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

NCT01048632 | Completed Phase 1 DMC

• 1 other identifier: 41510
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

Malnutrition

Keywords

Neonates with HLHS or variant with plan to undergo Norwood Procedure

Outcome Measures

Primary Outcomes (1)

• PK testing

  48 hours

Secondary Outcomes (1)

• 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

Study Arms (1)

Oxandrolone

EXPERIMENTAL

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

Interventions

Oxandrolone DRUG

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.)

Oxandrolone

Eligibility Criteria

• Age: Up to 1 Month
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Utah: lead

Study Sites (1)

Primary Childrens Hospital

Salt Lake City, Utah, 84113, United States

Location

MeSH Terms

Conditions

Malnutrition

Interventions

Oxandrolone

Condition Hierarchy (Ancestors)

Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Androstanols; Androstanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Phillip T Burch, MD

  University of Utah Dept. of Surgery-Div Cardiothoracic surgery

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: January 8, 2010
• First Posted: January 13, 2010
• Study Start: July 1, 2010
• Primary Completion: May 1, 2016
• Study Completion: May 1, 2016
• Last Updated: January 29, 2020

Record last verified: 2020-01

Locations

US (1)