Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency (STEP-OTC)
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|ClinicalTrials.gov Identifier: NCT03767270|
Recruitment Status : Withdrawn (Program was discontinued.)
First Posted : December 6, 2018
Last Update Posted : September 17, 2019
Translate Bio, Inc.
Information provided by (Responsible Party):
Translate Bio, Inc.
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.
|Condition or disease||Intervention/treatment||Phase|
|Ornithine Transcarbamylase Deficiency||Biological: MRT5201 Other: Placebo||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
|Estimated Study Start Date :||December 2019|
|Estimated Primary Completion Date :||July 2022|
|Estimated Study Completion Date :||July 2022|
Resource links provided by the National Library of Medicine
Genetic and Rare Diseases Information Center resources: Urea Cycle Disorders Ornithine Transcarbamylase Deficiency Inborn Amino Acid Metabolism Disorder
Single Ascending Low, Mid, and High doses of MRT5201
Codon-optimized human ornithine transcarbamylase messenger ribonucleic acid with lipid-based nanoparticles
Placebo Comparator: Placebo
Placebo comparator using 5% dextrose in water at the same administration rate as study drug.
5% dextrose in water
Primary Outcome Measures :
- The incidence of treatment-emergent adverse events by treatment group [ Time Frame: Week 24 ]The incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) for each dosing cohort by treatment group, assessed by severity and relationship to study product.
Secondary Outcome Measures :
- Pharmacokinetics parameters of MRT5201 [ Time Frame: 1 month after single dose ]Pharmacokinetics of MRT5201 as measured by levels of mRNA
- Effect of a single dose of MRT5201 on ureagenesis [ Time Frame: Up to 1 month after single dose ]Change from Baseline in 4-hour ureagenesis AUC at weeks 2, 3, 4, and 5 after a single dose of MRT5201
- Effect of single dose of MRT5201 on metabolic markers of OTCD [ Time Frame: 6 months after single dose ]Change from Baseline in 8-hour ammonia AUC
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