ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency

Phase 3RecruitingNCT07473973

Inozyme Pharma12 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Autosomal Recessive Hypophosphatemic Rickets Generalized Arterial Calcification of Infancy 1

Interventions

INZ-701DRUG

Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.

Eligibility

Sex: ALLMin age: 0 YearsMax age: 1 Year

Medical Language ↔ Plain English

Participants must meet all of the following: Inclusion Criteria: 1. Infant aged ≤ 1 year at the time of enrollment. 2. Confirmed diagnosis of ENPP1 deficiency, based on genetic testing. 3. Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement). 4. Medically stable to participate in a 52-week treatment study. 5. Written informed consent provided by a parent or legal guardian. Exclusion Criteria Participants will not be eligible if any of the following apply: 1. Receiving end-of-life or hospice care. 2. Prior treatment with INZ-701, unless received through an approved expanded access program. 3. Concurrent participation in another interventional clinical trial. 4. Planned major surgery during the study period that would interfere with study participation.

Locations (8)

Hospital Universitario Pedro Ernesto/Rio de Janeiro

Rio de Janeiro, Brazil

RECRUITING

Hopital Necker - Enfants Malades

Paris, France

RECRUITING

Gyermekgyogyaszat, DE

Outcomes

Primary Outcomes

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.