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 range18 Years
SexMALE
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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…