Actively Recruiting
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
Led by Inozyme Pharma · Updated on 2026-03-24
16
Participants Needed
7
Research Sites
228 weeks
Total Duration
On this page
Sponsors
I
Inozyme Pharma
Lead Sponsor
B
BioMarin Pharmaceutical
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Infant aged 64 1 year at the time of enrollment
- Confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency
- Clinical signs of generalized arterial calcification of infancy (GACI) or GACI-2 including ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, or cardiomegaly
- Infant weighs 64 0.5 kg at first dose of INZ-701
- Written informed consent from parent or legal guardian
You will not qualify if you...
- Any clinically significant disease or lab abnormality that prevents participation or affects study results, per Investigator
- Receiving end of life or hospice care
- Known cancer
- Participation in another non-Inozyme interventional study
- Treatment with any non-Inozyme product or investigational device during study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 7 locations
1
Rady Children's Hospital
San Diego, California, United States, 92123
Actively Recruiting
2
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Withdrawn
3
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
4
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
5
The University of Utah
Salt Lake City, Utah, United States, 84108
Withdrawn
6
Hospital Sant Joan de Déu
Barcelona, Spain
Terminated
7
Royal Manchester Children's Hospital
Manchester, United Kingdom, M13 9WL
Terminated
Research Team
T
Trial Specialist
CONTACT
M
Medical Director, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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