NCT06462547
ENPP1 Deficiency, ABCC6 Deficiency, Calciphylaxis, Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Pseudoxanthoma Elasticum, Gene Mutations, Arterial Calcification, Autosomal Recessive Hypophosphatemic Rickets Type 2
Following BioMarin’s acquisition of Inozyme in 2025, INZ-701 is now known as BMN 401.
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
The purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
All
From 1 Year
No
INZ-701
Phase 2
Interventional
200
2024-06-19
2024-11-29
Rochester, Minnesota, United States
Trial Status Recruiting
Robert Wermers, MD
508-284-4738 wermers.robert@mayo.edu
Eatontown, New Jersey, United States
Trial Status Recruiting
Magdy Shenouda, MD
732-754-9329 mshenouda@clinilabs.com
Paris, France
Trial Status Recruiting
Alix Besancon, MD
01-44-73-66-31 alix.besancon@aphp.fr
Hamburg, Germany
Trial Status Recruiting
Ralf Oheim, MD
49-40-7410-53714 r.oheim@uke.de
Oxford, United Kingdom
Trial Status Recruiting
Rosia Shah, MD
07-97-605-8027 rosia.shaw@thevctc.com
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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