ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
NCT ID: NCT06462547
Last Updated: 2024-11-29
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
200 participants
INTERVENTIONAL
2024-06-19
2030-12-31
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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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
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).
Interventions
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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 Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
Exclusion Criteria
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
1 Year
ALL
No
Sponsors
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Inozyme Pharma
INDUSTRY
Responsible Party
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Principal Investigators
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Kurt Gunter, MD
Role: STUDY_DIRECTOR
Inozyme Pharma, Inc.
Locations
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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
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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INZ701-304
Identifier Type: -
Identifier Source: org_study_id