hereditary angioedema, and bilateral congenital anorchia. Growth failure is a common feature of infants with complex congenital heart disease, and can adversely affect outcome. This therapy has not been previously implemented in neonates thus we will evaluate the safety and efficacy of administering oxandrolone to improve growth in neonates with complex congenital heart disease who have undergone surgical palliation or repair by collecting anthropometric measurements and pharmacokinetic data.

Neonates with HLHS or variant with planned Norwood Procedure. The primary aims of this pilot study are to assess safety and efficacy of oxandrolone in this population. Our goal will be to enroll 5 patients in each phase of this pilot study. The incidence of adverse events will also be monitored and compared to untreated patients. Enrollment will continue until the target of 20 total patients has been met.

Study Overview

• Status: Completed
• Conditions: Malnutrition
• Intervention / Treatment: Drug: Oxandrolone

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • Primary Childrens Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 1 year (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Neonates with HLHS, with plan to undergo the Norwood Procedures

Exclusion Criteria:

• Neonates with a birth weight ≤2.5 kg and gestational age ≥38 weeks (indicating intrauterine growth retardation), gestational age < 35 weeks (premature infants), and/or chromosomal or recognizable phenotypic syndrome of non-cardiac congenital abnormalities associated with growth failure (e.g. Trisomy 21, Trisomy 13, Noonan's syndrome, Turner's syndrome) will be excluded from enrollment. Neonates who will not be placed on the high risk feeding protocol postoperatively will also be excluded.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Oxandrolone; Following clinical evaluation, all patients will be receive oxandrolone in addition to their usual medications. Based upon the patient's body weight, the coordinator, PI or sub-PI will determine the appropriate dose of oxandrolone (0.1 mg/kg/dose twice daily via the buccal mucosa.)	Drug: Oxandrolone; 2.5 mg oxandrolone tablets.Based upon the patient's body weight, the coordinator, PI or sub-PI will determine the appropriate dose of oxandrolone (0.1 mg/kg/dose twice daily via the buccal mucosa.)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
PK testing	48 hours

Secondary Outcome Measures

Outcome Measure	Time Frame
Measures of Severity of Malnutrition to include pre albumin, C-reactive protein, nitrogen balance and measurements of somatic growth.	Measurements: Baseline, Day #1, Day #7, Day #14, Day #21, Day #28

Collaborators and Investigators

• Sponsor: University of Utah
• Investigators: Principal Investigator: Phillip T Burch, MD, University of Utah Dept. of Surgery-Div Cardiothoracic surgery

Study record dates

Study Major Dates

• Study Start: July 1, 2010
• Primary Completion (Actual): May 1, 2016
• Study Completion (Actual): May 1, 2016

Study Registration Dates

• First Submitted: January 8, 2010
• First Submitted That Met QC Criteria: January 11, 2010
• First Posted (Estimate): January 13, 2010

Study Record Updates

• Last Update Posted (Actual): January 29, 2020
• Last Update Submitted That Met QC Criteria: January 27, 2020
• Last Verified: January 1, 2020

More Information

Terms related to this study

• Keywords: Neonates with HLHS or variant with plan to undergo Norwood Procedure
• Additional Relevant MeSH Terms: Nutrition Disorders; Malnutrition; Infant Nutrition Disorders; Physiological Effects of Drugs; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Androgens; Anabolic Agents; Oxandrolone
• Other Study ID Numbers: 41510