Safety and Tolerability of RTX-134 in Adults With Phenylketonuria

NCT04110496 | Terminated Phase 1 FDA Drug

• 1 other identifier: RTX-134-01
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Phenylketonurias

Outcome Measures

Primary Outcomes (5)

• 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 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

• 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

Study Arms (1)

RTX-134

EXPERIMENTAL

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

Drug: RTX-134

Interventions

RTX-134 DRUG

RTX-134 is a cellular therapy containing AvPAL

RTX-134

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 years or older with:
• A clinical diagnosis of PKU, and
• Average blood phenylalanine level ≥ 600 µmol/L based on 2 assessments up to 3 weeks apart during the 6-month period before Day 0 (per available data)
• Stable diet, including medical formula
• Must be a man or a woman not of childbearing potential and agree to use adequate contraception throughout and for one year following study participation.
• Adequate organ function
• Negative antibody detection on type and screen and no evidence of clinical hemolysis

You may not qualify if:

• Known hypersensitivity to any component of study treatment
• Prior treatment with Pegaliase
• Start of sapropterin dihydrochloride within 3 weeks of study dosing
• Use of an investigational agent within 28 days of study dosing
• Concurrent participation in an interventional trial involving ongoing treatment, including placebo.
• Infections requiring antimicrobial treatment within 7 days of study dosing
• Chronic infections, such as HIV, hepatitis B, or untreated hepatitis C
• Conditions that may alter survival of red blood cells, (e.g., autoimmune diseases, splenectomy, etc)
• Pregnant or breastfeeding

Sponsors & Collaborators

• Rubius Therapeutics: lead

Study Sites (1)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single Ascending Dose

Study Record Dates

• First Submitted: September 23, 2019
• First Posted: October 1, 2019
• Study Start: January 29, 2020
• Primary Completion: December 1, 2022
• Study Completion: December 1, 2022
• Last Updated: December 8, 2022

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)