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This Phase 1/2, first-in-human study will evaluate the safety and tolerability of single escalating doses of MRT5201 administered intravenously to subjects with OTC Deficiency (OTCD). This study will also evaluate the effect of a single dose of MRT5201 on metabolic markers of OTCD and ureagenesis; and determine an acceptable dosing interval of MRT5201.
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Inclusion criteria
Have a documented diagnosis of OTCD.
Documented history of ≥1 symptomatic hyperammonemia event with ammonia ≥100 µmol/L
Subject's OTCD is stable as evidenced by meeting the following criteria:
If using nitrogen scavenger therapy, must be on a stable regimen for ≥28 days prior to signing informed consent
Subject has maintained a stable protein restricted diet (which may or may not include medical foods) and/or amino acid supplementation with no changes in calorie or protein goals and no changes in medical food and/or amino acid supplementation for ≥ 28 days prior to signing informed consent.
Exclusion criteria
Any laboratory abnormality that may put the subject at increased risk by participating in this study.
Have any significant concurrent or past medical condition that would represent an unacceptable risk to the subject or might jeopardize the collection of high-quality data from the study. These include but are not limited to:
Participation in previous clinical studies evaluating investigational OTCD therapies directed at expressing functional OTC protein (eg, OTC gene therapy studies, other mRNA replacement therapy) that has led to the presence of anti-OTC antibodies.
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0 participants in 2 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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