A Phase 3 interventional study of INZ-701 in Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets and Generalized Arterial Calcification of Infancy 1, sponsored by Inozyme Pharma. Terminated at 8 sites in 8 countries. Open to participants aged 0 Years to 1 Year. Per ClinicalTrials.gov, last updated 2026-09-29.
Sponsored by Inozyme Pharma (part of BioMarin Pharmaceutical) · Phase 3, Interventional, and Treatment
The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
ENPP1 Deficiency is an ultra-rare genetic disorder in which inactivating mutations in the ENPP1 gene lead to a deficiency in the ENPP1 enzyme.
ENERGY 2 (Study INZ701-105) is a multicenter, single-arm, open-label Phase 3 study to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
The study will consist of a Screening Period of up to 60 days, a Treatment Period of 52 weeks, a 52-week Extension Period, and an End of Treatment (EOT) Visit 30 days after the last dose of INZ-701.
Inozyme Pharma is the lead sponsor of 12 studies on the registry; none are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 2 (33%) have results posted.
First submitted after Inozyme Pharma became part of BioMarin Pharmaceutical.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Participants must meet all of the following:
Inclusion Criteria:
Exclusion Criteria
Participants will not be eligible if any of the following apply:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants receive INZ-701 (rhENPP1-Fc) administered by subcutaneous injection once weekly at the protocol-specified dose. The visit final volume to administer (mL) is determined using protocol-defined parameters.
Drug: INZ-701
Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.
Also known as: (rhENPP1-Fc).
To determine if INZ-701 increases inorganic pyrophosphate (PPi) levels
For each subject, their change from baseline in Plasma Inorganic Pyrophosphate (PPi) concentration will be assessed.
Time frame: 52 weeks (Baseline through Week 52)
To determine if INZ-701 increases overall survival
For each subject, their change in overall survival based on time from date of birth to event of all-cause mortality will be assessed.
Time frame: 52 weeks (Baseline through Week 52)
To determine if INZ-701 prevents decline in cardiac ejection fraction
For each subject, their change from baseline in left ventricular ejection fraction will be assessed via echocardiography.
Time frame: 52 weeks (Baseline through Week 52)
To determine if INZ-701 prevents heart failure
For each subject, their incidence of heart failure will be assessed.
Time frame: 52 weeks (Baseline through Week 52)
To determine if INZ-701 attenuates progression of arterial calcification
For each subject, their change from baseline in vascular calcification in the coronary arteries and aorta will be examined via CT scan.
Time frame: 52 weeks (Baseline through Week 52)
Ran from Mar 2025 to primary completion in Aug 2026, 17 months.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is terminated, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Efmirenpase alfa
Hypophosphatemic Rickets, Autosomal Recessive, 1