The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency

Condition(s) studied

Investigational drug(s) / intervention(s)

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.

Study summary

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.

Eligibility

Primary outcome measure(s)

• Number of Treatment Emergent Adverse Events (TEAEs) — 52 weeks (Treatment Period)
  Treatment-emergent AEs are defined as any AE occurring from the first dose of INZ-701 through 30 days after the last dose of INZ-701.
• Incidence of Anti-Drug Antibodies (ADA) — 52 weeks (Treatment Period)
  For each participant, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
• Left Ventricular Ejection Fraction — 52 weeks (Treatment Period)
  For each participant, an echocardiogram will be collected, and used to assess heart function. (Including measurement of left ventricular ejection fraction), and to identify any other abnormalities, for example, calcification of heart valves.

Trial sites (5)