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

Generalized Arterial Calcification of Infancy Clinical Trial

Official title:
The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infant Subjects With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency

Status Recruiting

Clinical Trial 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.

Clinical Trial Description

INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy in development for the treatment of the ultra rare genetic disorder, ENPP1 Deficiency. Study INZ701-104 (the ENERGY study) is a Phase 1b, open-label study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in infant study participants with ENPP1 Deficiency. The study will consist of up to a 60-day Screening Period, a 52-week Treatment Period during which study participants will receive INZ-701, an Extension Period during which study participants may continue to receive INZ-701, and an End of Treatment (EOT) visit 30 days after the last dose of INZ-701. Upon treatment discontinuation, study participants may continue to be followed for their ongoing disposition for survival outcome at least quarterly, if feasible through the end of the study. If the study site is not able to complete the survival outcome follow-up for up to 26 weeks post-treatment discontinuation, then the study participant can be considered "lost to follow up. ;

Study Design

Related Conditions & MeSH terms

Autosomal Recessive Hypophosphatemic Rickets
Calcinosis
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
Familial Hypophosphatemic Rickets
Generalized Arterial Calcification of Infancy
Rickets
Vascular Calcification

Clinical Trial Details

NCT number	NCT05734196
Study type	Interventional
Source	Inozyme Pharma
Contact	Inozyme Clinical Trial Information
Phone	+1 857 330 4340
Email	clinicaltrials@inozyme.com
Status	Recruiting
Phase	Phase 1
Start date	May 25, 2023
Completion date	April 1, 2025

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT06046820` - The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency	Phase 3
Recruiting	`NCT04686175` - Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency	Phase 1/Phase 2
Not yet recruiting	`NCT05050669` - Natural History Study of ENPP1 Deficiency and and the Early-onset Form of ABCC6 Deficiency
Completed	`NCT03478839` - Study of People With Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2)
Active, not recruiting	`NCT05030831` - Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE	Phase 1/Phase 2