Status:
TERMINATED
Safety and Tolerability of RTX-134 in Adults With Phenylketonuria
Lead Sponsor:
Rubius Therapeutics
Conditions:
Phenylketonurias
Eligibility:
All Genders
18+ years
Phase:
PHASE1
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 ...
Eligibility Criteria
Inclusion
- Inclusion Criteria
- 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
- Exclusion Criteria
- 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
Exclusion
Key Trial Info
Start Date :
January 29 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2022
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT04110496
Start Date
January 29 2020
End Date
December 1 2022
Last Update
December 8 2022
Active Locations (1)
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1
Children's Hospital Colorado
Aurora, Colorado, United States, 80045