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

Study Overview
=================
Brief Summary
-----------------
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.

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

Conditions
-----------------
Malnutrition

Intervention / Treatment
-----------------
* Drug: Oxandrolone


Participation Criteria
=================
Eligibility Criteria
-----------------
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.

Ages Eligible for Study
-----------------
Maximum Age: 1 Month

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Oxandrolone<br>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<br>* 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.)<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| PK testing |  | 48 hours | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | 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 | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Neonates with HLHS or variant with plan to undergo Norwood Procedure