The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
Recruiting
Phase 1
Interventional Study
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
Autosomal Recessive Hypophosphatemic Rickets
Generalized Arterial Calcification of Infancy
ATP-Binding Cassette Subfamily C Member 6 Deficiency
Pseudoxanthoma Elasticum
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- Up to 1
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 16 (estimated)
- Sponsor
- Inozyme Pharma · Industry
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
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 (G…
Contacts