PhenylketonuriasPhase 1NCT04110496
Safety and Tolerability of RTX-134 in Adults With Phenylketonuria
A Phase 1 study testing RTX-134 in people with Phenylketonurias, run by Rubius Therapeutics at 1 site in United States.
Trial summary
This study will evaluate the safety and tolerability of RTX-134 in adult patients with PKU. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- RTX-134
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Jan 2020to 1 Dec 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital Colorado
Aurora, Colorado, 80045
About this study
Official title: A Phase 1b Open-Label Single Dose Safety, Tolerability, and Pharmacokinetics Study of RTX-134 in Adults With Phenylketonuria
This study will evaluate the safety and tolerability of RTX-134 in adult patients with PKU.
Read the 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).
- Conditions
- Phenylketonurias
- Being tested
- RTX-134
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RTX-134Drug
RTX-134 is a cellular therapy containing AvPAL
| Study group | Role | What they receive |
|---|---|---|
| RTX-134 | Experimental | Escalating doses of RTX-134 will be administered by intravenous infusion one time Receives: Drug: RTX-134 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerability of RTX-134 as measured by frequency of treatment emergent adverse events
Measured: Baseline to 28 days after last detection of RTX-134
To correlate dose with percent reduction in serum phenylalanine levels relative to baseline
Measured: Baseline to 28 days after last detection of RTX-134
To determine a preliminary dose to achieve serum phenylalanine levels < 600 µmol/L
Measured: Baseline to 28 days after last detection of RTX-134
To determine a preliminary dose to achieve serum phenylalanine levels < 360 µmol/L
Measured: 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.
Measured: Baseline to 28 days after last detection of RTX-134
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2019
- Study start29 Jan 2020
- Primary completion1 Dec 2022
- Study completion1 Dec 2022
Why it stopped: The trial was discontinued by the Sponsor
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriasStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single Ascending Dose
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rubius Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04110496
- Sponsor's protocol ID
- RTX-134-01
- Status last verified
- Dec 2022
- First submitted
- 23 Sep 2019
- Last updated
- 8 Dec 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b Open-Label Single Dose Safety, Tolerability, and Pharmacokinetics Study of RTX-134 in Adults With Phenylketonuria
- ClinicalTrials.gov ID
- NCT04110496
- Acronym / sponsor ID
- RTX-134-01
- Conditions
- Phenylketonurias
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The trial was discontinued by the Sponsor
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Rubius TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 29 Jan 2020 (actual)
- Primary completion
- 1 Dec 2022 (actual)
- Study completion
- 1 Dec 2022 (actual)
- First posted
- 1 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single Ascending Dose
| Arm | Type | Receives |
|---|---|---|
| RTX-134 | Experimental | Drug: RTX-134Escalating doses of RTX-134 will be administered by intravenous infusion one time |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age 18 years or older with:
- 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
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RTX-134RTX-134 is a cellular therapy containing AvPAL | Drug | RTX-134 |
Primary outcome
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
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
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
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
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
Results and safety
The study was stopped early (reason given: The trial was discontinued by the Sponsor). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RTX-134 in people with Phenylketonurias, run by Rubius Therapeutics at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: The trial was discontinued by the Sponsor). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Dec 2022.