Long-term safety assessment of INZ-701 for patients with ENPP1 and ABCC6 deficiencies
The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
This study is testing the long-term safety of a drug called INZ-701 for people with ENPP1 and ABCC6 deficiencies who have already received it in a previous study.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 200 (estimated) |
| Ages | 1 Year and up |
| Sex | All |
| Sponsor | Inozyme Pharma Industry-sponsored |
| Locations | 5 sites (Rochester, Minnesota and 4 other locations) |
| Trial ID | NCT06462547 on ClinicalTrials.gov |
What this trial studies
This study evaluates the long-term safety of INZ-701, a therapeutic protein designed for treating ENPP1 and ABCC6 deficiencies. Participants who have previously received INZ-701 in another clinical study will continue to receive weekly subcutaneous doses of the drug. The study includes a 30-day screening period followed by an open-label treatment phase, with safety follow-up visits after treatment. The goal is to monitor the safety profile of INZ-701 until it becomes commercially available or development is discontinued.
Who should consider this trial
Good fit: Ideal candidates are individuals over 1 year of age who have previously participated in a clinical study involving INZ-701 for ENPP1 or ABCC6 deficiencies.
Not a fit: Patients who have not previously received INZ-701 or do not have ENPP1 or ABCC6 deficiencies may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a safe and effective long-term treatment option for patients with ENPP1 and ABCC6 deficiencies.
How similar studies have performed: Other studies involving similar therapeutic approaches have shown promise, but the specific use of INZ-701 in this context is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Rochester, Minnesota and 4 other locations
- Mayo Clinic — Rochester, Minnesota, United States (Recruiting)
- Clinilabs Drug Development Corporation — Eatontown, New Jersey, United States (Recruiting)
- Necker-Enfants Malades Hospital — Paris, France (Recruiting)
- Universitätsklinikum Hamburg-Eppendorf (UKE) — Hamburg, Germany (Recruiting)
- Vctc — Oxford, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Inozyme Clinical Trial Information
- Email: clinicaltrials@inozyme.com
- Phone: +1 857 330 4340
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.