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Safety and Tolerability of RTX-134 in Adults With Phenylketonuria

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ClinicalTrials.gov Identifier: NCT04110496
Recruitment Status : Recruiting
First Posted : October 1, 2019
Last Update Posted : October 1, 2019
Sponsor:
Information provided by (Responsible Party):
Rubius Therapeutics

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

Condition or disease Intervention/treatment Phase
Phenylketonurias Drug: RTX-134 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).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Single 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
Estimated Study Start Date : September 28, 2019
Estimated Primary Completion Date : April 2020
Estimated Study Completion Date : May 2020


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




Primary Outcome Measures :
  1. To evaluate the safety and tolerability of RTX-134 as measured by frequency of treatment emergent adverse events [ Time Frame: Baseline to 28 days after last detection of RTX-134 ]
  2. To correlate dose with percent reduction in serum phenylalanine levels relative to baseline [ Time Frame: Baseline to 28 days after last detection of RTX-134 ]
  3. To determine a preliminary dose to achieve serum phenylalanine levels < 600 µmol/L [ Time Frame: Baseline to 28 days after last detection of RTX-134 ]
  4. To determine a preliminary dose to achieve serum phenylalanine levels < 360 µmol/L [ Time Frame: 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. [ Time Frame: Baseline to 28 days after last detection of RTX-134 ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  1. Age 18 years or older with:

    1. A clinical diagnosis of PKU, and
    2. 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)
  2. Stable diet, including medical formula
  3. 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.
  4. Adequate organ function
  5. 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04110496


Contacts
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Contact: Dawn Pinchasik (617) 679-9600 dawn.pinchasik@rubiustx.com

Locations
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United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Sponsors and Collaborators
Rubius Therapeutics

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Responsible Party: Rubius Therapeutics
ClinicalTrials.gov Identifier: NCT04110496     History of Changes
Other Study ID Numbers: RTX-134-01
First Posted: October 1, 2019    Key Record Dates
Last Update Posted: October 1, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Phenylketonurias
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
Metabolic Diseases