A Phase 1/2 interventional study of Oxandrolone in Hypoplastic Left Heart and Congenital Heart Disease, sponsored by Carelon Research. Terminated at 10 sites in 2 countries. Open to participants aged Up to 14 Days. Per ClinicalTrials.gov, last updated 2024-05-23.
Sponsored by Carelon Research · Phase 1/2, Interventional, and Supportive care
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.
The proposed investigation is a Phase I/II randomized trial of 28 days of open label oxandrolone vs. no oxandrolone treatment to assess optimal dosing, safety/tolerability, and preliminary efficacy of this therapy in post-Norwood neonates with HLHS. Control subjects will receive standard therapy with no placebo and no oxandrolone.
This trial is aimed at cumulative dose finding as well as a preliminary assessment of safety/tolerability and efficacy. The design and dosing are based upon preliminary phase I data obtained as part of an ongoing protocol under IND #107706. This trial will initially include two arms (control and 0.1 mg/kg oxandrolone BID). This initial oxandrolone dose was chosen based on the preliminary data collected in the background studies conducted for this trial. There were no adverse safety outcomes in the small cohort of subjects receiving 0.1 mg/kg of oxandrolone BID.
In Cohort 1, subjects will be block randomized into each arm in a 1:4 (control to oxandrolone) ratio. An interim analysis of the safety data will be performed after the first 25 subjects in Cohort 1 have been randomized and have completed 28 days of oxandrolone therapy or observation (control group). If there are no significant differences in the primary safely/tolerability outcome and safety reviews are favorable for BID dosing, then Cohort 2 (25 subjects) will be randomized in a 1:4 ratio to the control and TID dosing arms. A similar interim analysis will be performed after Cohort 2 subjects have been randomized and completed 28 days of oxandrolone therapy. Enrollment will again be suspended during this second interim analysis to determine if dose escalation is warranted. Cohort 3A, utilizing 0.15 mg/kg oxandrolone TID would be possible if both Cohorts 1 (0.1 mg/kg BID) and 2 (0.1 mg/kg/dose TID) do not demonstrate any differences in the primary safety/tolerability outcome compared to controls and safety reviews are favorable (Figure 4). If the safety threshold is crossed, then a dose of 0.1 mg/kg/dose BID will be used for cohort 3B. An interim safety analysis will be performed after 25 subjects have been enrolled in this highest dosing arm. If at any point a risk-benefit balance in any cohort is found to be negative, then further enrollment will proceed at the lower dosing arm determined to be safe/tolerable based on the primary outcome and safety review with a 1:4 control:oxandrolone ratio and a total subject number of 100.
If the second interim safety analysis leads to the conclusion that the lower dose (0.1 mg/kg oxandrolone BID) appears to be safe and well tolerated, while the higher dose (0.1 mg/kg oxandrolone TID) is not, then the enrollment will proceed in the 0.1 mg/kg BID arm with a 1:4 ratio. If the lowest dose of oxandrolone (0.1 mg/kg BID) is found to be unsafe, then the trial will be stopped. The benefit of this approach lies in the ability to allocate patients to the highest safe dose arm thus enriching the relevance of safety/tolerability and efficacy information obtained. A higher-dose treatment arm will be used if the data reveal the initial treatment arm is not different from control with regards to the primary outcome. If no safety/tolerability effect is demonstrated, the trial will, by design, function as a randomized, controlled trial with dose-escalation. It is anticipated that the study will conclude with approximately 80 oxandrolone patients (in up to three dosing arms) and 20 control patients.
65 studies on the registry are indexed under Hypoplastic Left Heart Syndrome; 12 are open to participants now.
This study's enrollment of 34 is above the median of 29 across 39 interventional studies indexed under Hypoplastic Left Heart Syndrome.
Browse Hypoplastic Left Heart Syndrome studies →Carelon Research is the lead sponsor of 59 studies on the registry; 4 are open to participants now.
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Exclusion Criteria:
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
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.
Oxandrolone 2.5mg tabs will be suspended in multi-chain triglyceride (MCT) oil and administered buccally.
Also known as: Anavar, Oxandrin
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
Time frame: 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
Time frame: 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
Time frame: From date of treatment initiation until the pre-SCPC evaluation or end of study participation, whichever comes first, up to 9 months
Length-for-age z-score
The efficacy of buccally administered oxandrolone will be evaluated by measuring length-for-age z-score at the end of study drug therapy
Time frame: At the time of completion of study drug therapy, up to 28 days after date of treatment initiation
Weight-for-age z-score
The efficacy of buccally administered oxandrolone will be evaluated by measuring weight-for-age z-score at the end of study drug therapy
Time frame: 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
The efficacy of buccally administered oxandrolone will be evaluated by measuring the change in weight-for-age z-score at the end of study drug therapy
Time frame: From date of pre-Norwood procedure until completion of study drug therapy, up to 28 days
Change in length-for-age z-score
The efficacy of buccally administered oxandrolone will be evaluated by measuring the change in length-for-age z-score at the end of study drug therapy
Time frame: From date of pre-Norwood procedure until completion of study drug therapy, up to 28 days
Prealbumin levels
Serum prealbumin levels will be measured weekly
Time frame: During the duration of therapy
Lean Body Mass
Lean body mass will be assessed using dual energy x-ray absorptiometry (DXA)
Time frame: At the completion of study drug therapy, assessed up to 35 days after initiation of study drug therapy
Decreased right ventricular systolic function
Evidence of ≥moderate right ventricular systolic dysfunction or tricuspid valve regurgitation based on qualitative assessment of clinical echocardiograms if performed
Time frame: At the time of Norwood discharge and at the time of pre-SCPC evaluation, up to 9 months
Plan to share: No
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This study is terminated, as verified in Jul 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Oxandrolone
Hypoplastic Left Heart Syndrome →
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