ENPP1 Deficiency Adult Trial

Study Overview

The clinical trial for ENPP1 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 ENPP1 Deficiency supported by prior genetic identification of biallelic ENPP1 mutations
Be male or female, 18 to < 65 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

Find a Study Site

Contacts:

Inozyme Clinical Trial Information
1-857-330-4340
clinicaltrials@inozyme.com

Locations:

United States, New Jersey
Clinilabs Recruiting
Eatontown, New Jersey, United States, 07724
Contact: Imani Beard
1-212-994-4567
ARHR2-PXEstudies@clinilabs.com