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

Conditions

• ATP-Binding Cassette Subfamily C Member 6 Deficiency
• Autosomal Recessive Hypophosphatemic Rickets
• Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
• Generalized Arterial Calcification of Infancy
• Pseudoxanthoma Elasticum

Eligibility Criteria

Sex

ALL

Age

N/A - 1 Year

Healthy Volunteers

Not accepted

Interventions

• INZ-701 — DRUG
  Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.

Study Details

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.

Key Dates

Start date

Jun 25, 2023

Status verified

Mar 2026

Primary completion

Nov 11, 2027

Completion

Nov 11, 2027

Study Design

Enrollment

16 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: INZ-701
  The first 2 study participants will receive 1 dose of 0.2 mg/kg on Day 1 and start at Dose Level A (0.2 mg/kg twice weekly) on Day 8. The Data Review Committee (DRC) comprised of representatives of the Sponsor, the study Investigators, and a physician who is a subject matter expert not affiliated with the study or Sponsor, will review the safety data of the first study participant through Day 8. Contingent upon this review, the Sponsor will decide if additional study participants can begin receiving INZ-701. After the second study participant completes Day 32, the DRC will perform a cumulative review of safety and PK/PD data and will make dosing recommendations, for example, modifying the dose of the ongoing study participants and/or changing the starting dose for future participants. Dose Level A: 0.2 mg/kg twice weekly Dose Level B: 0.6 mg/kg twice weekly Dose Level C: 0.2 mg/kg once weekly Dose Level D: 0.6 mg/kg once weekly Dose Level E: 1.8 mg/kg once weekly

Primary Outcome Measure

Number of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: 52 weeks (Treatment Period) ]

Central Contacts

• Trial Specialist
  +1 800-983-4587
  [email protected]
• Medical Director, MD
  +1 800-983-4587
  [email protected]

Locations (5)

Find similar trials in San Diego, CA

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