RECOMBINANT HUMAN ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE 1 FUSED TO THE FC FRAGMENT OF IGG1 for Ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency | ABCC6 deficiency | Generalized arterial calcification of infancy (GACI) | Pseudoxanthoma elasticum (PXE)

Inclusion criteria

• {"criterion_text":"- Provide written or electronic informed consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP)"}
• {"criterion_text":"- Provide assent in accordance with local regulations, if <18 years of age"}
• {"criterion_text":"- Male or female, greater than 1 year of age"}
• {"criterion_text":"- Must have completed the protocol-required safety and PK/PD and/or efficacy period(s) of the INZ-701-101, INZ701-201, INZ701-104, or INZ701-106 clinical studies in ENPP1 or ABCC6 Deficiency, as confirmed by the Sponsor"}
• {"criterion_text":"- Women of childbearing potential (WOCBP), as defined in Clinical Trials Coordination Group [CTCG 2024]) must have a negative serum pregnancy test at Screening and must not be breastfeeding"}
• {"criterion_text":"- Males who are sexually active must agree to use condoms from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701"}
• {"criterion_text":"- WOCBP and partners of fertile males who are WOCBP must be using or agree to use a highly effective form of contraception (as per CTCG 2024) from at least 1 month before the first dose of INZ-701 through 30 days after the last dose of INZ-701 (greater than 5 half-lives of INZ-701)"}
• {"criterion_text":"- In the opinion of the Investigator, able to complete all aspects of the study"}

Exclusion criteria

• {"criterion_text":"- In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency or ABCC6 Deficiency, that will preclude study participation and/or may confound interpretation of study results"}
• {"criterion_text":"- Known intolerance to INZ-701 or any of its excipients"}
• {"criterion_text":"- Concurrent participation in another interventional clinical study and/or has received an investigational drug other than INZ-701 within 5 half-lives or within 4 weeks prior to the first dose of INZ-701 in this study, whichever is longer, or use of an investigational device"}
• {"criterion_text":"- Pregnant, trying to become pregnant, or breastfeeding"}
• {"criterion_text":"- Male participants trying to father a child"}

Primary endpoints

• {"endpoint_text":"- Incidence, frequency, and severity of adverse events (AEs), treatment-emergent adverse event (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Changes in clinical laboratory tests including chemistry, hematology, and urinalysis resulting in an AE","definition_or_measurement_approach":""}
• {"endpoint_text":"- Concomitant medications","definition_or_measurement_approach":""}
• {"endpoint_text":"- Titers of anti-drug antibodies (ADA)","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- INZ-701 plasma PK parameters measured by immunoassay and enzymatic activity","definition_or_measurement_approach":"Plasma PK parameters measured by immunoassay and enzymatic activity"}
• {"endpoint_text":"- Plasma PPi concentrations based on a validated assay","definition_or_measurement_approach":"Plasma PPi concentrations measured using a validated assay"}

Methods

• K1 Recruitment arrangements document submitted (recruitment arrangements documents present for Germany and France)
• Patient-facing materials and outreach: Patient welcome letters, Patient and Caregiver Privacy Notice, Subject ID Card, Expense Claim Form, Payment Card Letter (materials present in DE/FR/EN and other translations)
• Use of home nursing and home administration support (third party duties: MD Group listed for Home nursing, participant travel reimbursement, participant stipends)
• Site-to-patient study drug deliveries (Marken listed for site-to-patient study drug deliveries)
• Use of central and remote processes: Central ethics board for USA (Advarra), remote monitoring and site/vendor management (Kps Life LLC)

Germany

Earliest CTIS Part Ii Submission Date

12-09-2024

Latest Decision Or Authorization Date

14-08-2025

Processing Time Days

336

Number Of Sites

2

Number Of Participants

7

France

Earliest CTIS Part Ii Submission Date

27-09-2024

Latest Decision Or Authorization Date

14-08-2025

Processing Time Days

321

Number Of Sites

1

Number Of Participants

7

Primary sponsor

Full Name

Inozyme Pharma Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

PAREXEL International GmbH

Responsibilities

Early Phase Clinical Unit operations / site support (listed as a trial site organization)

Name

Phlexglobal

Responsibilities

eTMF Management

Name

Medrio Inc.

Responsibilities

Electronic data capture / data management (sponsor duties code 7)

Name

Marken

Responsibilities

Site-to-patient study drug deliveries

Third parties

• {"country":"France","full_name":"Voisin Consulting Life Sciences","duties_or_roles":"Sponsor duties codes: 12, 8","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Labconnect LLC","duties_or_roles":"Lab kit build; sponsor duties code 4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Llx Solutions LLC","duties_or_roles":"Sponsor duties codes: 10, 6","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Advarra Inc.","duties_or_roles":"Central Ethics Board for the USA","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Phlexglobal","duties_or_roles":"eTMF Management","organisation_type":"Industry"}
• {"country":"Germany","full_name":"Catalent Germany Schorndorf GmbH","duties_or_roles":"Depot/Clinical Supplies","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"MD Group","duties_or_roles":"Home nursing, participant travel reimbursement, participant stipends","organisation_type":"Health care"}
• {"country":"United States","full_name":"Kps Life LLC","duties_or_roles":"Sponsor duties include code 1; Remote monitoring, site and vendor management","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Medrio Inc.","duties_or_roles":"Sponsor duties code 7","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Marken","duties_or_roles":"Site-to-patient study drug deliveries","organisation_type":"Industry"}
• {"country":"United States","full_name":"Resolian Bioanalytics","duties_or_roles":"Sponsor duties code 4","organisation_type":"Industry"}