About this trial
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
Eligibility criteria
Qualifiers
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;
Disqualifiers
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;
Trial design
Treatments tested in this trial
- Piclidenoson