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Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency — STEP-OTC  

Ornithine Transcarbamylase Deficiency Clinical Trial

Official title:
A Phase 1/2 Single Ascending Dose Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenously Administered MRT5201 in Subjects With Ornithine Transcarbamylase Deficiency

Clinical Trial Summary

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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

  • Ornithine Transcarbamylase Deficiency
Clinical Trial Details
NCT number NCT03767270
Study type Interventional
Source Translate Bio, Inc.
Contact
Status Withdrawn
Phase Phase 1/Phase 2
Start date December 2019
Completion date July 2022
View Details
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