A Phase 2 interventional study of AEB1102 in Arginase I Deficiency and Hyperargininemia, sponsored by Immedica Pharma AB. Completed at 7 sites in 4 countries. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2026-09-24.
Sponsored by Immedica Pharma AB · Phase 2, Interventional, and Treatment
The purpose of this study is to determine the safety, tolerability, and efficacy of long-term intravenous and subcutaneous administration of pegzilarginase for the treatment of pediatric and adult patients with ARG1-D who complete Study CAEB1102-101A.
Purpose of this study is to determine the safety, tolerability, and efficacy of long-term intravenous and subcutaneous administration of pegzilarginase for the treatment of pediatric and adult patients with ARG1-D who complete Study CAEB1102-101A.
Immedica Pharma AB is the lead sponsor of 8 studies on the registry; 1 is open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 3 (50%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
1. concurrent disease, serious intercurrent illness, or other extenuating circumstances
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Each patient may receive pegzilarginase administered IV or SC for up to 4 years.
Drug: AEB1102
modified human arginase I
Also known as: Co-ArgI-PEG, Pegzilarginase
Number of Participants With Treatment-related Adverse Events
Time frame: From start of study up to 262 weeks
Number of Participants With Anti-pegzilarginase and Anti-PEG Antibodies
Time frame: From start of study up to 262 weeks
Plasma Arginine Levels
Change in plasma arginine concentrations from study Baseline
Time frame: From start of study up to 240 weeks
Plasma GC Levels (Argininic Acid)
Change in plasma argininic acid concentrations from study Baseline
Time frame: From start of study up to 240 weeks
Plasma GC Levels (Guanidinoacetic Acid)
Change in plasma guanidinoacetic acid concentrations from study Baseline
Time frame: From start of study up to 240 weeks
Plasma GC Levels (α-keto-δ-guanidinovaleric Acid)
Change in plasma α-keto-δ-guanidinovaleric acid concentrations from study Baseline
Time frame: From start of study up to 240 weeks
Plasma GC Levels (α-N-acetylarginine)
Change in plasma α-N-acetylarginine concentrations from study Baseline
Time frame: From start of study up to 240 weeks
Six-Minute Walk Test (6MWT)
Change in 6MWT distance from study Baseline
Time frame: From start of study up to 262 weeks
Gross Motor Function Measure (GMFM) Part D
Change from study Baseline. The GMFM-D evaluates the patient's ability to stand via assessment of 13 activities, each of which is scored on a scale of 0 to 3, with a scale range of 0-39 points. An increase in GMFM-D score indicates improvement in standing.
Time frame: From start of study up to 262 weeks
Gross Motor Function Measure (GMFM) Part E
Change from study Baseline. The GMFM-E evaluates a subject's ability to walk, run, and jump via assessment of 24 activities, each of which is scored on a scale of 0 to 3, , with a scale range of 0-72. A higher GMFM-E score indicates greater ability, with increasing scores indicating improvement in walking, running, and jumping.
Time frame: From start of study up to 262 weeks
Modified Ashworth Scale (MAS)
Spasticity improvement for lower body muscle groups based on MAS from study Baseline. The MAS is used to measure the resistance to passive movement about a joint due to spasticity using a 6-point scoring scale with possible values of 0, 1, 1+ (scored as 1.5), 2, 3, and 4, where 0 is no increase in muscle tone (no spasticity) and 4 is affected part(s) rigid in flexion or extension (total rigidity). Thus, a decrease in MAS score indicates improvement in spasticity.
Time frame: From start of study up to 262 weeks
Berg Balance Scale (BBS)
Change from study Baseline. The BBS, a measure of balance, utilizes a 5-point scoring scale of 0 (indicating the lowest level of function) to 4 (indicating the highest level of function) for 14 tasks, giving a scale range of 0-56, with a higher score representing better balance.
Time frame: From start of study up to 262 weeks
Purdue Pegboard Test (Dominant Hand)
Change from study Baseline. The Purdue Pegboard Test evaluates manipulative dexterity and bimanual coordination, measuring performance speed in a fine motor task via gross movements of the arms, hands, and fingers, as well as fine motor extremity dexterity. A higher Purdue Pegboard Test score indicates better dexterity and coordination. Scores were compared to normative data, compiled from healthy individuals, to produce a Z-score (0 being population mean). A positive Z-score indicates the raw score is higher than the mean average; a negative Z-score indicates the raw score is below the mean average.
Time frame: From start of study up to 262 weeks
Purdue Pegboard Test (Non-dominant Hand)
Change from study Baseline. The Purdue Pegboard Test evaluates manipulative dexterity and bimanual coordination, measuring performance speed in a fine motor task via gross movements of the arms, hands, and fingers, as well as fine motor extremity dexterity. A higher Purdue Pegboard Test score indicates better dexterity and coordination. Scores were compared to normative data, compiled from healthy individuals, to produce a Z-score (0 being population mean). A positive Z-score indicates the raw score is higher than the mean average; a negative Z-score indicates the raw score is below the mean average.
Time frame: From start of study up to 262 weeks
Number of Participants With Hyperammonemic Episodes
Hyperammonemic episodes" are defined as either an AE for which an Investigator observed symptoms considered to be consistent with a "hyperammonemic episode", or an AE in a patient meeting all 3 of the following criteria: 1. Confirmed ammonia level ≥100 μM, 2. One or more symptoms related to hyperammonemia, 3. Requiring hospitalization or emergency room management.
Time frame: From study start up to 262 weeks
Number of Participants With Seizures
Time frame: From study start up to 262 weeks
| Milestone | Pegzilarginase (AEB1102) |
|---|---|
| Started | 14 |
| Completed | 13 |
| Not completed | 1 |
| Withdrew: Withdrawal by subject | 1 |
| Participants | Pegzilarginase (AEB1102) |
|---|---|
| Number of Participants With Treatment-related Adverse Events | 7 |
| Participants | Pegzilarginase (AEB1102) |
|---|---|
| Number of Participants With Anti-pegzilarginase and Anti-PEG Antibodies | 0 |
Change in plasma arginine concentrations from study Baseline
| micromolar | Pegzilarginase (AEB1102) |
|---|---|
| Plasma Arginine Levels | -212.3 ± 16.55 |
Change in plasma argininic acid concentrations from study Baseline
| micromolar | Pegzilarginase (AEB1102) |
|---|---|
| Plasma GC Levels (Argininic Acid) | -1.7 ± 0.97 |
Change in plasma guanidinoacetic acid concentrations from study Baseline
| micromolar | Pegzilarginase (AEB1102) |
|---|---|
| Plasma GC Levels (Guanidinoacetic Acid) | 2.4 ± 3.73 |
Change in plasma α-keto-δ-guanidinovaleric acid concentrations from study Baseline
| micromolar | Pegzilarginase (AEB1102) |
|---|---|
| Plasma GC Levels (α-keto-δ-guanidinovaleric Acid) | -1.0 ± 0.54 |
Change in plasma α-N-acetylarginine concentrations from study Baseline
| micromolar | Pegzilarginase (AEB1102) |
|---|---|
| Plasma GC Levels (α-N-acetylarginine) | -0.5 ± 0.13 |
Change in 6MWT distance from study Baseline
| meters | Pegzilarginase (AEB1102) |
|---|---|
| Six-Minute Walk Test (6MWT) | 63.8 ± 87.37 |
Change from study Baseline. The GMFM-D evaluates the patient's ability to stand via assessment of 13 activities, each of which is scored on a scale of 0 to 3, with a scale range of 0-39 points. An increase in GMFM-D score indicates improvement in standing.
| points | Pegzilarginase (AEB1102) |
|---|---|
| Gross Motor Function Measure (GMFM) Part D | 2.3 ± 3.79 |
Change from study Baseline. The GMFM-E evaluates a subject's ability to walk, run, and jump via assessment of 24 activities, each of which is scored on a scale of 0 to 3, , with a scale range of 0-72. A higher GMFM-E score indicates greater ability, with increasing scores indicating improvement in walking, running, and jumping.
| points | Pegzilarginase (AEB1102) |
|---|---|
| Gross Motor Function Measure (GMFM) Part E | 1.1 ± 3.0 |
Spasticity improvement for lower body muscle groups based on MAS from study Baseline. The MAS is used to measure the resistance to passive movement about a joint due to spasticity using a 6-point scoring scale with possible values of 0, 1, 1+ (scored as 1.5), 2, 3, and 4, where 0 is no increase in muscle tone (no spasticity) and 4 is affected part(s) rigid in flexion or extension (total rigidity). Thus, a decrease in MAS score indicates improvement in spasticity.
| Participants | Pegzilarginase (AEB1102) |
|---|---|
| Improvement in spasticity | 5 |
| Worsening in spasticity | 0 |
Change from study Baseline. The BBS, a measure of balance, utilizes a 5-point scoring scale of 0 (indicating the lowest level of function) to 4 (indicating the highest level of function) for 14 tasks, giving a scale range of 0-56, with a higher score representing better balance.
| points | Pegzilarginase (AEB1102) |
|---|---|
| Berg Balance Scale (BBS) | 3.0 ± 5.46 |
Change from study Baseline. The Purdue Pegboard Test evaluates manipulative dexterity and bimanual coordination, measuring performance speed in a fine motor task via gross movements of the arms, hands, and fingers, as well as fine motor extremity dexterity. A higher Purdue Pegboard Test score indicates better dexterity and coordination. Scores were compared to normative data, compiled from healthy individuals, to produce a Z-score (0 being population mean). A positive Z-score indicates the raw score is higher than the mean average; a negative Z-score indicates the raw score is below the mean average.
| Z-score | Pegzilarginase (AEB1102) |
|---|---|
| Purdue Pegboard Test (Dominant Hand) | 0.3 ± 1.71 |
Change from study Baseline. The Purdue Pegboard Test evaluates manipulative dexterity and bimanual coordination, measuring performance speed in a fine motor task via gross movements of the arms, hands, and fingers, as well as fine motor extremity dexterity. A higher Purdue Pegboard Test score indicates better dexterity and coordination. Scores were compared to normative data, compiled from healthy individuals, to produce a Z-score (0 being population mean). A positive Z-score indicates the raw score is higher than the mean average; a negative Z-score indicates the raw score is below the mean average.
| Z-score | Pegzilarginase (AEB1102) |
|---|---|
| Purdue Pegboard Test (Non-dominant Hand) | 0.4 ± 1.2 |
Hyperammonemic episodes" are defined as either an AE for which an Investigator observed symptoms considered to be consistent with a "hyperammonemic episode", or an AE in a patient meeting all 3 of the following criteria: 1. Confirmed ammonia level ≥100 μM, 2. One or more symptoms related to hyperammonemia, 3. Requiring hospitalization or emergency room management.
| Participants | Pegzilarginase (AEB1102) |
|---|---|
| Hyperammonemic episode = Yes | 6 |
| Hyperammonemic episode = No | 8 |
| Participants | Pegzilarginase (AEB1102) |
|---|---|
| Number of Participants With Seizures | 1 |
Collected over From study start up to 262 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pegzilarginase (AEB1102) | 0/14 (0%) | 8/14 (57.1%) | 14/14 (100%) |
| Event | Pegzilarginase (AEB1102) |
|---|---|
| HyperammonemiaMetabolism and nutrition disorders | 5/14 |
| GastroenteritisInfections and infestations | 2/14 |
| Respiratory syncytial virus infectionInfections and infestations | 2/14 |
| InfluenzaInfections and infestations | 1/14 |
| Respiratory tract infectionInfections and infestations | 1/14 |
| Abdominal painGastrointestinal disorders | 1/14 |
| VomitingGastrointestinal disorders | 1/14 |
| Ammonia increasedInvestigations | 1/14 |
| Post-traumatic headacheNervous system disorders | 1/14 |
| Social problemSocial circumstances | 1/14 |
| Event | Pegzilarginase (AEB1102) |
|---|---|
| CoughRespiratory, thoracic and mediastinal disorders | 11/14 |
| VomitingGastrointestinal disorders | 8/14 |
| NasopharyngitisInfections and infestations | 7/14 |
| HeadacheNervous system disorders | 7/14 |
| COVID-19Infections and infestations | 6/14 |
| Abdominal pain upperGastrointestinal disorders | 6/14 |
| NauseaGastrointestinal disorders | 6/14 |
| Ammonia increasedInvestigations | 6/14 |
| HyperammonaemiaMetabolism and nutrition disorders | 6/14 |
| Upper respiratory tract infectionInfections and infestations | 5/14 |
| Age, Continuous(Years) | Pegzilarginase (AEB1102) |
|---|---|
| Mean | 15.7 ± 9.21 |
| Sex: Female, Male(Participants) | Pegzilarginase (AEB1102) |
|---|---|
| Female | 11 |
| Male | 3 |
| Race (NIH/OMB)(Participants) | Pegzilarginase (AEB1102) |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 2 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 10 |
| More than one race | 0 |
| Unknown or Not Reported | 1 |
| Ethnicity (NIH/OMB)(Participants) | Pegzilarginase (AEB1102) |
|---|---|
| Hispanic or Latino | 7 |
| Not Hispanic or Latino | 7 |
| Unknown or Not Reported | 0 |
| Age at initial symptoms(Years) | Pegzilarginase (AEB1102) |
|---|---|
| Mean | 2.7 ± 5.68 |
| Number of seizures in the last year(Seizures) | Pegzilarginase (AEB1102) |
|---|---|
| Mean | 0.4 ± 0.55 |
| Level of spasticity(Participants) | Pegzilarginase (AEB1102) |
|---|---|
| None | 4 |
| Mild | 3 |
| Moderate | 3 |
| Severe | 4 |
| Gross Motor Function Classification System (GMFCS) level(Participants) | Pegzilarginase (AEB1102) |
|---|---|
| Level I | 7 |
| Level II | 4 |
| Level III | 2 |
| Level IV | 1 |
1 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Dec 2017 to primary completion in Dec 2022, 60 months.
Results were first posted in Sep 2026, 3 years 9 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Pegzilarginase
Hyperargininemia