An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome

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

Timeline

Start

2026-06-01

Primary completion

2027-08-31

Completion

2027-08-31

Drugs

Indications