Arginase I DeficiencyPhase 2NCT03378531
A Study of AEB1102 (Pegzilarginase) in Patients With Arginase 1 Deficiency
A Phase 2 study testing AEB1102 in people with Arginase I Deficiency and Hyperargininemia, run by Immedica Pharma AB at 7 sites in 4 countries.
Trial summary
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. 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?
- AEB1102
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 7 sitesin 4 countries
- Study dates
- 7 Dec 2017to 15 Dec 2022
- Phase
- Phase 2
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
- Complete treatment in Study CAEB1102-101A without experiencing any clinically significant adverse event or other unmanageable drug toxicity that would preclude continued dosing
- Confirmation by the Investigator and the Sponsor determine that it is acceptable for the patient to continue dosing with AEB1102
- If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment
- If sexually active (male or female), must be surgically sterile, post-menopausal (female), or must agree to use a physician-approved method of birth control during the study and for a minimum of 30 days after the last study drug administration
- Patient or legal guardian is able and willing to provide written informed consent and where required assent, and to comply with all requirements of study participation (including all study procedures and continuation of prescribed diet without modification), prior to any screening procedures
Who cannot join
- 1. Clinically significant concurrent disease, serious intercurrent illness, or other extenuating circumstances
Trial locations and contacts
7 sites in 4 countries.
7 sites in 4 countries
United States4 sites
UTSW
Dallas, Texas, 75390
University of Florida
Gainsville, Florida, 32610
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Stanford University School of Medicine
Stanford, California, 94305
About this study
Official title: An Open-label, Multicentre Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of AEB1102 in Patients With Arginase 1 Deficiency
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.
Read the detailed description
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.
- Conditions
- Arginase I Deficiency, Hyperargininemia
- Being tested
- AEB1102
- Medical subject headings
- Hyperargininemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AEB1102Drug
Also called Co-ArgI-PEG, Pegzilarginase
modified human arginase I
| Study group | Role | What they receive |
|---|---|---|
| pegzilarginase (AEB1102) | Experimental | Each patient may receive pegzilarginase administered IV or SC for up to 4 years. Receives: Drug: AEB1102 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-related Adverse Events
Measured: From start of study up to 262 weeks
Number of Participants With Anti-pegzilarginase and Anti-PEG Antibodies
Measured: From start of study up to 262 weeks
Secondary outcomes (14)
Plasma Arginine Levels
Change in plasma arginine concentrations from study Baseline
Measured: From start of study up to 240 weeks
Plasma GC Levels (Argininic Acid)
Change in plasma argininic acid concentrations from study Baseline
Measured: From start of study up to 240 weeks
Plasma GC Levels (Guanidinoacetic Acid)
Change in plasma guanidinoacetic acid concentrations from study Baseline
Measured: 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
Measured: From start of study up to 240 weeks
Plasma GC Levels (α-N-acetylarginine)
Change in plasma α-N-acetylarginine concentrations from study Baseline
Measured: From start of study up to 240 weeks
Six-Minute Walk Test (6MWT)
Change in 6MWT distance from study Baseline
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From study start up to 262 weeks
Number of Participants With Seizures
Measured: From study start up to 262 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2017
- Study start7 Dec 2017
- Primary completion15 Dec 2022
- Study completion15 Dec 2022
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 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
- NCT03378531
- Sponsor's protocol ID
- CAEB1102-102A
- Status last verified
- Aug 2026
- First submitted
- 11 Dec 2017
- Last updated
- 24 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Diaz GA, Schulze A, McNutt MC, Leao-Teles E, Merritt JL 2nd, Enns GM, Batzios S, Bannick A, Zori RT, Sloan LS, Potts SL, Bubb G, Quinn AG. Clinical effect and safety profile of pegzilarginase in patients with arginase 1 deficiency. J Inherit Metab Dis. 2021 Jul;44(4):847-856. doi: 10.1002/jimd.12343. Epub 2021 Jan 26.
PubMed 33325055