The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency
Status: Recruiting
Location: See all (7) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
SUMMARY
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.
Eligibility
Participation Requirements
Sex: All
Maximum Age: 1
Healthy Volunteers: f
View:
• Infant aged ≤ 1 year at the time of enrollment
• Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency
• 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.
• Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
• Written informed consent provided by a parent or legal guardian
Locations
United States
California
Rady Children's Hospital
RECRUITING
San Diego
Massachusetts
Boston Children's Hospital
WITHDRAWN
Boston
Ohio
Nationwide Children's Hospital
RECRUITING
Columbus
Pennsylvania
The Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Utah
The University of Utah
WITHDRAWN
Salt Lake City
Other Locations
Spain
Hospital Sant Joan de Déu
TERMINATED
Barcelona
United Kingdom
Royal Manchester Children's Hospital
TERMINATED
Manchester
Contact Information
Primary
Trial Specialist
medinfo@bmrn.com
+1 800-983-4587
Backup
Medical Director, MD
medinfo@bmrn.com
+1 800-983-4587
Time Frame
Start Date:2023-06-25
Estimated Completion Date:2027-11-11
Participants
Target number of participants:16
Treatments
Experimental: INZ-701
The first 2 study participants will receive 1 dose of 0.2 mg/kg on Day 1 and start at Dose Level A (0.2 mg/kg twice weekly) on Day 8.~The Data Review Committee (DRC) comprised of representatives of the Sponsor, the study Investigators, and a physician who is a subject matter expert not affiliated with the study or Sponsor, will review the safety data of the first study participant through Day 8. Contingent upon this review, the Sponsor will decide if additional study participants can begin receiving INZ-701.~After the second study participant completes Day 32, the DRC will perform a cumulative review of safety and PK/PD data and will make dosing recommendations, for example, modifying the dose of the ongoing study participants and/or changing the starting dose for future participants.~Dose Level A: 0.2 mg/kg twice weekly~Dose Level B: 0.6 mg/kg twice weekly~Dose Level C: 0.2 mg/kg once weekly~Dose Level D: 0.6 mg/kg once weekly~Dose Level E: 1.8 mg/kg once weekly