The TEMP-PREVENT trial: a study protocol for a randomized, double-blind, placebo-controlled clinical trial of etidronate for treatment in young adult patients with pseudoxanthoma elasticum
PXE is a rare genetic disease for which there is currently no registered treatment. The TEMP-PREVENT trial is set out to evaluate the effects of etidronate in young adult patients with PXE. TRIAL REGISTRATION: The trial is registered at the Dutch Central Committee on Research Involving Human Subjects (CCMO) (registration number: NL75350.041.21), European Union Clinical Trials Register (EudraCT number: 2021-000434-34), and at ClinicalTrials.gov (ID: NCT05832580, registration date 2023-04-14).
Authors
- Wilko Spiering (ORCID: https://orcid.org/0000-0002-2493-6407)
- Jeannette Ossewaarde‐van Norel (ORCID: https://orcid.org/0000-0003-2864-6025)
- Evelien van Valen (ORCID: https://orcid.org/0000-0001-7412-2404)
- Tim C. van den Beukel (ORCID: https://orcid.org/0000-0003-3597-6217)
- Margje H. Haverkamp
- Redmer van Leeuwen (ORCID: https://orcid.org/0000-0002-9007-7276)
- Iris M Harmsen
- Pim A. de Jong
- Madeleine Kok
Institutions
- Utrecht University (NL)
- University Medical Center Utrecht (NL)
Publication Details
- Journal
- Trials
- Published
- 2025-10-23
- DOI
- https://doi.org/10.1186/s13063-025-09064-6
- Primary Topic
- Dermatological and Skeletal Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Ögonfonden
- Oogfonds
- Stichting Dioraphte