An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lo… (NCT07410455) | Clinical Trial Compass
Not Yet RecruitingPhase 2
An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome
Italy5 participantsStarted 2026-06-01
Plain-language summary
The primary objective of this trial is to:
1\. Evaluate the efficacy of piclidenoson to increase renal uptake of 99mTc-labeled DMSA, in comparison to baseline, after 6 months (26 weeks) of treatment as a measure the reabsorption capacity of LMWPs by renal proximal tubules.
The secondary objectives of this trial are to:
1. Evaluate changes in urinary excretion of LMWPs and other clinical parameters of renal Fanconi syndrome
2. Evaluate safety of piclidenoson in patients with Lowe syndrome
Who can participate
Age range
18 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Males 18 years and above;
* Documentation of genetically-proven Lowe Syndrome;
* Estimated glomerular filtration rate (eGFR) ≥ 40 mL/min/1.73m2, as calculated by the CKD-EPI equation;
* Male subjects must refrain from sperm donation during treatment and until at least 1 month after the last dose of study medication. Male subjects must agree to use condoms throughout the course of the trial and for 1 month after the last dose of study medication;
* Ability to complete the study in compliance with the protocol; and
* Ability to understand and provide written informed consent (subject or legal guardian).
Exclusion Criteria:
* Subjects receiving chronic therapies not related to Lowe syndrome; Estimated glomerular filtration rate (eGFR) \<40 mL/min/1.73m2 by the CKD-EPI equation;
* Liver aminotransferase levels greater than 1.5 times the laboratory's upper limit of normal;
* QTcF interval \> 450 milliseconds (msec) on ECG (average of triplicate ECGs) (except when QT prolongation is associated with right or left bundle branch block or cardiac pacemaker, in which case enrollment is allowed);
* A condition which increases proarrhythmic risk, including hypokalemia, hypomagnesemia, or congenital Long QT Syndrome;
* Ongoing or planned use of a concomitant medication that is on the CredibleMedsTM list of drugs known to cause Torsades des Pointes; https://crediblemeds.org/;
* Active gastrointestinal disease which could interfere with the absorption of oral medicat…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.