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Status: Withdrawn prior to enrolment
Phase of Trial: Phase I/II
Latest Information Update: 18 Sep 2019
Price : $35 *
At a glance
- Drugs MRT-5201 (Primary)
- Indications Ornithine carbamoyltransferase deficiency disease
- Focus Adverse reactions; First in man
- Acronyms STEP-OTC
- Sponsors Translate Bio
- 12 Sep 2019 Status changed from not yet recruiting to withdrawn prior to enrolment.
- 26 Jun 2019 According to a Translate Bio media release, the U.S. Food and Drug Administration (FDA) has removed the clinical hold and allowed to proceed with a single-ascending dose trial after the discussions with the FDA and after amending the protocol.
- 26 Jun 2019 Planned number of patients changed from 24 to 12.