NCT04110496

Safety and Tolerability of RTX-134 in Adults With Phenylketonuria

A Phase 1b Open-Label Single Dose Safety, Tolerability, and Pharmacokinetics Study of RTX-134 in Adults With Phenylketonuria

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Rubius Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the safety and tolerability of RTX-134 in adult patients with PKU.

Detailed description

This is a Phase 1b first-in-human trial in adult subjects with PKU. The primary objective of this study is to evaluate the safety and tolerability of RTX-134 following intravenous administration of a single dose. RTX-134 consists of allogeneic human red cells expressing the AvPAL (Anabaena variabilis phenylalanine ammonia lyase) gene inside the cell. The trial is designed to determine a preliminary dose and inform a dosing schedule that is deemed safe, tolerable, and potentially effective. Four dose levels are planned, additional dose levels may be explored. Following administration, subjects will be monitored until 28 days after last detection of RTX-134. Detection of RTX-134 will be evaluated using multiple pharmacokinetic (PK) and pharmacodynamic (PD) assessments including measurement of trans-cinnamic acid (tCA).

Conditions

• Phenylketonurias

Interventions

Type	Name	Description
DRUG	RTX-134	RTX-134 is a cellular therapy containing AvPAL

Timeline

• Start date: 2020-01-29
• Primary completion: 2022-12-01
• Completion: 2022-12-01
• First posted: 2019-10-01
• Last updated: 2022-12-08

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study