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
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;
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;
Single group
Piclidenoson
DrugPiclidenoson will be administered orally at a dose of 3 mg twice per day for 6 months
99mTc-DMSA
The primary end-point is to assess whether treatment for 6 months with piclidenoson improves the functioning of renal proximal tubular cells (PTCs), as assessed by the increase in 99mTc-DMSA uptake as compared to Baseline
beta-2 microglobulin
Change in low-molecular weight proteinuria as assessed by changes urinary excretion of retinol-binding protein and beta-2 microglobulin, as compared to baseline, after 3 and 6 months of treatment
urinary excretion of sodium
Change in the Fanconi syndrome as assessed by 24-hour urine volume, as compared to baseline; urinary excretion of sodium, glucose, phosphate, and amino acids; and changes in serum bicarbonate, after 3 and 6 months of treatment
urinary excretion glucose
Change in Fanconi syndrome as assessed by 24-hour urine volume; urinary excretion of sodium, glucose, phosphate, and amino acids; and changes in serum bicarbonate, after 3 and 6 months of treatment, as compared to baseline
urinary excretion of phosphate
Change in Fanconi syndrome as assessed by 24-hour urine volume; urinary excretion of sodium, glucose, phosphate, and amino acids; and changes in serum bicarbonate, after 3 and 6 months of treatment, as compared to baseline
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