ClinicaliQ Trial Snapshot
- The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency — Recruiting • Phase I • Cardiology / Cardiovascular • NCT05734196.
- The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency. Lead.
- Sponsor: Inozyme Pharma.
Verify eligibility, endpoints and current status on the original source registry before acting on this summary.
Use This Page For
- Quick orientation before opening the registry record.
- Checking recruitment status, phase and sponsor at a glance.
- Connecting this trial to nearby guidelines, Drug Science and education.
What This Trial Is Studying
The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency. Lead Sponsor: Inozyme Pharma Start Date: 2023-06-25 Primary Completion: 2027-11-11
Eligibility Snapshot
- : 1. Infant aged ≤ 1 year at the time of enrollment 2. Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency 3. Study participants must have clinical manifestations of generalized arterial calcification of infancy (GACI) or GACI-2, which must include at least one of the following: ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly. 4. Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study 5. Written informed consent provided by a parent or legal guardian
Use the source registry for the full inclusion and exclusion criteria before discussing referral or enrolment.