ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

Interested in this Trial?

Contact Us

Trial Status Recruiting

Trial Identifier

NCT06462547

Condition

ENPP1 Deficiency, ABCC6 Deficiency, Calciphylaxis, Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Pseudoxanthoma Elasticum, Gene Mutations, Arterial Calcification, Autosomal Recessive Hypophosphatemic Rickets Type 2

Notes

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.

Trial Summary

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.

Eligibility Criteria

Birth Sex

All

Age

From 1 Year

Healthy Volunteers

No

Drug/Treatment:

INZ-701

Phase:

Phase 2

Study Type:

Interventional

Number of Participants:

200

Study Started:

2024-06-19

Study Updated:

2024-11-29

Trial Locations

Eligibility 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

Contact BioMarin Clinical Trials

Please complete and submit the following form to express interest in participating and request more information about one of our active clinical trials

*required fields

"*" indicates required fields

Please select the trial that you are interested in from the dropdown below. Note that this list only includes currently active trials. If you cannot find the trial you are seeking, please enter the study identifier and title in the comments box below, along with any other details you would like us to know to help address your inquiry.
For more Information about BioMarin's privacy obligations or to exercise your privacy rights, please visit BioMarin's Data Privacy Center.
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form