The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency (NCT06739980) | Clinical Trial Compass
WithdrawnPhase 2
The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency
Stopped: Study was not initiated as it is no longer consistent with the Sponsor's development plans.
0Started 2025-01-31
Plain-language 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.
Who can participate
Age range1 Year
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. 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)
✓. Provide assent in accordance with local regulations, if \<18 years of age
✓. Male or female, ≥1 year of age
✓. 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
✓. Must have at least one of the following clinical signs and/or symptoms consistent with ENPP1 Deficiency:
✓. ≥1 atraumatic vertebral fracture
✓. ≥2 fractures as an adult (eg, long bones, digits, vertebrae)
✓. 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)
Exclusion criteria
✕. 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
✕. Eligible for another Inozyme sponsored study of INZ-701 treatment that is open for enrollment
What they're measuring
1
Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) concentration through Week 52
✕. 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
✕. Received previous treatment with INZ-701
✕. 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
✕. Pregnant, trying to become pregnant, or breastfeeding