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

Phase 2

Recruiting

• Conditions: Gene Mutations; Pseudoxanthoma Elasticum; Arterial Calcification; Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency; Autosomal Recessive Hypophosphatemic Rickets Type 2
• Interventions: Drug: INZ-701

• Registration Number: NCT06462547
• Lead Sponsor: Inozyme Pharma

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.

Detailed Description

The investigational product INZ-701 is being developed as a therapeutic protein for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency and adenosine triphosphate (ATP)-binding cassette subfamily C member 6 (ABCC6) Deficiency. INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin (Ig) G1 antibody.

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.

The study will consist of a 30-day Screening Period, followed by an open-label Treatment Period during which all participants will receive once-weekly subcutaneous (SC) doses of INZ-701 and continue with treatment until INZ-701 is commercially available in the country/region of the participant's residence or until Inozyme chooses to discontinue development of INZ-701. Participants will complete an End of Study (EOS) safety follow-up visit approximately 30 days after their last designated study visit assigned by the Investigator and/or Sponsor.

Study Timeline

• Study First Submitted: 2024-06-12
• Study First Submitted QC: 2024-06-14
• Study First Posted: 2024-06-17
• Study Start: 2024-06-19
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-26
• Last Update Posted: 2024-11-28
• Primary Completion: 2030-11
• Study Completion: 2030-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 200

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
INZ-701	INZ-701	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

Primary Outcome Measures

Name	Time	Method
Number of Treatment Emergent Adverse Events (TEAEs)	6 years (long term safety assessment)	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.
Incidence of Anti-Drug Antibodies (ADA)	6 years (long term safety assessment)	For each subject, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.

Secondary Outcome Measures

Name	Time	Method
Mean Change from Baseline in Plasma PPi Concentration	6 years (long term safety assessment)	For each subject, their plasma PPi concentrations based on a validated assay will be collected and assessed throughout the study, comparing the participant's baseline value over time.
Time to maximum serum concentration (Tmax)	6 years (long term safety assessment)	For each subject, the time to reach maximum concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the participant's baseline value over time.
Maximum Plasma Concentration (Cmax) of INZ-701	6 years (long term safety assessment)	For each subject, the maximum concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the participant's baseline value over time.collected and assessed.

Trial Locations

Locations (5)

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

Clinilabs Drug Development Corporation; 🇺🇸 Eatontown, New Jersey, United States

Necker-Enfants Malades Hospital; 🇫🇷 Paris, France

Universitätsklinikum Hamburg-Eppendorf (UKE); 🇩🇪 Hamburg, Germany

VCTC; 🇬🇧 Oxford, United Kingdom