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A no-profit, open-label, phase II pilot study on the efficacy and safety of Alpelisib in patients with Dent 2 disease (ALPEDENT study)
EU CTIS ID: 2024-514196-17-00
What this study is testing
The primary aim of the study is to test the efficacy of alpelisib to increase renal uptake of 99mTc-labeled DMSA as a measure the reabsorption capacity of LMWPs by renal proximal tubules. Patients will be treated for 4 weeks at a starting oral dose of 50 mg/day for 1 week, followed by 150 mg/day for 3 weeks.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Subjects who meet all of the following criteria are eligible for this clinical trial: a. Age ≥18 years b. Genetically proven Dent 2 disease c. eGFR ≥ 50 ml/min/1.73 m2, as calculated by the CKD-EPI equation d. Expected compliance to the study protocol e. Signed Informed Consent Form (ICF)/Assent by the subject f. Since Dent 2 is an X–linked disease, only male patients will be enrolled
You likely can't join if
- Subjects meeting one or more of the following criteria cannot be included in the study: a. Patients suffering from co-morbidities, including malignancies b. Patients with any chronic infectious condition c. Patients receiving therapies not related to the OCRL mutation d. Patients with mental disabilities e. Patients with diabetes mellitus or baseline fasting glucose levels > 105 mg/dl f. Participants who do not consent to abstinence or to use a highly effective method of contraception for the duration of the study and for one week following discontinuation of alpelisib. g. Patients with hypersensitivity to the active substance or to any of the excipients of alpelisib or the 99-mTc labelled-DMSA
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.