ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

NCT06462547 | Recruiting Phase 2 FDA Drug

• 1 other identifier: INZ701-304
• Study Type: interventional
• Target: 200
• Locations: 4 countries
• Sites: 5

Brief Summary

The purpose of this study (Study INZ701-304 \[ADAPT\]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.

Conditions

Gene Mutations; Pseudoxanthoma Elasticum; Arterial Calcification; Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency; Autosomal Recessive Hypophosphatemic Rickets Type 2

Keywords

ABCC6; ATP-Binding Cassette Subfamily C Member 6 Deficiency; PXE; Pseudoxanthoma elasticum; GACI; Generalized Arterial Calcification of Infancy; ARHR2; Autosomal Recessive Hypophosphatemic Rickets Type 2; ENPP1; ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency; hypopyrophosphatemia

Outcome Measures

Primary Outcomes (2)

• Number of Treatment Emergent Adverse Events (TEAEs)

  Treatment-emergent AEs are defined as any AE occurring from the first dose of INZ-701 through 30 days after the last dose of INZ-701.

  6 years (long term safety assessment)

• Incidence of Anti-Drug Antibodies (ADA)

  For each subject, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.

  6 years (long term safety assessment)

Secondary Outcomes (3)

• Time to maximum serum concentration (Tmax)

  6 years (long term safety assessment)

• Maximum Plasma Concentration (Cmax) of INZ-701

  6 years (long term safety assessment)

• Mean Change from Baseline in Plasma PPi Concentration

  6 years (long term safety assessment)

Study Arms (1)

INZ-701

EXPERIMENTAL

INZ-701 will be administered by subcutaneous injection on a once-weekly basis as follows: * Study participants from 1 year to \<13 years of age will receive a dose of 2.4 mg/kg * Study participants ≥13 years will either receive a 1.8 mg/kg dose or a 150 mg flat dose of INZ-701

Drug: INZ-701

Interventions

INZ-701 DRUG

INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody (rhENPP1-Fc).

Also known as: rhENPP1-Fc

INZ-701

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• Provide assent in accordance with local regulations, if \<18 years of age
• Male or female, greater than 1 year of age
• Must have completed the protocol-required safety and PK/PD and/or efficacy period(s) of a previous INZ-701 clinical study in ENPP1 or ABCC6 Deficiency, as confirmed by the Sponsor
• Female participants of childbearing potential who are sexually active must be using or agree to use 1 highly effective form of contraception (per CTFG 2020) from at least 1 month before the first dose of INZ-701 through 30 days after last dose of INZ-701 (greater than 5 half-lives of INZ-701); participants must agree to not donate ova from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701
• Male participants 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; participants must agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701
• In the opinion of the Investigator, able to complete all aspects of the study

You may not qualify if:

• 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
• Known intolerance to INZ-701 or any of its excipients
• 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
• Pregnant, trying to become pregnant, or breastfeeding
• Male participants trying to father a child

Sponsors & Collaborators

• Inozyme Pharma: lead

Study Sites (5)

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Clinilabs Drug Development Corporation

Eatontown, New Jersey, 07724, United States

RECRUITING

Necker-Enfants Malades Hospital

Paris, France

RECRUITING

Universitätsklinikum Hamburg-Eppendorf (UKE)

Hamburg, Germany

RECRUITING

VCTC

Oxford, United Kingdom

RECRUITING

MeSH Terms

Conditions

Pseudoxanthoma Elasticum; Arterial calcification of infancy

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Officials

• Kurt Gunter, MD

  Inozyme Pharma, Inc.

  STUDY DIRECTOR

Central Study Contacts

Inozyme Clinical Trial Information

CONTACT

+1 857 330 4340; clinicaltrials@inozyme.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The ADAPT Study (INZ701-304) is an open-label study to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.

Study Record Dates

• First Submitted: June 12, 2024
• First Posted: June 17, 2024
• Study Start: June 19, 2024
• Primary Completion (Estimated): November 1, 2030
• Study Completion (Estimated): December 1, 2030
• Last Updated: November 29, 2024

Record last verified: 2024-11

Locations

GB (1); US (2); FR (1); DE (1)