Use of Oxandrolone to Promote Growth in Infants With HLHS

NCT04090697 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: Oxandrolone
• Study Type: interventional
• Target: 34
• Locations: 2 countries
• Sites: 10

Brief Summary

The primary aim of this study is to determine if clinically relevant doses of buccally administered oxandrolone are safe and tolerable in neonates with hypoplastic left heart syndrome (HLHS) or other single right ventricular anomalies who have undergone a Norwood procedure. The secondary aim is to evaluate the efficacy of buccally administered oxandrolone in improving objective indices of growth and nutrition in neonates who have undergone a Norwood procedure.

Conditions

Hypoplastic Left Heart; Congenital Heart Disease

Outcome Measures

Primary Outcomes (3)

• Biochemical evidence of hepatic dysfunction

  Elevation of serum transaminase levels (alanine transaminase (ALT) and/or aspartate transaminase (AST)) \>4 times the local laboratory upper limit of normal

  From date of treatment initiation until the pre-SCPC evaluation or end of study participation, whichever comes first, up to 9 months

• Virilization

  Standardized physical examination will be performed. Because there are no standard normal values for the various measurements included, each subject will serve as their own control

  From date of treatment initiation until the completion of study drug therapy or end of study participation, whichever comes first, up to 28 days

• SAE probably or definitely related to oxandrolone therapy

  Any SAE probably or definitely related to oxandrolone therapy in the opinion of the medical monitor

  From date of treatment initiation until the pre-SCPC evaluation or end of study participation, whichever comes first, up to 9 months

Secondary Outcomes (7)

• Length-for-age z-score

  At the time of completion of study drug therapy, up to 28 days after date of treatment initiation

• Weight-for-age z-score

  At the time of completion of study drug therapy, up to 28 days after date of treatment initiation

• Change in Weight-for-age z-score

  From date of pre-Norwood procedure until completion of study drug therapy, up to 28 days

• Change in length-for-age z-score

  From date of pre-Norwood procedure until completion of study drug therapy, up to 28 days

• Prealbumin levels

  During the duration of therapy

Study Arms (2)

Oxandrolone Cohort 1

EXPERIMENTAL

Participants randomized to Oxandrolone Cohort 1 will receive 0.1mg/kg of oxandrolone suspended in a multi-chain triglyceride (MCT) oil buccally twice per day.

Drug: Oxandrolone

Standard of Care

NO INTERVENTION

Participants randomized to standard of care will receive the standard therapies provided at the institution at which they are being treated. Control subjects will receive standard therapy with no placebo and no oxandrolone.

Interventions

Oxandrolone DRUG

Oxandrolone 2.5mg tabs will be suspended in multi-chain triglyceride (MCT) oil and administered buccally.

Also known as: Anavar, Oxandrin

Oxandrolone Cohort 1

Eligibility Criteria

• Age: Up to 14 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• HLHS and other single ventricle of right ventricular morphology
• Age and Norwood procedure ≤14 days of age
• Informed consent from parent/guardian

You may not qualify if:

• Small for gestational age (birth weight \<10th percentile for gestational age)
• Prematurity, defined as gestational age \<37 weeks
• Intrauterine growth retardation (birth weight ≤2.5 kg and gestational age ≥38 weeks)
• Chromosomal abnormality, recognizable genetic syndrome or congenital anomalies of more than minor severity associated with growth failure
• Moderate or greater right ventricular systolic dysfunction and/or moderate or greater tricuspid regurgitation prior to the Norwood procedure
• Extracorporeal membrane oxygenation support (ECMO) prior to or within 24 hours of Norwood procedure
• Pre-Norwood interventions (fetal intervention, balloon atrial septostomy for an intact or restrictive atrial septum)
• Pre-Norwood pulmonary venous obstruction
• Pre-Norwood procedure necrotizing enterocolitis and/or other gastrointestinal syndromes
• Known contraindication to oxandrolone
• Planned or current warfarin therapy at screening (warfarin effects are increased by anabolic drugs)
• Significant hepatic dysfunction (elevation of serum transaminase levels greater than two times the upper limit of normal local laboratory standard at screening)
• Hypercalcemia (\>1.5 times upper normal range for lab)
• Nephrotic syndrome
• Unwillingness or inability to return to surgical center for follow-up evaluation

Sponsors & Collaborators

• Carelon Research: lead

Study Sites (10)

Children's Hospital of Atlanta

Atlanta, Georgia, 30322, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Michigan Health System, Ann Arbor

Ann Arbor, Michigan, 48109, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Primary Children's Medical Center

Salt Lake City, Utah, 84113, United States

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

MeSH Terms

Conditions

Hypoplastic Left Heart Syndrome; Heart Defects, Congenital

Interventions

Oxandrolone

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

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

Study Officials

• Phillip T Burch, MD

  University of Mississippi Medical Center

  PRINCIPAL INVESTIGATOR

• Richard V Williams, MD

  University of Utah

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 4, 2019
• First Posted: September 16, 2019
• Study Start: December 20, 2019
• Primary Completion: September 19, 2023
• Study Completion: September 19, 2023
• Last Updated: May 23, 2024

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (9); CA (1)