INZ-701

DRUG12 trials

Sponsors

Inozyme Pharma Inc., Inozyme Pharma

Conditions

ATP-Binding Cassette Subfamily C Member 6 DeficiencyArterial CalcificationAutosomal Recessive Hypophosphatemic RicketsAutosomal Recessive Hypophosphatemic Rickets Type 2CalciphylaxisEctonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) DeficiencyEctonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency and ATP-binding cassette sub-family C member 6 (ABCC6) DeficiencyEctonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency

Phase 1

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency

CompletedNCT04686175

Inozyme PharmaAutosomal Recessive Hypophosphatemic Rickets, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Generalized Arterial Calcification of Infancy

Start: 2021-11-21End: 2024-12-13Updated: 2025-02-05

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE

CompletedNCT05030831

Inozyme PharmaATP-Binding Cassette Subfamily C Member 6 Deficiency, Generalized Arterial Calcification of Infancy, Pseudoxanthoma Elasticum

Start: 2022-04-11End: 2024-07-09Updated: 2025-02-05

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

Active, not recruitingNCT05734196

Inozyme PharmaATP-Binding Cassette Subfamily C Member 6 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency +2

Start: 2023-06-25End: 2026-10-14Target: 16Updated: 2026-08-10

SEAPORT 1: Study of INZ-701 in Adults With Hemodialysis-Dependent End-Stage Kidney Disease

CompletedNCT06283589

Inozyme PharmaCalciphylaxis

Start: 2024-02-12End: 2024-12-13Updated: 2026-08-24

The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants with Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency

CompletedCTIS2023-507384-20-00

Inozyme Pharma Inc.Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency

Start: 2024-10-21End: 2025-04-10Target: 2Updated: 2025-04-10

Phase 2

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

Active, not recruitingNCT06462547

Inozyme PharmaArterial Calcification, Autosomal Recessive Hypophosphatemic Rickets Type 2, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency +2

Start: 2024-06-19End: 2026-10-01Updated: 2026-08-28

The ADAPT Study: An Open-Label, Long-term Safety Study of INZ-701 in Patients with ENPP1 Deficiency and ABCC6 Deficiency

RecruitingCTIS2024-512715-42-00

Inozyme Pharma Inc.Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency and ATP-binding cassette sub-family C member 6 (ABCC6) Deficiency, Generalized Arterial Calcification of Infancy (GACI), Pseudoxanthoma elasticum (PXE)

Start: 2024-10-08Target: 14Updated: 2025-08-14

The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency

WithdrawnNCT06739980

Inozyme PharmaAutosomal Recessive Hypophosphatemic Rickets, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Generalized Arterial Calcification of Infancy 1

Start: 2025-01-31End: 2028-03-31Updated: 2025-05-01

Phase 3

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

Active, not recruitingNCT06046820

Inozyme PharmaAutosomal Recessive Hypophosphatemic Rickets, Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Generalized Arterial Calcification of Infancy

Start: 2023-11-05End: 2026-02-28Updated: 2025-05-01

The ENERGY 3 Study: A Randomized, Controlled, Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Children with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

Active, not recruitingCTIS2023-507382-26-00

Inozyme Pharma Inc.Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

Start: 2024-05-24Target: 8Updated: 2025-07-30

The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

RecruitingCTIS2024-512991-36-00

Inozyme Pharma Inc.Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

Start: 2024-10-24Target: 6Updated: 2026-01-26

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

Active, not recruitingNCT07473973

Inozyme PharmaAutosomal Recessive Hypophosphatemic Rickets, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Generalized Arterial Calcification of Infancy 1

Start: 2025-03-26End: 2026-10-14Target: 12Updated: 2026-08-10

Related Papers

7217 Impact of INZ-701 on Bone and Mineral Metabolism Biomarkers and Clinical Outcomes in Adults with ENPP1 Deficiency - Results from 48-week Phase 1/2 Open Label Study

Journal of the Endocrine Society2024-10-01