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 range
1 Year
Sex
ALL
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:
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) concentration through Week 52
. ≥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
. Receiving prohibited medications
. 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