Pseudoxanthoma Elasticum clinical trials
The registry lists 32 studies for Pseudoxanthoma Elasticum across 8 countries. 6 are recruiting or about to start. 30 of them are mainly about Pseudoxanthoma Elasticum (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-19.
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Who runs them
University Hospital, Angers (9) · University Hospital, Bonn (4) · Inozyme Pharma (4) · Centre Hospitalier Universitaire de Nice (2) · Daiichi Sankyo (2) · National Cancer Institute (NCI) (1) · UMC Utrecht (1) · European Georges Pompidou Hospital (1)
What is being tested here
Drugs under test in Pseudoxanthoma Elasticum studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Etidronic Acid | 1 | 1 | 1 |
| Efmirenpase alfa | 0 | 0 | 3 |
| Ranibizumab | 0 | 0 | 2 |
| Magnesium Oxide | 0 | 0 | 1 |
| Aflibercept | 0 | 0 | 1 |
| Lansoprazole | 0 | 0 | 1 |
Drug classes tested here Angiogenesis inhibitors 3
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 5, phase 3 first
- Mar 2027The Prevention of Systemic Ectopic Mineralization in Pseudoxanthoma Elasticum · Phase 3 · UMC Utrecht
- Nov 2026CHOPXE - Analysis of Choriocapillaris Flow Deficits in Patients With Pseudoxanthoma Elasticum · University Hospital, Angers
- Dec 2026PPI Supplementation to Fight ECtopIc Calcification in PXE · Centre Hospitalier Universitaire de Nice
- Jan 2027Purinergic Compounds in Pseudoxanthoma Elasticum · Centre Hospitalier Universitaire de Nice
- Oct 2027Progression Assessment of PXE-associated Alterations · University Hospital, Bonn
Who these studies take
Of the 6 Pseudoxanthoma Elasticum studies recruiting now, 4 (67%) set no upper age limit and 2 (33%) stop at 75 or younger. 2 (33%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 2 (33%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 2 (33%) | 2 (33%) |
| 18 | 6 (100%) | 6 (100%) |
| 30 | 6 (100%) | 6 (100%) |
| 40 | 6 (100%) | 6 (100%) |
| 50 | 6 (100%) | 6 (100%) |
| 65 | 5 (83%) | 5 (83%) |
| 75 | 4 (67%) | 4 (67%) |
| 85 | 4 (67%) | 4 (67%) |
Across all 32 studies, recruiting or not: 28% stop at 75 or younger and 19% at 65; 38% take children; 0% are for women only and 0% for men only; 13% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 13, 2026.
Results posted1
Stopped2
- ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 DeficiencyTerminatedOct 19, 2026NCT06462547
- The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 DeficiencyTerminatedOct 15, 2026NCT05734196
Studies
Recruiting first, then most recently updated. Showing 32 of 32. search them.
- Purinergic Compounds in Pseudoxanthoma ElasticumRecruitingNCT07323082Not applicable1 country
- Impact of Optimized Recruitment and Follow-up of Patients With Pseudoxanthoma Elasticum (PXE)RecruitingNCT066363441 country
- CHOPXE - Analysis of Choriocapillaris Flow Deficits in Patients With Pseudoxanthoma ElasticumRecruitingNCT07006649Not applicable1 country
- Progression Assessment of PXE-associated AlterationsRecruitingNCT070481061 country
- PPI Supplementation to Fight ECtopIc Calcification in PXERecruitingNCT04868578Not applicable1 country
- The Prevention of Systemic Ectopic Mineralization in Pseudoxanthoma ElasticumRecruitingNCT05832580Phase 31 country
- ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 DeficiencyTerminatedNCT06462547Phase 24 countries
- The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 DeficiencyTerminatedNCT05734196Phase 13 countries
- Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXECompletedNCT05030831Phase 1/22 countries
- Natural History Study: ENPP1 Deficiency or the Early-Onset Form of ABCC6 DeficiencyCompletedNCT077451794 countries
- Study of People With Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2)CompletedNCT034788391 country
- Progression Rate of Pseudoxanthoma Elasticum-associated Choroidal and Retinal DegenerationActive, not recruitingNCT056620851 country
- A Study of DS-1211b in Individuals With PseudoXanthoma ElasticumCompletedNCT05569252Phase 22 countries
- The Characteristics of Backscattering With Depth in the Progression of KeratoconusCompletedNCT060506291 country
- Employment of Patients With Pseudoxanthoma ElasticumCompletedNCT052461891 country
- Pseudoxanthoma Elasticum (PXE) Natural History Biomarkers in PXE Individuals and Their Biological Non-PXE SiblingsCompletedNCT050257221 country
- Oral Pyrophosphate Absorption in PXE DiseaseWithdrawnNCT04441671Phase 2
- Magnesium Supplements In The Treatment Of Pseudoxanthoma Elasticum (PXE)CompletedNCT01525875Phase 21 country
- Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)Status unknownNCT04660461Phase 41 country
- Genetic Analysis of Patients With Pseudoxanthoma ElasticumCompletedNCT003414191 country
- Intravitreal Aflibercept for Therapy of Patients With Pseudoxanthoma Elasticum (PXE)CompletedNCT02537054Phase 21 country
- Intestinal Microbiota and Vitamin K Levels in PXE Patients (IMPROVE Study)Status unknownNCT03813550Not applicable2 countries
- Natural History of GACI With or Without ARHR2 or PXEStatus unknownNCT037585341 country
- What's Happen Under the Calcification Process in Pseudoxanthoma ElasticumCompletedNCT03070860Not applicable1 country
- Characterization of Pseudoxanthoma ElasticumStatus unknownNCT021083921 country
- Biological Collection of Kidney CellsStatus unknownNCT03364504Not applicable
- Arterial Wall Calcium Load in Pseudoxanthoma ElasticumStatus unknownNCT017310801 country
- Functional and Structural Characterization of Arteriopathy in Pseudoxanthoma Elasticum (PXE)CompletedNCT014463931 country
- Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related MaculopathyCompletedNCT00470977Phase 1/21 country
- Ranibizumab to Treat Choroidal Neovascularization (CNV) in Patients With Pseudoxanthoma Elasticum (PXE)CompletedNCT00510965Phase 21 country
- Phenotypic Expressions in a French Pseudoxanthoma-Elasticum CohortStatus unknownNCT014463801 country
- Evolution of Visual Impairment During Pseudoxanthoma ElasticumCompletedNCT005551131 country