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

November 26, 2024 updated by: Inozyme Pharma

The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

200

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • France
      • Paris, France
        • Recruiting
        • Necker-Enfants Malades Hospital
        • Contact:
  • Germany
      • Hamburg, Germany
        • Recruiting
        • Universitätsklinikum Hamburg-Eppendorf (UKE)
        • Contact:
  • United Kingdom
      • Oxford, United Kingdom
  • United States
    • Minnesota
      • Rochester, Minnesota, United States, 55905
    • New Jersey
      • Eatontown, New Jersey, United States, 07724
        • Recruiting
        • Clinilabs Drug Development Corporation
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Individuals eligible to participate must meet all of the following inclusion criteria:

  1. 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)
  2. Provide assent in accordance with local regulations, if <18 years of age
  3. Male or female, greater than 1 year of age
  4. 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
  5. 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
  6. 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
  7. In the opinion of the Investigator, able to complete all aspects of the study

Individuals who meet any of the following exclusion criteria will not be eligible to participate:

  1. 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
  2. Known intolerance to INZ-701 or any of its excipients
  3. 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
  4. Pregnant, trying to become pregnant, or breastfeeding
  5. Male participants trying to father a child

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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
Drug: INZ-701
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).
Other Names:
  • rhENPP1-Fc

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Treatment Emergent Adverse Events (TEAEs)
Time Frame: 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.
6 years (long term safety assessment)
Incidence of Anti-Drug Antibodies (ADA)
Time Frame: 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.
6 years (long term safety assessment)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to maximum serum concentration (Tmax)
Time Frame: 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.
6 years (long term safety assessment)
Maximum Plasma Concentration (Cmax) of INZ-701
Time Frame: 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.
6 years (long term safety assessment)
Mean Change from Baseline in Plasma PPi Concentration
Time Frame: 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.
6 years (long term safety assessment)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Inozyme Pharma

Investigators

  • Study Director: Kurt Gunter, MD, Inozyme Pharma, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 19, 2024

Primary Completion (Estimated)

November 1, 2030

Study Completion (Estimated)

December 1, 2030

Study Registration Dates

First Submitted

June 12, 2024

First Submitted That Met QC Criteria

June 14, 2024

First Posted (Actual)

June 17, 2024

Study Record Updates

Last Update Posted (Actual)

November 29, 2024

Last Update Submitted That Met QC Criteria

November 26, 2024

Last Verified

November 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • INZ701-304

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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