ABCC6 Deficiency Trial
Study Overview
The clinical trial for ABCC6 Deficiency will determine if a new medication, INZ-701, is suitable to treat those affected with the disease. Participants will be split into 3 groups. Each group will receive a different dose of INZ-701. INZ-701 is a subcutaneous injection (under the skin).
Researchers will monitor participants on increasing doses of INZ-701 to:
- Understand the safety of the medication
- Study how the medication is processed throughout the body
- Study changes in PPi and other markers
For the extension period of the study, researchers will monitor participants on increasing doses of INZ-701 to evaluate skeletal, vascular, and physical function as well as outcomes reported by participants.
Subject participation consists of a screening period, a 32-day dose-evaluation period, and a 48-week extension period following completion of the dose-evaluation period.
Eligibility Criteria
Participants must:
- Provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures
- Be diagnosed with ABCC6 Deficiency supported by prior genetic identification of biallelic ABCC6 mutations
- Be male or female, 18 to < 70 years old at screening
- Have a PPi level at screening that meets the requirement
- Be willing and able to complete all aspects of the study in the opinion of the researcher
- Agree to provide access to relevant medical records
People being treated with statins or proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors must:
- Be on stable doses for 6 months prior to enrollment through end of study unless previously cleared
Women of child-bearing potential must:
- Have a negative serum pregnancy test at screening
Women of child-bearing potential and partners of fertile males who are of child-bearing potential must agree to:
- Use 1 highly effective form of contraception and a barrier method from at least 1 month before the first dose through 30 days after last dose of treatment
- Not donate ova from the period following the first dose through 30 days after last dose of treatment
Males who are sexually active must agree to:
- Use condoms from the period following first dose through 30 days after the last dose of treatment
- Not donate sperm from the period following the first dose through 30 days after last dose of treatment
