Safety and Tolerability of RTX-134 in Adults With Phenylketonuria

Phase 1

Terminated

• Conditions: Phenylketonurias
• Interventions: Drug: RTX-134

• Registration Number: NCT04110496
• Lead Sponsor: Rubius Therapeutics

Brief 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).

Study Timeline

• Study First Submitted: 2019-09-23
• Study First Submitted QC: 2019-09-27
• Study First Posted: 2019-10-01
• Study Start: 2020-01-29
• Status Verified: 2022-12
• Primary Completion: 2022-12-01
• Study Completion: 2022-12-01
• Last Update Submitted: 2022-12-07
• Last Update Posted: 2022-12-08

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 1

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
RTX-134	RTX-134	Escalating doses of RTX-134 will be administered by intravenous infusion one time

Primary Outcome Measures

Name	Time	Method
To evaluate the safety and tolerability of RTX-134 as measured by frequency of treatment emergent adverse events	Baseline to 28 days after last detection of RTX-134
To evaluate the pharmacokinetics of RTX-134 as measured by presence of AvPal expressing red cells, AvPAL protein in red cells and serum, and AvPAL enzymatic activity.	Baseline to 28 days after last detection of RTX-134
To correlate dose with percent reduction in serum phenylalanine levels relative to baseline	Baseline to 28 days after last detection of RTX-134
To determine a preliminary dose to achieve serum phenylalanine levels < 600 µmol/L	Baseline to 28 days after last detection of RTX-134
To determine a preliminary dose to achieve serum phenylalanine levels < 360 µmol/L	Baseline to 28 days after last detection of RTX-134

Trial Locations

Locations (1)

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States