The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency

The ENABLE Study: An Open-Label, Long-Term Safety and Efficacy Study of INZ-701 in Patients With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency

NCT06739980•Inozyme Pharma

View on ClinicalTrials.gov

Summary

The purpose of Study INZ701-108 (ENABLE) is to assess the safety and efficacy of INZ-701 in patients 1 year of age and older with ENPP1 Deficiency who have not previously received INZ-701 and are not eligible for existing Inozyme-sponsored clinical studies that are still open to enrollment.

Detailed Description

INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy in development for the treatment of the ultra-rare genetic disorder, ENPP1 Deficiency. Study INZ701-108 (ENABLE) is an open-label study to assess the long-term safety and efficacy of INZ-701 in patients 1 year of age and older with ENPP1 Deficiency who have not previously received INZ-701 and are not eligible for an Inozyme-sponsored clinical study that is open to recruitment. 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 treatment through 104 weeks. The dose will be determined by the participant's age and weight.

Eligibility

Age

1 - No limit years

Sex

ALL

Healthy Volunteers

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, ≥1 year of age
•4. A confirmed postnatal molecular genetic diagnosis of ENPP1 Deficiency with biallelic variants (ie, homozygous or compound heterozygous) performed using assays that meet CE-marked requirements, or from a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory, or regional equivalent
•5. Must have at least one of the following clinical signs and/or symptoms consistent with ENPP1 Deficiency:
•1. ≥1 atraumatic vertebral fracture
•2. ≥2 fractures as an adult (eg, long bones, digits, vertebrae)
•3. For participants \<55 years of age, low bone mineral density measured at any of the following sites: lumbar spine, radius, and hip (DXA Z-score less than -1.5)
•4. History of myocardial infarction, unstable angina, transient ischemic attack, or low cardiac output before the age of 40 years
•5. Renal vascular hypertension or other evidence for vascular insufficiency/stenosis
+9 more criteria

Exclusion Criteria

•1. In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality not due to ENPP1 Deficiency that may confound interpretation of study results
•2. Eligible for another Inozyme sponsored study of INZ-701 treatment that is open for enrollment
•3. Receiving prohibited medications
•4. Unable or unwilling to discontinue calcitriol or other active forms of vitamin D3 (or analogs) within 7 days prior to Day 1 and/or oral phosphate supplements within 36 hours prior to Day 1
•5. Received previous treatment with INZ-701
•6. Concurrent participation in another interventional clinical study and/or has received an investigational drug within 5 half-lives or within 4 weeks prior to the first dose of INZ-701, whichever is longer
•7. Pregnant, trying to become pregnant, or breastfeeding
•8. Male participants trying to father a child

Key Dates

Start Date

January 31, 2025

Primary Completion Date

March 31, 2027

Completion Date

March 31, 2028

Last Updated

May 1, 2025

Conditions

Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic RicketsGeneralized Arterial Calcification of Infancy 1

Interventions

INZ-701

DRUG

The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency

NCT06046820

Ectonucleotide Pyrophosphatase/Phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic Rickets+1 more

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UNKNOWN

Natural History of GACI With or Without ARHR2 or PXE

NCT03758534

Generalized Arterial Calcification in InfancyAutosomal Recessive Hypophosphatemic Rickets+1 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: May 1, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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