The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
NCT ID: NCT05734196
Last Updated: 2025-02-07
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
PHASE1
16 participants
INTERVENTIONAL
2023-06-25
2026-04-01
Brief Summary
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Detailed Description
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Study INZ701-104 (the ENERGY study) is a Phase 1b, open-label study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in infant study participants with ENPP1 Deficiency or ABCC6 Deficiency.
The study will consist of up to a 60-day Screening Period, a 52-week Treatment Period during which study participants will receive INZ-701, an Extension Period during which participants may continue to receive INZ-701 until it is commercially available in the country where the participant resides, or until an alternative study of INZ-701 is available, and an End of Treatment (EOT) visit 30 days after the last dose of INZ-701. Upon treatment discontinuation, participants will continue to be followed for their ongoing disposition for survival outcome at least quarterly through the end of the study.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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INZ-701
The first 2 study participants will receive 1 dose of 0.2 mg/kg on Day 1 and start at Dose Level A (0.2 mg/kg twice weekly) on Day 8.
The Data Review Committee (DRC) comprised of representatives of the Sponsor, the study Investigators, and a physician who is a subject matter expert not affiliated with the study or Sponsor, will review the safety data of the first study participant through Day 8. Contingent upon this review, the Sponsor will decide if additional study participants can begin receiving INZ-701.
After the second study participant completes Day 32, the DRC will perform a cumulative review of safety and PK/PD data and will make dosing recommendations, for example, modifying the dose of the ongoing study participants and/or changing the starting dose for future participants.
Dose Level A: 0.2 mg/kg twice weekly
Dose Level B: 0.6 mg/kg twice weekly
Dose Level C: 0.2 mg/kg once weekly
Dose Level D: 0.6 mg/kg once weekly
Dose Level E: 1.8 mg/kg once weekly
INZ-701
Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.
Interventions
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INZ-701
Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory
3. Study participants must have clinical manifestations of GACI or GACI-2, which may include, but are not limited to, pathologic ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly
4. Study participant must be male or female from birth to \<1 year of age at Baseline (Day 1)
5. Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
6. In the opinion of the Investigator, the study participant must be able to complete all aspects of the study
7. Study participant's caregiver(s) must agree to provide access to their child's relevant medical records
Exclusion Criteria
2. Care has been withdrawn or study participant is receiving end of life care or hospice only
3. Known malignancy
4. Known intolerance to INZ-701 or any of its excipients
5. Concurrent participation in another non-Inozyme interventional study
6. Receipt of any non-Inozyme investigational new drug within 5 half-lives of the last dose of the other investigational product or within 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device, through completion of participation in the study
1 Year
ALL
No
Sponsors
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Inozyme Pharma
INDUSTRY
Responsible Party
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Principal Investigators
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Alex Lai, MD
Role: STUDY_DIRECTOR
Inozyme Pharma
Locations
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Rady Children's Hospital
San Diego, California, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
The University of Utah
Salt Lake City, Utah, United States
Hospital Sant Joan de Déu
Barcelona, , Spain
Royal Manchester Children's Hospital
Manchester, , 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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INZ701-104
Identifier Type: -
Identifier Source: org_study_id
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