15 completed, 7 unknown, 6 recruiting, 2 terminated. 8 Phase 2, 6 Not applicable, 3 Phase 1, 1 Phase 3, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 8 | Completed 6, Terminated 1, Withdrawn 1 |
| Not applicable | 6 | Recruiting 3, Unknown 2, Completed 1 |
| Phase 1 | 3 | Completed 2, Terminated 1 |
| Phase 3 | 1 | Recruiting 1 |
| Phase 4 | 1 | Unknown 1 |
Follow Pseudoxanthoma Elasticum: new trials by email Literature Review of Pseudoxanthoma Elasticum trials Research Report on Pseudoxanthoma Elasticum
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 |
|---|---|---|---|---|---|---|
| Impact of Optimized Recruitment and Follow-up of Patients With Pseudoxanthoma Elasticum (PXE) Pseudoxanthoma Elasticum - Pseudoxanthoma Elasticum | Recruiting | University Hospital, Angers | 650 | 2026-02-09 | 2026-04-06 | |
| Purinergic Compounds in Pseudoxanthoma Elasticum supplementary tubes, SCANNER - Pseudoxanthoma Elasticum | Not applicable | Recruiting | Centre Hospitalier Universitaire de Nice | 45 | 2026-01-20 | 2026-04-24 |
| CHOPXE - Analysis of Choriocapillaris Flow Deficits in Patients With Pseudoxanthoma Elasticum Opthtalmologic diagnosis tests - Pseudoxanthoma Elasticum, Tomography, Optical Coherence | Not applicable | Recruiting | University Hospital, Angers | 60 | 2025-11-28 | 2025-12-08 |
| Progression Assessment of PXE-associated Alterations Pseudoxanthoma Elasticum, Angioid Streaks | Recruiting | University Hospital, Bonn | 100 | 2024-09-01 | 2025-07-02 | |
| 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 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 |
| The Prevention of Systemic Ectopic Mineralization in Pseudoxanthoma Elasticum Etidronate, Placebo - Pseudoxanthoma Elasticum | Phase 3 | Recruiting | UMC Utrecht | 76 | 2023-04-26 | 2023-04-27 |
| PPI Supplementation to Fight ECtopIc Calcification in PXE study treatment PPI, Placebo comparator - Pseudoxanthoma Elasticum | Not applicable | Recruiting | Centre Hospitalier Universitaire de Nice | 99 | 2022-12-13 | 2024-05-17 |
| A Study of DS-1211b in Individuals With PseudoXanthoma Elasticum DS-1211b, Placebo - Pseudoxanthoma Elasticum | Phase 2 | Completed | Daiichi Sankyo | 65 | 2022-10-20 | 2024-12-31 |
| Progression Rate of Pseudoxanthoma Elasticum-associated Choroidal and Retinal Degeneration No intervention - Pseudoxanthoma Elasticum | Active not recruiting | University Hospital, Basel, Switzerland | 25 | 2022-10-01 | 2025-04-22 | |
| 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 |
| Employment of Patients With Pseudoxanthoma Elasticum Telephone Call - Pseudoxanthoma Elasticum, Employment | Completed | University Hospital, Angers | 99 | 2022-04-07 | 2023-02-13 | |
| Pseudoxanthoma Elasticum (PXE) Natural History Biomarkers in PXE Individuals and Their Biological Non-PXE Siblings Non Interventional - Pseudoxanthoma Elasticum | Completed | Daiichi Sankyo | 48 | 2021-08-30 | 2022-09-06 | |
| Oral Pyrophosphate Absorption in PXE Disease Disodium Pyrophosphate - Pseudoxanthoma Elasticum | Phase 2 | Withdrawn | Tampere University Hospital | 2020-12-08 | 2021-04-22 | |
| Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) Lansoprazole 30mg, Placebo - Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) | Phase 4 | Unknown | Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud | 20 | 2020-02-04 | 2020-12-09 |
| The Characteristics of Backscattering With Depth in the Progression of Keratoconus Elasticum, Incomplete Pseudoxanthoma | Completed | Tianjin Eye Hospital | 223 | 2020-01-01 | 2023-09-22 | |
| Intestinal Microbiota and Vitamin K Levels in PXE Patients (IMPROVE Study) Fecal and blood samples - Pseudoxanthoma Elasticum | Not applicable | Unknown | University Hospital, Angers | 20 | 2019-01-21 | 2019-02-04 |
| 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 |
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Arterial calcification of infancy (6) · Angioid Streaks (4) · Hypophosphatemic Rickets, Autosomal Recessive, 1 (4) · Calcinosis (3) · Choroidal Neovascularization (3) · Dermatitis (1) · Diabetes Mellitus, Type 2 (1) · Myopia, Degenerative (1) · Polypoidal Choroidal Vasculopathy (1) · Renal Insufficiency, Chronic (1) · Retinal Diseases (1) · Retinal Telangiectasis (1) · Vitelliform Macular Dystrophy (1) · von Hippel-Lindau Disease (1)
Cardiovascular Diseases (32) · Congenital Abnormalities (32) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (32) · Connective Tissue Diseases (32) · Genetic Diseases, Inborn (32) · Hematologic Diseases (32) · Hemic and Lymphatic Diseases (32) · Hemorrhagic Disorders (32) · Hemostatic Disorders (32) · Skin Abnormalities (32) · Skin Diseases (32) · Skin Diseases, Genetic (32) · Skin and Connective Tissue Diseases (32) · Vascular Diseases (32) · Eye Diseases (6)
Ranibizumab (2) · ABCC6 protein, human (1) · Blood Specimen Collection (1) · Defecation (1) · Etidronic Acid (1) · Lansoprazole (1) · Magnesium Oxide (1) · Magnetic Resonance Spectroscopy (1) · Urine Specimen Collection (1) · aflibercept (1) · sodium pyrophosphate (1)
University Hospital, Angers (9) · Inozyme Pharma (4) · University Hospital, Bonn (4) · Centre Hospitalier Universitaire de Nice (2) · Daiichi Sankyo (2) · European Georges Pompidou Hospital (1) · Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud (1) · Manhattan Eye, Ear & Throat Hospital (1) · Mark Lebwohl (1) · National Cancer Institute (NCI) (1) · National Human Genome Research Institute (NHGRI) (1) · Tampere University Hospital (1) · Tianjin Eye Hospital (1) · UMC Utrecht (1) · University Hospital, Basel, Switzerland (1)
France (13) · United States (10) · Germany (7) · United Kingdom (4) · Netherlands (3) · Spain (2) · China (1) · Switzerland (1)
Carlos R Ferreira Lopez, M.D. (1) · Frank G. Holz, MD (1) · Frank G. Holz, MD, DPhil (1) · Frank Rutsch, MD (1) · Hendrik PN Scholl, MD, MA (1) · Kristina Pfau, MD (1) · Kurt Gunter, MD (1) · Lawrence A. Yannuzzi, MD (1)
PMID 15894595, PMID 21704964, PMID 22152229, PMID 24277820, PMID 24651258, PMID 24969777, PMID 26586530, PMID 28658601, PMID 33925341, PMID 39881395, PMID 39913163, PMID 41131563, PMID 41230901, PMID 8537157
2019 (1)