NCT05832580 The Prevention of Systemic Ectopic Mineralization in Pseudoxanthoma Elasticum
| NCT ID | NCT05832580 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | UMC Utrecht |
| Condition | Pseudoxanthoma Elasticum |
| Study Type | INTERVENTIONAL |
| Enrollment | 76 participants |
| Start Date | 2023-04-26 |
| Primary Completion | 2027-03-29 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 76 participants in total. It began in 2023-04-26 with a primary completion date of 2027-03-29.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The goal of this randomized clinical trial is to assess the effect of etidronate on ectopic calcification in relatively young patients with Pseudoxanthoma elasticum. The main question it aims to answer are: What is the difference in the arterial calcification scores in the legs and the carotid syphon measured on low-dose CT scan after 24 months of treatment compared to baseline between etidronate and placebo. Participants will be asked to do take etidronate or placebo for 24 months.
Eligibility Criteria
Inclusion Criteria: In order to be eligible to participate in this study, a subject must meet all of the following criteria: * 1\. Be between 18 years and 50 years. * 2\. Have a definitive diagnosis of PXE according to the Plomp criteria, which confirm a diagnosis of PXE when at least two (or more) criteria not belonging to the same category (skin, eye, genetic) are met: 1. Skin 1. Yellowish papules and/or plaques on the lateral side of the neck and/or flexural areas of the body or 2. Increase of morphologically altered elastin with fragmentation, clumping and calcification of elastic fibers in a skin biopsy taken. 2. Eye 1. Peau d'orange of the retina or 2. One or more angioid streaks (AS), each at least as long as one disk diameter. When in doubt, fluorescein or indocyanine green angiography of the fundus is needed for confirmation. 3. Genetics 1. A pathogenic mutation of both alleles of the ABCC6 gene or 2. A first-degree relative (parent, sibling or child) who meets independently the diagnostic criteria for definitive PXE * 3\. Fertile women must take adequate anticonception. Exclusion Criteria: A potential subject who meets any of the following criteria will be excluded from participation in this study: 1. Patients that are unable or unwilling to sign for informed consent. 2. Pregnant, lactating, or fertile women who might wish to become pregnant within three years.\*\* 3. Patients with an estimated glomerular filtration rate below 30 ml/min/1.73m2 according to the CKD-EPI equation.31 4. Patients with a known abnormality of the oesophagus that would interfere with passage of the drug (e.g. oesophagus stenosis). 5. Patients with chronic diarrhoea (\> 1 month). 6. Patients with known osteomalacia; 7. Patients with hypocalcaemia (calcium \<2.20 mmol/L corrected for albumin)\*. 8. Patients with a vitamin D deficiency (\<35 nmol/L)\*. 9. Patients that used a bisphosphonate in the last 5 years. 10. Patients with known sensitivity to etidronate. 11. Any other medical or social condition that, at the discretion of the Principal Investigator, might put the subject at risk of harm during the study or might adversely affect the interpretation of the study data. * After correction a patient is again suitable for participation, as long as inclusion criteria are met (more information in paragraph 7.3.3). * Subjects that become pregnant during the trial will be excluded from further participation.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05832580 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 50 Years, studying Pseudoxanthoma Elasticum. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05832580 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 76 participants.
Is NCT05832580 currently recruiting?
Yes, NCT05832580 is actively recruiting participants. Contact the research team at w.spiering@umcutrecht.nl for enrollment information.
Where is the NCT05832580 trial being conducted?
This trial is being conducted at Utrecht, Netherlands.
Who is sponsoring the NCT05832580 clinical trial?
NCT05832580 is sponsored by UMC Utrecht. The trial plans to enroll 76 participants.