Can a replacement enzyme keep calcium buildup in check?
NCT ID NCT06462547
First seen Aug 28, 2026 · Last updated Aug 28, 2026
Summary
This trial tests the long-term safety of a drug called INZ-701 in people with rare genetic conditions that cause abnormal calcium buildup in the body. The study enrolls people who have already received INZ-701 in an earlier trial and want to continue treatment. Researchers will monitor side effects and check whether the body develops antibodies against the drug. The goal is to see if INZ-701 can be a safe, ongoing treatment for these conditions.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- INZ-701, a recombinant fusion protein designed to replace the missing ENPP1 enzyme
- What this could lead to
- If successful, this could support a long-term treatment option for people with ENPP1 or ABCC6 deficiency, potentially slowing or preventing harmful calcium buildup in blood vessels and tissues.
- What could go wrong
- This is a small, open-label study focused on safety, not effectiveness. The drug may cause side effects or immune reactions, and long-term benefits are not yet proven.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
22 people
The number who actually took part.
- Started
-
Jun 2024
- Expected to finish
-
Oct 2026
An estimate. End dates often move.
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
1 year and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Individuals eligible to participate must meet all of the following 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, 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 Individuals who meet any of the following exclusion criteria will not be eligible to participate: 1. In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency or ABCC6 Deficiency, that will preclude study participation and/or may confound interpretation of study results 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Clinilabs Drug Development Corporation
Eatontown, New Jersey, 07724, United States
-
Mayo Clinic
Rochester, Minnesota, 55905, United States
-
Medical Research Network Ltd.
Milton Keynes, United Kingdom
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Necker-Enfants Malades Hospital
Paris, France
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Universitätsklinikum Hamburg-Eppendorf (UKE)
Hamburg, Germany
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