Arginase I DeficiencyPhase 3NCT05676853
A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency
A Phase 3 study testing Pegzilarginase in people with Arginase I Deficiency and Hyperargininemia, run by Aeglea Biotherapeutics at 1 site in Canada.
Trial summary
This is an open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Arginase I Deficiency, Hyperargininemia
- What is being tested?
- Pegzilarginase
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteCanada
- Study dates
- 4 Apr 2023to 27 Apr 2023
- Phase
- Phase 3
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Prior enrollment in pegzilarginase clinical studies, with completion of treatment or continued dosing as of the study termination date
- Written informed consent by subject/parent/legal guardian, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol
- Negative pregnancy test within 7 days prior to dosing in childbearing subjects if they are sexually active
- If the subject (male or female) is engaging in sexual activity that could lead to pregnancy, must be surgically sterile, post-menopausal (female), or must agree to use a highly effective method of birth control during the study and for a minimum of 30 days after the last study drug administration. Highly effective methods of contraception include combined (estrogen and progestogen-containing) hormonal contraception associated with inhibition of ovulation, progesterone-only hormonal contraception associated with inhibition of ovulation, intrauterine device, or intrauterine hormone-releasing system
Who cannot join
- Subjects with acute or ongoing hyperammonemia within 6 weeks of the first dose
- Subjects with medical conditions or underlying issues that, in the opinion of the Investigator, would preclude participation in the study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 3 Open-Label Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency
This is an open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.
Read the detailed description
Open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.
- Conditions
- Arginase I Deficiency, Hyperargininemia
- Being tested
- Pegzilarginase
- Keywords
- ARG1-D
- Medical subject headings
- Hyperargininemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PegzilarginaseDrug
Also called Co-ArgI-PEG, AEB1102
Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated
| Study group | Role | What they receive |
|---|---|---|
| Pegzilarginase | Experimental | Weekly subcutaneous dosing of pegzilarginase plus individualized disease management for 52 weeks Receives: Drug: Pegzilarginase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment emergent adverse events
To evaluate the safety of long-term subcutaneous pegzilarginase in subjects with arginase 1 deficiency (ARG1-D)
Measured: Baseline through week 54
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2023
- Study start4 Apr 2023
- Primary completion27 Apr 2023
- Study completion27 Apr 2023
Why it stopped: Sponsor Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Arginase I DeficiencyStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aeglea Biotherapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Cortney Caudill, Study director, Aeglea BioTherapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05676853
- Sponsor's protocol ID
- CAEB1102-305
- Status last verified
- Jul 2023
- First submitted
- 21 Dec 2022
- Last updated
- 27 Jul 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Open-Label Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency
- ClinicalTrials.gov ID
- NCT05676853
- Acronym / sponsor ID
- CAEB1102-305
- Conditions
- Arginase I Deficiency, Hyperargininemia
- Keywords
- ARG1-D
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor Decision
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Aeglea BiotherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 4 Apr 2023 (actual)
- Primary completion
- 27 Apr 2023 (actual)
- Study completion
- 27 Apr 2023 (actual)
- First posted
- 9 Jan 2023 (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 is an open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.
Detailed description
Open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pegzilarginase | Experimental | Drug: PegzilarginaseWeekly subcutaneous dosing of pegzilarginase plus individualized disease management for 52 weeks |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Prior enrollment in pegzilarginase clinical studies, with completion of treatment or continued dosing as of the study termination date
- Written informed consent by subject/parent/legal guardian, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol
- Negative pregnancy test within 7 days prior to dosing in childbearing subjects if they are sexually active
- If the subject (male or female) is engaging in sexual activity that could lead to pregnancy, must be surgically sterile, post-menopausal (female), or must agree to use a highly effective method of birth control during the study and for a minimum of 30 days after the last study drug administration. Highly effective methods of contraception include combined (estrogen and progestogen-containing) hormonal contraception associated with inhibition of ovulation, progesterone-only hormonal contraception associated with inhibition of ovulation, intrauterine device, or intrauterine hormone-releasing system
Key exclusion criteria (2 listed)
- Subjects with acute or ongoing hyperammonemia within 6 weeks of the first dose
- Subjects with medical conditions or underlying issues that, in the opinion of the Investigator, would preclude participation in the study
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 |
|---|---|---|
| PegzilarginaseAlso: Co-ArgI-PEG, AEB1102Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated | Drug | Pegzilarginase |
Primary outcome
Treatment emergent adverse events
Time frame: Baseline through week 54
To evaluate the safety of long-term subcutaneous pegzilarginase in subjects with arginase 1 deficiency (ARG1-D)
Results and safety
The study was stopped early (reason given: Sponsor Decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2023.
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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.
Cortney Caudill study director
Aeglea BioTherapeutics, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Pegzilarginase in people with Arginase I Deficiency and Hyperargininemia, run by Aeglea Biotherapeutics at 1 site in Canada.
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 4 inclusion and 2 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 27 Jul 2023. 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 Canada. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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: Sponsor Decision). 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?
Treatment emergent adverse events (time frame: Baseline through week 54). 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 27 Jul 2023.