The clinical trial for ENPP1 deficiency will determine if a new medication, INZ-701, is suitable to treat those affected. Participants will be split into 3 groups. Each group will receive a different dose of INZ-701. Every 4 weeks, the dose given will increase if it is deemed safe to do so.
INZ-701 is a subcutaneous injection (under the skin).
Researchers will monitor participants on increasing doses of INZ-701 to:
The estimated timing of participation is 7 weeks. Results from this study will help scientists further develop INZ-701 to treat ENPP1 deficiency.
- Understand the safety of the medication
- Study how the medication is processed throughout the body
- Study changes in PPi and other markers
Participants must be:
Women of child-bearing potential (and their male partner) must:
- Male or female, 18-65 years old
- Diagnosed with ARHR2 by a doctor with a confirmation of the ENPP1 gene
- Able to provide written consent by themselves or through a legal caregiver before research
- Able to discontinue the use of oral PPi from at least 14 days before and throughout treatment, if applicable
- Able to discontinue use of bisphosphonates for 6 months prior to study start, if applicable
Males who are sexually active must agree to:
- Have a negative pregnancy test at screening and during the study
- Agree to use 2 forms of birth control from the start through 25 days after treatment
- Agree to not donate ova from the period from the start through 25 days after treatment
- Use condoms from the period from the start through 25 days after treatment
- Not donate sperm from the period from the start through 25 days after treatment
If enrolled, participants will complete a number of tests and visits throughout the study. The tests and visits may be time-consuming and/or require assistance from a caregiver. A researcher will thoroughly explain all requirements before enrollment.
If you have a confirmed ENPP1
diagnosis and meet the criteria described, please see additional information
about study enrollment. Otherwise, keep reading to see how you can get tested for ENPP1 deficiency.