A Multiple-Part Phase 1/2 Study Evaluating the Safety, Tolerability, PK, and PD of Intravenously Administered MRT5201 in Subjects With Ornithine Transcarbamylase Deficiency, Comprising Single Ascending Dose and Multiple Dose Parts
Phase of Trial: Phase I/II
Latest Information Update: 22 Jan 2019
At a glance
- Drugs MRT-5201 (Primary)
- Indications Inborn urea cycle disorders; Ornithine carbamoyltransferase deficiency disease
- Focus Adverse reactions; First in man
- Acronyms STEP-OTC
- 22 Jan 2019 Status changed from not yet recruiting to suspended, according to a Translate Bio media release.
- 22 Jan 2019 According to a Translate Bio media release, company received verbal notification from the US FDA that the agency has completed its review of the IND submission for MRT 5201 for the treatment of ornithine transcarbamylase (OTC) deficiency and has additional clinical and non-clinical questions. The FDA placed a clinical hold on the IND until these questions are resolved. IND was submitted in December 2018.
- 04 Dec 2018 Status changed from planning to not yet recruiting.