Clincosm LogoSign in Get a demo

Safety and Tolerability of RTX-134 in Adults With Phenylketonuria

Sponsor
Rubius Therapeutics (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04110496
Collaborator
(none)
12
Enrollment
1
Location
1
Arm
181
Anticipated Duration (Months)
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
Phase 1

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 Design

Study Type:
Interventional
Anticipated Enrollment :
12 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Intervention Model Description:
Single Ascending DoseSingle Ascending Dose
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 1b Open-Label Single Dose Safety, Tolerability, and Pharmacokinetics Study of RTX-134 in Adults With Phenylketonuria
Actual Study Start Date :
Jan 29, 2020
Anticipated Primary Completion Date :
Mar 1, 2035
Anticipated Study Completion Date :
Mar 1, 2035

Arms and Interventions

Arm Intervention/Treatment
Experimental: RTX-134

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

Drug: RTX-134
RTX-134 is a cellular therapy containing AvPAL

Outcome Measures

Primary Outcome Measures

  1. 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]

  2. To correlate dose with percent reduction in serum phenylalanine levels relative to baseline [Baseline to 28 days after last detection of RTX-134]

  3. To determine a preliminary dose to achieve serum phenylalanine levels < 600 µmol/L [Baseline to 28 days after last detection of RTX-134]

  4. To determine a preliminary dose to achieve serum phenylalanine levels < 360 µmol/L [Baseline to 28 days after last detection of RTX-134]

  5. 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]

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No

Inclusion Criteria

  1. Age 18 years or older with:

  2. A clinical diagnosis of PKU, and

  3. 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)

  4. Stable diet, including medical formula

  5. 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.

  6. Adequate organ function

  7. Negative antibody detection on type and screen and no evidence of clinical hemolysis

Exclusion Criteria

  1. Known hypersensitivity to any component of study treatment

  2. Prior treatment with Pegaliase

  3. Start of sapropterin dihydrochloride within 3 weeks of study dosing

  4. Use of an investigational agent within 28 days of study dosing

  5. Concurrent participation in an interventional trial involving ongoing treatment, including placebo.

  6. Infections requiring antimicrobial treatment within 7 days of study dosing

  7. Chronic infections, such as HIV, hepatitis B, or untreated hepatitis C

  8. Conditions that may alter survival of red blood cells, (e.g., autoimmune diseases, splenectomy, etc)

  9. Pregnant or breastfeeding

Contacts and Locations

Locations

Site City State Country Postal Code
1 Children's Hospital Colorado Aurora Colorado United States 80045

Sponsors and Collaborators

  • Rubius Therapeutics

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Rubius Therapeutics
ClinicalTrials.gov Identifier:
NCT04110496
Other Study ID Numbers:
  • RTX-134-01
First Posted:
Oct 1, 2019
Last Update Posted:
Jan 10, 2022
Last Verified:
Jun 1, 2021
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Phenylketonurias

Study Results

No Results Posted as of Jan 10, 2022