An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome
brief 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
detailed description
Objectives
1. Primary Objective:
\- The primary objective of the study is to test the efficacy of piclidenoson to increase renal uptake of 99mTc-labeled DMSA after 6 months (26 weeks) of treatment as a measure the reabsorption capacity of LMWPs by renal proximal tubules. 2. Secondary Objectives of the study are:
* to evaluate changes in urinary excretion of LMWPs and other clinical parameters of renal Fanconi syndrome, * to evaluate the safety of piclidenoson in patients with Lowe syndrome.
Primary Endpoint
\- Improvement in the renal uptake, as compared to Baseline, of 99mTc-DMSA after 6 months (26 weeks) of treatment with piclidenoson (a p-value of ≤ 0.05 will be used to determine statistical significance), as a measure of the reabsorption capacity of LMWPs by renal proximal tubules.
Secondary Endpoints
* Improvement of LMW 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 (a p-value of ≤ 0.05 will be used to determine statistical significance). * Improvement of 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 (a p-value of ≤ 0.05 will be used to determine statistical significance). * Safety of piclidenoson in Lowe syndrome including treatment-emergent adverse events (TEAEs) and changes in vital signs, physical examination, neurological examination, clinical laboratory tests (liver, kidney, hematology, chemistry and urinalysis), and ECG.