Trials / Withdrawn
WithdrawnNCT03767270
Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency
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
- Status
- Withdrawn
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 0 (actual)
- Sponsor
- Translate Bio, Inc. · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | MRT5201 | Codon-optimized human ornithine transcarbamylase messenger ribonucleic acid with lipid-based nanoparticles |
| OTHER | Placebo | 5% dextrose in water |
Timeline
- Start date
- 2019-12-01
- Primary completion
- 2022-07-01
- Completion
- 2022-07-01
- First posted
- 2018-12-06
- Last updated
- 2019-09-17
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03767270. Inclusion in this directory is not an endorsement.