The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

27

Participants

Timeline

Start Date

November 5, 2023

Primary Completion Date

January 31, 2026

Study Completion Date

February 28, 2026

Conditions
Ectonucleotide Pyrophosphatase/Phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic RicketsGeneralized Arterial Calcification of Infancy
Interventions
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.

DRUG

Control Arm (Conventional Therapy)

Conventional therapy is defined as oral phosphate supplements and calcitriol or other active forms of vitamin D3 (or analogs). No other agents for treatment of ENPP1 Deficiency are allowed in the control arm.

Trial Locations (15)

4101

Queensland Children's Hospital, South Brisbane

12713

King Faisal Specialist Hospital and Research Centre, Riyadh

19104

The Children's Hospital of Philadelphia, Philadelphia

30726

Al Jalila Children's Specialty Hospital, Dubai

34764

Umraniye Training and Research Hospital, Istanbul

43205

Nationwide Children's Hospital, Columbus

60611

Ann & Robert H. Lurie Children's Hospital, Chicago

76104

Cook Children's Medical Center, Fort Worth

80045

Children's Hospital of Colorado, Aurora

94270

Hôpital Bicêtre, Service d'endocrinologie et diabète de l'enfant (Childhood Endocrinology and Diabetes Department), Le Kremlin-Bicêtre

02115

Boston Children's Hospital, Boston

H3T 1C5

Centre Hospitalier Universitaire (CHU) Sainte-Justine, Montreal

08950

Hospital San Joan de Deu, Barcelona

01330

Cukurova Universitesi Tip Fakultesi, Sarıçam

M13 9WL

Royal Manchester Children's Hospital, Manchester

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Inozyme Pharma

INDUSTRY

NCT06046820 - The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency | Biotech Hunter | Biotech Hunter