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

NCT03767270 · Status: WITHDRAWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL

Last updated 2019-09-17

No results posted yet for this study

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.

Conditions

  • Ornithine Transcarbamylase Deficiency

Interventions

BIOLOGICAL

MRT5201

Codon-optimized human ornithine transcarbamylase messenger ribonucleic acid with lipid-based nanoparticles

OTHER

Placebo

5% dextrose in water

Sponsors & Collaborators

  • Translate Bio, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-12-31
Primary Completion
2022-07-31
Completion
2022-07-31
FDA Drug
Yes

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03767270 on ClinicalTrials.gov