6 completed, 5 terminated, 1 unknown, 1 withdrawn. 4 Phase 2, 3 Phase 1, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Completed 2, Terminated 1, Withdrawn 1 |
| Phase 1 | 3 | Completed 2, Terminated 1 |
| Phase 3 | 2 | Terminated 2 |
Follow Arterial calcification of infancy: new trials by email Literature Review of Arterial calcification of infancy trials Research Report on Arterial calcification of infancy
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency INZ-701 - Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets | Phase 3 | Terminated | Inozyme Pharma | 2 | 2025-03-26 | 2026-09-29 |
| The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency INZ-701 - Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets | Phase 2 | Withdrawn | Inozyme Pharma | 2025-01-31 | 2025-05-01 | |
| PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency No Intervention for this observational study - Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency, ATP-Binding Cassette Subfamily C Member 6 Deficiency | Terminated | Inozyme Pharma | 54 | 2024-07-25 | 2026-10-01 | |
| ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency INZ-701 - Gene Mutations, Pseudoxanthoma Elasticum | Phase 2 | Terminated | Inozyme Pharma | 22 | 2024-06-19 | 2026-10-19 |
| The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency INZ-701, Control Arm (Conventional Therapy) - Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets | Phase 3 | Terminated | Inozyme Pharma | 27 | 2023-11-05 | 2026-10-01 |
| The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency INZ-701 - Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets | Phase 1 | Terminated | Inozyme Pharma | 9 | 2023-06-25 | 2026-10-15 |
| Natural History Study of ENPP1 Deficiency and the Early-onset Form of ABCC6 Deficiency No Intervention for this observational study - Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Generalized Arterial Calcification of Infancy | Completed | Inozyme Pharma | 12 | 2022-06-28 | 2026-07-06 | |
| Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE INZ-701 - ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum | Phase 1 / Phase 2 | Completed | Inozyme Pharma | 10 | 2022-04-11 | 2026-10-13 |
| Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency INZ-701 - Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets | Phase 1 / Phase 2 | Completed | Inozyme Pharma | 13 | 2021-11-21 | 2026-10-27 |
| Understanding the Spectrum of ENPP1 Deficiency and Acute ABCC6 Deficiency Generalized Arterial Calcification in Infancy, Autosomal Recessive Hypophosphatemic Rickets Type 2 | Completed | Inozyme Pharma | 38 | 2020-05-06 | 2021-04-01 | |
| Natural History Study: ENPP1 Deficiency or the Early-Onset Form of ABCC6 Deficiency Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, ATP-Binding Cassette Subfamily C Member 6 Deficiency | Completed | Inozyme Pharma | 23 | 2018-12-05 | 2026-08-04 | |
| Study of People With Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2) Generalized Arterial Calcification of Infancy, Autosomal Recessive Hypophosphatemic Rickets Type2 | Completed | National Human Genome Research Institute (NHGRI) | 48 | 2018-04-17 | 2026-07-16 | |
| Natural History of GACI With or Without ARHR2 or PXE No intervention - Generalized Arterial Calcification in Infancy, Autosomal Recessive Hypophosphatemic Rickets | Unknown | Universität Münster | 80 | 2018-03-15 | 2018-11-29 |
Hypophosphatemic Rickets, Autosomal Recessive, 1 (9) · Pseudoxanthoma Elasticum (6)
Cardiovascular Diseases (6) · Congenital Abnormalities (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Connective Tissue Diseases (6) · Genetic Diseases, Inborn (6) · Hematologic Diseases (6) · Hemic and Lymphatic Diseases (6) · Hemorrhagic Disorders (6) · Hemostatic Disorders (6) · Skin Abnormalities (6) · Skin Diseases (6) · Skin Diseases, Genetic (6) · Skin and Connective Tissue Diseases (6) · Vascular Diseases (6)
Inozyme Pharma (11) · National Human Genome Research Institute (NHGRI) (1) · Universität Münster (1)
United States (10) · United Kingdom (9) · France (5) · Germany (5) · Canada (4) · Spain (3) · Turkey (Türkiye) (3) · Italy (2) · Saudi Arabia (2) · Australia (1) · Brazil (1) · Hungary (1) · Japan (1) · Oman (1) · United Arab Emirates (1)
Carlos R Ferreira Lopez, M.D. (1) · Frank Rutsch, MD (1) · Kurt Gunter, MD (1) · Pedro Huertas, MD (1)