Trials Sponsors Conditions Drugs Pricing

Conditions

Arginase 1 deficiency (ARG1-D)Arginase 1 Deficiency

Outcome Measures

Primary Outcomes (1)

Change From Baseline in Plasma Arginine Concentrations in Subjects <24 Months of Age With Arginase 1 Deficiency (ARG1-D).
To evaluate the effect of pegzilarginase on plasma arginine concentrations in subjects \<24 months of age with arginase 1 deficiency (ARG1-D).
From baseline up to 12 weeks.

Secondary Outcomes (7)

Pharmacokinetic (PK) Profile of Pegzilarginase: Half-life (T½).
From baseline up to 12 weeks. Within 1 hour pre-dose a sample was taken on visits 1, 2, 4, 6, 8, 10, and 13. A post-dose sample was taken 12 - 48 hours after dosing on visits 2, 4, and 10.
Pharmacokinetic (PK) Profile of Pegzilarginase: Maximum Observed Concentration (Tmax).
From baseline up to 12 weeks. Within 1 hour pre-dose a sample was taken on visits 1, 2, 4, 6, 8, 10, and 13. A post-dose sample was taken 12 - 48 hours after dosing on visits 2, 4, and 10.
Pharmacokinetic (PK) Profile of Pegzilarginase: Maximum Observed Concentration (Cmax).
From baseline up to 12 weeks. Within 1 hour pre-dose a sample was taken on visits 1, 2, 4, 6, 8, 10, and 13. A post-dose sample was taken 12 - 48 hours after dosing on visits 2, 4, and 10.
Pharmacokinetic (PK) Profile of Pegzilarginase: Area Under the Plasma Drug Concentration-time Curve.
From baseline up to 12 weeks. Within 1 hour pre-dose a sample was taken on visits 1, 2, 4, 6, 8, 10, and 13. A post-dose sample was taken 12 - 48 hours after dosing on visits 2, 4, and 10.
Pharmacodynamic (PD) Response of Pegzilarginase: Anti-drug Antibodies (ADAs).

Study Arms (1)

Weekly subcutaneous (SC) administration of pegzilarginase

EXPERIMENTAL

All subjects will receive a once weekly (QW) SC dose of pegzilarginase for 12 weeks

Drug: Pegzilarginase

Interventions

PegzilarginaseDrug

SC administration of pegzilarginase over 12 weeks in subjects with ARG1-D who are \< 24 months of age

Also known as: Loargys

Weekly subcutaneous (SC) administration of pegzilarginase

Loargys 5 mg/ml solution for injection/infusionType not reported

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age1 Day - 24 Months

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

Subjects must be \< 24 months of age on the date of informed consent
Confirmed diagnosis of ARG1-D documented in medical records by at least 1 of the following methods:

Sponsors & Collaborators

Immedica Pharma AB
Industrylead

Study Sites (3)

Univ. Klinik für Kinder- und Jugendheilkunde Medizinische Universität

Graz, A-8036, Austria

Unidade de Doenças Metabólicas Pediatria, Hospital Santa Maria

Lisbon, Portugal

Bradford Royal Infirmary Duckworth Lane

Bradford, BD9 6RJ, United Kingdom

MeSH Terms

Conditions

Hyperargininemia

Condition Hierarchy (Ancestors)

Urea Cycle Disorders, InbornBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Results Point of Contact

Title: VP Head of Global Integrated Evidence Generation
Organization: Immedica Pharma AB

Study Officials

Mattias Rudebeck, PhD MSc BMedSc
Immedica Pharma AB
STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee: No
Restriction Type: GT60
Restrictive Agreement: Yes

Enrollment

3 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 3
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

August 20, 2024

First Posted

September 3, 2024

Study Start

August 30, 2024

Actual (reported)

Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

Study Start

First Posted

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (7)

Study Arms (1)

Weekly subcutaneous (SC) administration of pegzilarginase

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (3)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source records

Documents

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

Study Start

First Posted

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (7)

Study Arms (1)

Weekly subcutaneous (SC) administration of pegzilarginase

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (3)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source records

Documents

Locations