Arginase I DeficiencyPhase 3NCT03921541
Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency
A Phase 3 study testing Pegzilarginase in people with Arginase I Deficiency and Hyperargininemia, run by Immedica Pharma AB at 32 sites in 7 countries.
Trial summary
CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension; and a follow up visit for final safety… Read more about this study
- Who is it for?
- Ages 2 years and older
- 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?
- Yes, some participants may receive a placebo
- How many people
- 32 participants
- Where
- 32 sitesin 7 countries
- Study dates
- 1 May 2019to 1 Feb 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects are eligible to be included in the study only if all the following criteria apply:
- The subject and/or parent/guardian provides written informed consent/assent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol
- A current diagnosis of ARG1 D as documented in medical records, which must include 1 of the following: elevated plasma arginine levels, a mutation analysis that results in a pathogenic variant, or reduced RBC arginase activity. For entry into this study, subjects must also fulfill the following plasma arginine criteria:
- The average of all measured values of plasma arginine during the screening period prior to the randomization visit (Visit 1, Study Day 1) is ≥ 250 µmol/L
- If a subject is re-screened, the only values that are considered for eligibility assessment are those in the current screening period
- Subjects must be ≥ 2 years of age on the date of informed consent/assent
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- The subject must be assessable for clinically meaningful within-subject change (clinical response) on at least one component of one assessment included in the key secondary/other secondary endpoints. To be considered assessable, the subject must be able to complete the assessment, and must have a baseline deficit in at least one component as defined in the protocol
- Have received documented confirmation from the investigator and/or dietician that the subject can maintain their diet in accordance with dietary information presented in the protocol, ie, can maintain the current level of protein consumption, including natural protein and EAA supplementation
- Subjects receiving ammonia scavenger therapy, anti-epileptic drugs, and/or medications for spasticity (eg, baclofen) must be on a stable dose of the medication for at least 4 weeks prior to randomization and be willing to remain on a stable dose during the double-blind portion and blinded follow-up portions of the study
- Female and male subjects may participate. Female subjects of childbearing potential must have a negative serum pregnancy test during the screening period before receiving the first dose of study treatment, and a negative urine pregnancy test on the day of the first dose, prior to the first dose. If the subject (male or female) is engaging in sexual activity that could lead to pregnancy, must be surgically sterile, postmenopausal (no menses for 12 months without an alternative medical cause or a high FSH level in the postmenopausal range in women not using hormonal contraception or hormonal replacement therapy), 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 (IUD); intrauterine hormone-releasing system (IUS); or abstinence (refraining from heterosexual intercourse during the entire period of risk associated with study treatment).
Who cannot join
- Hyperammonemic episode (defined as an event in which a subject has an ammonia level ≥100 µM with one or more symptoms related to hyperammonemia requiring hospitalization or emergency room management) within the 6 weeks before the first dose of study drug is administered
- Active infection requiring anti-infective therapy within 3 weeks prior to first dose
- Known active infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C
- Extreme mobility deficit, defined as either the inability to be assessed on the GFAQ or a score of 1 on the GFAQ
- Other medical conditions or comorbidities that, in the opinion of the investigator would interfere with study compliance or data interpretation (eg, severe intellectual disability precluding required study assessments)
- Has participated in a previous interventional study with pegzilarginase
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- Has a history of hypersensitivity to polyethylene glycol (PEG) that, in the judgment of the investigator, puts the subject at unacceptable risk for adverse events
- Subject is being treated with botulinum toxin-containing regimens or plans to initiate such regimens during the double-blind or blinded follow-up portions of the study or received surgical or botulinum-toxin treatment for spasticity-related complications within the 16 weeks prior to the first dose of study treatment in this study
- Is currently participating in another therapeutic clinical trial or has received any investigational agent within 30 days (or 5 half-lives whichever is longer) prior to the first dose of study treatment in this study
- Previous liver or hematopoietic transplant procedure.
Trial locations and contacts
32 sites in 7 countries.
32 sites in 7 countries
United States17 sites
Emory University
Atlanta, Georgia, 30322
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
UT Southwestern Medical Center
Dallas, Texas, 75390
Duke University Medical Center
Durham, North Carolina, 27710
University of Florida College of Medicine
Gainesville, Florida, 32610
University of Texas Health Science Center Medical School at Houston
Houston, Texas, 77030
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Children's Hospital of Orange County
Orange, California, 92868
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224
Cohen Children's Medical Center (Northwell Health)
Queens, New York, 11040
Harvey Pediatrics
Rogers, Arkansas, 72758
University of Utah Hospitals & Clinics
Salt Lake City, Utah, 84108
Seattle Children's Hospital
Seattle, Washington, 98105
Stanford University School of Medicine
Stanford, California, 94305
Children's National Medical Center
Washington D.C., District of Columbia, 20010
United Kingdom5 sites
Birmingham Children's Hospital
Birmingham
University Hospital of Wales
Cardiff
Great Ormond Street Hospital for Children
London
Willink Biochemical Genetics Unit
Manchester
Salford Royal
Salford
Italy3 sites
Fondazione MBBM
Monza
Ospedale Pediatrico Bambino Gesù
Roma
Azienda Ospedaliera Città della Salute e della Scienza di Torino
Torino
Austria2 sites
LKH Bregenz
Bregenz
Medizinische Universität Innsbruck
Innsbruck
France2 sites
Hôpital Necker - Enfants Malades
Paris
Hopital des Enfants
Talence
Germany2 sites
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz
Mainz, Rhineland-Palatinate
Universitaetsklinikum Muenster
Münster, North Rhine-Westphalia
About this study
Official title: PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints): A Randomized, Double-blind, Placebo-controlled Phase 3 Study of the Efficacy and Safety of Pegzilarginase in Children and Adults With Arginase 1 Deficiency
CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension; and a follow up visit for final safety assessments.
Read the detailed description
CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension; and a follow up visit for final safety assessments.
Subjects will be randomized to treatment following completion of all screening assessments and confirmation of study eligibility in a 2:1 ratio to receive weekly IV infusions of pegzilarginase plus individualized disease management (IDM) or placebo plus IDM during the 24-week double blind treatment period. After completion of the 24-week double-blind treatment period, each subject will enter the long term, open-label extension, the first 8 weeks of which are blinded. During the long-term extension, all subjects receive pegzilarginase plus IDM. After 8 weeks of the LTE study, patients have the option to receive treatment by subcutaneous administration (SC).
- Conditions
- Arginase I Deficiency, Hyperargininemia
- Being tested
- Pegzilarginase, Placebo
- 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
PlaceboDrug
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 IV infusions of pegzilarginase plus individualized disease management for 24 weeks Receives: Drug: Pegzilarginase |
| Placebo | Placebo comparator | Weekly IV infusions of placebo plus individualized disease management for 24 weeks Receives: Drug: Placebo |
| Pegzilarginase Long Term Extension | Experimental | After completion of 24 weeks DB treatment, weekly IV infusions of pegzilarginase plus individualized disease management for an additional 150 weeks, with the option to receive treatment by SC after 8 weeks of the LTE study. Receives: Drug: Pegzilarginase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Plasma Arginine Concentration After 24 Weeks of Treatment
The primary analysis will test the change in the level of plasma arginine between baseline and completion of week 24 assessments. It will compare the change from baseline in plasma arginine between participants treated with pegzilarginase and those treated with placebo.
Measured: Baseline through Week 24
Secondary outcomes (17)
Mean Change From Baseline in the Mobility Assessments of the Key Secondary Outcome Measure of the 2 Minute Walk Test
The Key Secondary outcome measure is the mean change from baseline in the 2 Minute Walk Test.
Measured: Baseline through Week 24
Mean Change From Baseline in the Mobility Assessments of the Key Secondary Outcome Measure of GMFM-E
The Key Secondary outcome measure is the mean change from baseline in GMFM-E
The Gross Motor Function Measure (GMFM) utilize a 4-point scoring system for each item across dimensions A-E. GMFM-E assesses walking, running, and jumping. The minimum score for GMFM-E is 0; the maximum score is 72, with a higher score representing better gross motor function
Measured: Baseline through Week 24
Proportion of Participants With Plasma Arginine Levels Below Target Guidance
Proportion of participants with plasma arginine levels below 200umol/L (target level set in disease management guidelines) after 24 weeks of treatment.
Measured: Baseline and week 24
Proportion of Participants With Plasma Arginine Levels in Normal Range
Proportion of participants with plasma arginine levels between 40 - 115 umol/L (normal range for plasma arginine) after 24 weeks.
Measured: Baseline to Week 24
Change in Ornithine
This analysis will measure the change from baseline in the level of ornithine after 24 weeks of treatment.
Measured: Baseline and week 24
Change in Guanidino Compound-ARGA
This analysis will measure the change from baseline in the level of ARGA after 24 weeks of treatment.
Measured: Baseline to week 24
Change in Guanidino Compound - GAA
This analysis will measure the change from baseline in the level of GAA compound after 24 weeks of treatment.
Measured: Baseline to week 24
Change in Guanidino Compound - GVA
This analysis will measure the change from baseline in the level of GVA compound after 24 weeks of treatment.
Measured: Baseline to week 24
Change in Guanidino Compound - NAArg
This analysis will measure the change from baseline in the level of NAArg compound after 24 weeks of treatment.
Measured: Baseline to week 24
Mean Change From Baseline at Week 24 in Other Aspects of Mobility Assessed by GMFM-D
To compare pegzilarginase with placebo with respect to other aspects of mobility.
The Gross Motor Function Measure (GMFM) utilize a 4-point scoring system for each item across dimensions A-E. GMFM-D assesses tasks related to standing. The minimum score for GMFM-D is 0; the maximum score is 39 with a higher score representing better gross motor function
Measured: Baseline to week 24
Mean Change From Baseline at Week 24 in Other Aspects of Mobility Assessed by the Functional Mobility Scale (FMS)
To compare pegzilarginase with placebo with respect to other aspects of mobility utilizing the FMS-5 Score.
The functional mobility scale (FMS) is a 6-point scale from Level 1 (uses wheelchair, stroller, scooter, shopping cart, wagon, or is carried OR walks for exercise only with highly specialized/ supportive walker OR does limited stepping with substantial support/assistance from another person) to Level 6 (independent walking and running on all surfaces without assistive devices or help from another person) that assesses the need for assistive devices for walks of 3 different lengths: 5 meters, 50 meters, and 500 meters.
Measured: Baseline to week 24
Mean Change From Baseline at Week 24 in Other Aspects of Mobility Assessed by the Functional Mobility Scale (FMS)
To compare pegzilarginase with placebo with respect to other aspects of mobility utilizing the FMS-50 Score.
The functional mobility scale (FMS) is a 6-point scale from Level 1 (uses wheelchair, stroller, scooter, shopping cart, wagon, or is carried OR walks for exercise only with highly specialized/ supportive walker OR does limited stepping with substantial support/assistance from another person) to Level 6 (independent walking and running on all surfaces without assistive devices or help from another person) that assesses the need for assistive devices for walks of 3 different lengths: 5 meters, 50 meters, and 500 meters.
Measured: Baseline to week 24
Mean Change From Baseline at Week 24 in Other Aspects of Mobility Assessed by the Functional Mobility Scale (FMS)
To compare pegzilarginase with placebo with respect to other aspects of mobility utilizing the FMS-500 Score.
The functional mobility scale (FMS) is a 6-point scale from Level 1 (uses wheelchair, stroller, scooter, shopping cart, wagon, or is carried OR walks for exercise only with highly specialized/ supportive walker OR does limited stepping with substantial support/assistance from another person) to Level 6 (independent walking and running on all surfaces without assistive devices or help from another person) that assesses the need for assistive devices for walks of 3 different lengths: 5 meters, 50 meters, and 500 meters.
Measured: Baseline to week 24
Mean Change From Baseline at Week 24 in Other Aspects of Mobility Assessed by the Gillette Functional Assessment Questionnaire (GFAQ)
To compare pegzilarginase with placebo with respect to other aspects of mobility.
The Gillette Functional Assessment Questionnaire (GFAQ) is a parent/caregiver assessment consisting of a single question describing a child's ability to walk using a 10-point scale from Level 1 (cannot take any steps at all) to Level 10 (walks, runs, and climbs on uneven terrain and does stairs without difficulty or assistance; is typically able to keep up with peers).
Measured: Baseline to week 24
Mean Change From Baseline at Week 24 in Adaptive Behavior Assessed Using the Vineland Adaptive Behavior Scales (VABS)-II
To compare pegzilarginase with placebo with respect to adaptive behavior.
The Vineland Adaptive Behavior Scales (VABS-II) is a scale designed to measure adaptive behavior of individuals from birth to age 90 years. The VABS-II contains 4 domains: communication, daily living skills, socialization, and motor skills. The domains are made up of 11 subdomains in which the scores are added to form the domain composite scores. The 4 domain composite scores then combine to form the adaptive behavior composite for those individuals aged birth to 6 years 11 months. Three domain composite scores (communication, daily living skills, and socialization) combine to form the adaptive behavior composite for those ages 7 through 90 years. The VABS-II scoring system describes adequate adaptive behavior by subdomain as 13 to 17 and 86 to 114 for the composite score, with higher scores indicating better adaptive functioning.
Measured: Baseline to week 24
Evaluate Safety of Pegzilarginase
Number of participants developing treatment related adverse events.
Measured: Reporting will be from signing consent through follow-up (Baseline to Week 24)
Evaluate Immunogenicity of Pegzilarginase
The proportion of participants who develop (ADA) anti-drug antibodies to pegzilarginase will be measured over the period of the clinical trial.
Measured: Baseline to week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2019
- Study start1 May 2019
- Primary completion1 Feb 2023
- Study completion1 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Immedica Pharma AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Aeglea Biotherapeutics
- Responsible party
- Sponsor
- Investigators
- Mattias Rudebeck, PhD MSc BMedSc, Study director, Immedica Pharma AB
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03921541
- Sponsor's protocol ID
- CAEB1102-300A
- Status last verified
- Aug 2026
- First submitted
- 9 Apr 2019
- Last updated
- 21 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Russo RS, Gasperini S, Bubb G, Neuman L, Sloan LS, Diaz GA, Enns GM; PEACE Investigators. Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE): a phase 3, randomized, double-blind, placebo-controlled, multi-centre trial. EClinicalMedicine. 2024 Jan 12;68:102405. doi: 10.1016/j.eclinm.2023.102405. eCollection 2024 Feb.
PubMed 38292042
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints): A Randomized, Double-blind, Placebo-controlled Phase 3 Study of the Efficacy and Safety of Pegzilarginase in Children and Adults With Arginase 1 Deficiency
- ClinicalTrials.gov ID
- NCT03921541
- Acronym / sponsor ID
- CAEB1102-300A
- Conditions
- Arginase I Deficiency, Hyperargininemia
- Keywords
- ARG1-D
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Immedica Pharma ABIndustry (pharma, biotech, devices)
- Collaborators
- Aeglea Biotherapeutics
- Study start
- 1 May 2019 (actual)
- Primary completion
- 1 Feb 2023 (actual)
- Study completion
- 1 Feb 2023 (actual)
- First posted
- 19 Apr 2019 (actual)
- Results
- Posted 19 Nov 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension; and a follow up visit for final safety assessments.
Detailed description
CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension; and a follow up visit for final safety assessments.
Subjects will be randomized to treatment following completion of all screening assessments and confirmation of study eligibility in a 2:1 ratio to receive weekly IV infusions of pegzilarginase plus individualized disease management (IDM) or placebo plus IDM during the 24-week double blind treatment period. After completion of the 24-week double-blind treatment period, each subject will enter the long term, open-label extension, the first 8 weeks of which are blinded. During the long-term extension, all subjects receive pegzilarginase plus IDM. After 8 weeks of the LTE study, patients have the option to receive treatment by subcutaneous administration (SC).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Pegzilarginase | Experimental | Drug: PegzilarginaseWeekly IV infusions of pegzilarginase plus individualized disease management for 24 weeks |
| Placebo | Placebo comparator | Drug: PlaceboWeekly IV infusions of placebo plus individualized disease management for 24 weeks |
| Pegzilarginase Long Term Extension | Experimental | Drug: PegzilarginaseAfter completion of 24 weeks DB treatment, weekly IV infusions of pegzilarginase plus individualized disease management for an additional 150 weeks, with the option to receive treatment by SC after 8 weeks of the LTE study. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Subjects are eligible to be included in the study only if all the following criteria apply:
- The subject and/or parent/guardian provides written informed consent/assent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol
- A current diagnosis of ARG1 D as documented in medical records, which must include 1 of the following: elevated plasma arginine levels, a mutation analysis that results in a pathogenic variant, or reduced RBC arginase activity. For entry into this study, subjects must also fulfill the following plasma arginine criteria:
- Subjects must be ≥ 2 years of age on the date of informed consent/assent
- The subject must be assessable for clinically meaningful within-subject change (clinical response) on at least one component of one assessment included in the key secondary/other secondary endpoints. To be considered assessable, the subject must be able to complete the assessment, and must have a baseline deficit in at least one component as defined in the protocol
Key exclusion criteria (10 listed)
- Hyperammonemic episode (defined as an event in which a subject has an ammonia level ≥100 µM with one or more symptoms related to hyperammonemia requiring hospitalization or emergency room management) within the 6 weeks before the first dose of study drug is administered
- Active infection requiring anti-infective therapy within 3 weeks prior to first dose
- Known active infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C
- Extreme mobility deficit, defined as either the inability to be assessed on the GFAQ or a score of 1 on the GFAQ
- Other medical conditions or comorbidities that, in the opinion of the investigator would interfere with study compliance or data interpretation (eg, severe intellectual disability precluding required study assessments)
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, Pegzilarginase Long Term Extension |
| PlaceboIndividualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated | Drug | Placebo |
Primary outcome
Change From Baseline in Plasma Arginine Concentration After 24 Weeks of Treatment
Time frame: Baseline through Week 24
The primary analysis will test the change in the level of plasma arginine between baseline and completion of week 24 assessments. It will compare the change from baseline in plasma arginine between participants treated with pegzilarginase and those treated with placebo.
17 secondary outcomes
Mean Change From Baseline in the Mobility Assessments of the Key Secondary Outcome Measure of the 2 Minute Walk Test
Time frame: Baseline through Week 24
Mean Change From Baseline in the Mobility Assessments of the Key Secondary Outcome Measure of GMFM-E
Time frame: Baseline through Week 24
Proportion of Participants With Plasma Arginine Levels Below Target Guidance
Time frame: Baseline and week 24
Proportion of Participants With Plasma Arginine Levels in Normal Range
Time frame: Baseline to Week 24
Change in Ornithine
Time frame: Baseline and week 24
Change in Guanidino Compound-ARGA
Time frame: Baseline to week 24
Change in Guanidino Compound - GAA
Time frame: Baseline to week 24
Change in Guanidino Compound - GVA
Time frame: Baseline to week 24
Change in Guanidino Compound - NAArg
Time frame: Baseline to week 24
Mean Change From Baseline at Week 24 in Other Aspects of Mobility Assessed by GMFM-D
Time frame: Baseline to week 24
Mean Change From Baseline at Week 24 in Other Aspects of Mobility Assessed by the Functional Mobility Scale (FMS)
Time frame: Baseline to week 24
Mean Change From Baseline at Week 24 in Other Aspects of Mobility Assessed by the Functional Mobility Scale (FMS)
Time frame: Baseline to week 24
Mean Change From Baseline at Week 24 in Other Aspects of Mobility Assessed by the Functional Mobility Scale (FMS)
Time frame: Baseline to week 24
Mean Change From Baseline at Week 24 in Other Aspects of Mobility Assessed by the Gillette Functional Assessment Questionnaire (GFAQ)
Time frame: Baseline to week 24
Mean Change From Baseline at Week 24 in Adaptive Behavior Assessed Using the Vineland Adaptive Behavior Scales (VABS)-II
Time frame: Baseline to week 24
Evaluate Safety of Pegzilarginase
Time frame: Reporting will be from signing consent through follow-up (Baseline to Week 24)
Evaluate Immunogenicity of Pegzilarginase
Time frame: Baseline to week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Aug 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38292042Russo RS, Gasperini S, Bubb G, Neuman L, Sloan LS, Diaz GA, Enns GM; PEACE Investigators. Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE): a phase 3, randomized, double-blind, placebo-controlled, multi-centre trial. EClinicalMedicine. 2024 Jan 12;68:102405. doi: 10.1016/j.eclinm.2023.102405. eCollection 2024 Feb.
- 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.
Mattias Rudebeck, PhD MSc BMedSc study director
Immedica Pharma AB
32 sites in 7 countries: 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 Immedica Pharma AB at 32 sites in 7 countries.
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 10 inclusion and 10 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: finished, as of the sponsor's last update on 21 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, United Kingdom, Italy, Austria, France and 2 more countries. 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 19 Nov 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline in Plasma Arginine Concentration After 24 Weeks of Treatment (time frame: Baseline through Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03921541, which also lists 1 linked publication.
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 21 Aug 2026.