NCT05734196
ENPP1 Deficiency, ABCC6 Deficiency, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, Pseudoxanthoma Elasticum
Following BioMarin’s acquisition of Inozyme in 2025, INZ-701 is now known as BMN 401.
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
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.
All
Up to 1 Year
No
INZ-701
Phase 1
Interventional
16
2023-06-25
2025-02-07
San Diego, California, United States
Trial Status Recruiting
Nathaly Sweeney, MD
858-966-5818 nsweeney@rchsd.org
Sarah Lazar, MPH
8585761700 slazar@health.ucsd.edu
Boston, Massachusetts, United States
Trial Status Recruiting
Alayna Dutcher
617-355-0741 Alayna.dutcher@childrens.harvard.edu
Andrea Hale, RN, MPH
617-919-2867 andrea.hale@childrens.harvard.edu
Columbus, Ohio, United States
Trial Status Recruiting
Bimal Chaudhari, MD
614-722-3535 bimal.chaudhari@nationwidechildrens.org
Marina Artemova, PhD
614-722-2655 marina.artemova@nationwidechildrens.org
Philadelphia, Pennsylvania, United States
Trial Status Recruiting
Olivia Lucas
267-432-0511 lucaso@chop.edu
Rachel Walega
267-586-5969 WALEGAR1@chop.edu
Salt Lake City, Utah, United States
Trial Status Recruiting
Carrie Bailey, BS
801-587-3605 ped_mgr@lists.hsc.utah.edu
Barcelona, Spain
Trial Status Not yet recruiting
Georgia Sarquella-Brugada, MD
georgia.sarquella@sjd.es
Manchester, United Kingdom
Trial Status Recruiting
Amish Chinoy, MD
0161 701 7901 amish.chinoy@mft.nhs.uk
Victoria Hamilton, RN
0161 701 8313 victoria.hamilton@mft.nhs.uk
1. Caregiver(s) must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council for Harmonisation (ICH) Good Clinical Practice (GCP)
2. Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory
3. Study participants must have clinical manifestations of GACI or GACI-2, which may include, but are not limited to, pathologic ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly
4. Study participant must be male or female from birth to <1 year of age at Baseline (Day 1)
5. Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
6. In the opinion of the Investigator, the study participant must be able to complete all aspects of the study
7. Study participant’s caregiver(s) must agree to provide access to their child’s relevant medical records
1. In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality (outside of those considered associated with the diagnosis of ENPP1 Deficiency or ABCC6 Deficiency) that precludes study participation or may confound interpretation of study results, including known uncontrolled thyroid disease or unrelated connective tissue, bone, mineral, or muscle disease
2. Care has been withdrawn or study participant is receiving end of life care or hospice only
3. Known malignancy
4. Known intolerance to INZ-701 or any of its excipients
5. Concurrent participation in another non-Inozyme interventional study
6. Receipt of any non-Inozyme investigational new drug within 5 half-lives of the last dose of the other investigational product or within 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device, through completion of participation in the study
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