166. Pseudoxanthoma elasticum Clinical trials / Disease details
Clinical trials : 15 / Drugs : 27 - (DrugBank : 5) / Drug target genes : 5 - Drug target pathways : 28
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05030831 (ClinicalTrials.gov) | September 22, 2021 | 26/8/2021 | Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE | A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults With ABCC6 Deficiency Manifesting as Pseudoxanthoma Elasticum (PXE) | ATP-Binding Cassette Subfamily C Member 6 Deficiency;Pseudoxanthoma Elasticum;Generalized Arterial Calcification of Infancy | Drug: INZ-701 | Inozyme Pharma | IQVIA Biotech | Recruiting | 18 Years | 64 Years | All | 9 | Phase 1/Phase 2 | United States;United Kingdom |
| 2 | NCT04868578 (ClinicalTrials.gov) | September 1, 2021 | 30/3/2021 | PyROphosPHate Supplementation to Fight ECtopIc Calcification in PseudoXanthoma Elasticum | PyROphosPHate Supplementation to Fight ECtopIc Calcification in PseudoXanthoma Elasticum: PROPHECI Study | Pseudoxanthoma Elasticum | Drug: study treatment PPI;Drug: Study comparator ETI | Centre Hospitalier Universitaire de Nice | NULL | Not yet recruiting | 18 Years | 65 Years | All | 99 | Phase 2 | France |
| 3 | EUCTR2020-004000-33-FR (EUCTR) | 10/06/2021 | 28/04/2021 | A Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of INZ-701 in Adults with ABCC6 Deficiency causing Pseudoxanthoma elasticum (PXE) | A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults with ABCC6 Deficiency Manifesting as Pseudoxanthoma elasticum (PXE) | Treatment of patients with ABCC6 Deficiency Manifesting as Pseudoxanthoma elasticum (PXE) MedDRA version: 20.0;Level: PT;Classification code 10037150;Term: Pseudoxanthoma elasticum;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: INZ-701 Product Code: INZ-701 INN or Proposed INN: RECOMBINANT HUMAN ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE 1 FUSED TO THE FC FRAGMENT OF IGG1 Other descriptive name: RECOMBINANT HUMAN ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE 1 FUSED TO THE FC FRAGMENT OF IGG1 | Inozyme Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 9 | Phase 1;Phase 2 | France | ||
| 4 | NCT04441671 (ClinicalTrials.gov) | December 8, 2020 | 20/1/2020 | Oral Pyrophosphate Absorption in PXE Disease | Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum (PXE) | Pseudoxanthoma Elasticum | Drug: Disodium Pyrophosphate | Tampere University Hospital | UMC Utrecht;Hungarian Academy of Sciences | Withdrawn | 18 Years | 80 Years | All | 0 | Phase 2 | NULL |
| 5 | NCT04660461 (ClinicalTrials.gov) | February 4, 2020 | 3/12/2020 | Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) | Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) | Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) | Drug: Lansoprazole 30mg;Drug: Placebo | Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud | NULL | Recruiting | 18 Years | N/A | All | 20 | Phase 4 | Spain |
| 6 | EUCTR2019-002109-24-FI (EUCTR) | 01/08/2019 | 01/07/2019 | Oral Pyrophosphate Absorption in PXE Disease | Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum - Oral Pyrophosphate Absorption in PXE | Pseudoxanthoma elasticum;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Disodiumpyrophosphate | Tampere University Hospital | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | Finland | ||
| 7 | EUCTR2016-004021-16-ES (EUCTR) | 31/10/2018 | 10/03/2017 | Response to oral lansoprazole in patients with Pseudoxanthoma Elasticum | Response to oral lansoprazole in inorganic pyrophosphate levels in patients with Grönblad-Stranberg disease (Pseudoxanthoma Elasticum) - FIM-PXE-2016-01 | Grönblad-Stranberg disease (Pseudoxanthoma Elasticum) MedDRA version: 19.1;Level: PT;Classification code 10037150;Term: Pseudoxanthoma elasticum;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Trade Name: Lansoprazole INN or Proposed INN: Lansolprazole Other descriptive name: LANSOPRAZOLE | Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud (FIMABIS) | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | Spain | ||
| 8 | EUCTR2018-001492-20-FI (EUCTR) | 20/06/2018 | 11/06/2018 | Pyrophosphate therapy in PXE disease | Pseudoxanthoma elasticum – supplementation therapy of pyrophosphate deficiency | Pseudoxanthoma elasticum;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: SODIUM ACID PYROPHOSPHATE Product Name: SODIUM ACID PYROPHOSPHATE SAPP ROR 28 FOOD GRADE Product Code: CAS No.: 7758-16-9 | Tampere university hospital | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2;Phase 3 | Finland | ||
| 9 | NCT02537054 (ClinicalTrials.gov) | September 2015 | 21/8/2015 | Intravitreal Aflibercept for Therapy of Patients With Pseudoxanthoma Elasticum (PXE) | Intravitreal Aflibercept (Eylea) for Therapy of Choroidal Neovascularization (CNV) and Fibrovascular Proliferation (FVP) in Patients With Pseudoxanthoma Elasticum (PXE) | Pseudoxanthoma Elasticum | Drug: Aflibercept | University Hospital, Bonn | NULL | Completed | 18 Years | 65 Years | All | 15 | Phase 2 | Germany |
| 10 | EUCTR2014-005263-33-DE (EUCTR) | 29/04/2015 | 26/02/2015 | Intravitreal Aflibercept (Eylea®) for therapy of choroidal neovascularization and fibrovascular proliferation in patients with Pseudoxanthoma elasticum | Intravitreal Aflibercept (Eylea®) for therapy of choroidal neovascularization and fibrovascular proliferation in patients with Pseudoxanthoma elasticum - EyNeP | Choroidal neovascularizations and fibrovascular proliferations in patients with Pseudoxanthoma elasticum MedDRA version: 20.0;Level: PT;Classification code 10037150;Term: Pseudoxanthoma elasticum;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Trade Name: Eylea | Medizinische Fakultät der Universität Bonn | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | Germany | ||
| 11 | EUCTR2013-001421-55-FR (EUCTR) | 13/06/2013 | 22/09/2015 | An open-label Extended Clinical Protocol of ranibizumab to evaluate Safety and Efficacy in rare VEGF driven ocular diseases. - ECLIPSE | An open-label Extended Clinical Protocol of ranibizumab to evaluate Safety and Efficacy in rare VEGF driven ocular diseases. - ECLIPSE | Choroidal neovascularization not related to wet Age-related macular degeneration (wAMD), pathologic myopia (PM)or Pseudoxanthoma elasticum (PXE), as well as in Macular Edema (ME) not related to Retinal Vein Occlusion (RVO) or Diabetic macular edema (DME) and other ocular neovascularization and/or complication such as Rubeosis Iridis and Neovacular Glaucoma and Proliferative Diabetic Retinopathy requiring Vitrectomy MedDRA version: 18.0;Level: HLGT;Classification code 10047060;Term: Retina, choroid and vitreous haemorrhages and vascular disorders;System Organ Class: 10015919 - Eye disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Trade Name: Lucentis Product Name: Lucentis Product Code: RFB002A INN or Proposed INN: RANIBIZUMAB | Novartis Pharma S.A.S | NULL | Not Recruiting | Female: yes Male: yes | 500 | Phase 3 | France | ||
| 12 | NCT01525875 (ClinicalTrials.gov) | August 2012 | 1/2/2012 | Magnesium Supplements In The Treatment Of Pseudoxanthoma Elasticum (PXE) | Magnesium Supplements In The Treatment Of Pseudoxanthoma Elasticum (PXE) | Pseudoxanthoma Elasticum | Drug: Magnesium Oxide;Drug: Placebo | Mark Lebwohl | NULL | Completed | 18 Years | N/A | All | 44 | Phase 2 | United States |
| 13 | NCT00510965 (ClinicalTrials.gov) | August 2007 | 1/8/2007 | Ranibizumab to Treat Choroidal Neovascularization (CNV) in Patients With Pseudoxanthoma Elasticum (PXE) | Ranibizumab in Choroidal Neovascularization (CNV) Due to Pseudoxanthoma Elasticum (PXE, Groenblad-Strandberg-Syndrome) | Choroidal Neovascularization | Drug: Intravitreal injection ranibizumab | University Hospital, Bonn | Novartis | Completed | 18 Years | 65 Years | Both | 10 | Phase 2 | Germany |
| 14 | EUCTR2006-006231-49-DE (EUCTR) | 24/07/2007 | 06/03/2007 | Ranibizumab in choroidal neovascularization (CNV) due to Pseudoxanthoma elasticum (PXE, Groenblad-Strandberg Syndrome) - PXE-CNV 06 | Ranibizumab in choroidal neovascularization (CNV) due to Pseudoxanthoma elasticum (PXE, Groenblad-Strandberg Syndrome) - PXE-CNV 06 | Pseudoxanthoma elasticum (PXE) is an inherited systemic disease characterized by changes in the elastic tissue. Pseudoxanthoma elasticum mainly affects the skin, eyes, heart, and GI system. The cutaneous and ocular findings of PXE are referred to as Grönblad-Strandberg syndrome. Pseudoxanthoma elasticum may be autosomal dominant or autosomal recessive. In Pseudoxanthoma elasticum, choroidal nevoscualrization (CNV) is a frequently occuring complication in late stage disease MedDRA version: 8.1;Level: LLT;Classification code 10037150;Term: Pseudoxanthoma elasticum | Trade Name: Lucentis Product Name: Lucentis Product Code: RFB002 INN or Proposed INN: ranibizumab Other descriptive name: RFB002, rhuFab V2 | Department of Ophthalmology, Bonn University | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | Germany | ||
| 15 | NCT00470977 (ClinicalTrials.gov) | May 2007 | 4/5/2007 | Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy | Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy With Intravitreal Injection of Lucentis (Ranibizumab Injection) | Coats' Disease;Idiopathic Retinal Telangiectasia;Retinal Angiomatous Proliferation;Polypoidal Choroidal Vasculopathy;Pseudoxanthoma Elasticum;Pathological Myopia;Multi-focal Choroiditis;Rubeosis Iridis;Von Hippel Lindau Disease;BEST VITELLIFORM MACULAR DYSTROPHY, MULTIFOCAL (Disorder) | Drug: ranibizumab injection (0.5 mg) | Manhattan Eye, Ear & Throat Hospital | Genentech, Inc. | Completed | 18 Years | N/A | Both | 18 | Phase 1/Phase 2 | United States |