Starting soon
Phase 2
An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome
Condition(s) studied
Lowe Syndrome
Investigational drug(s) / intervention(s)
Piclidenoson
Piclidenoson: Piclidenoson will be administered orally at a dose of 3 mg twice per day for 6 months
Study 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
Eligibility
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 medication;
* Active drug or alcohol dependence;
* Concomitant use of strong cytochrome P450 inducers, e.g., rifampin, phenobarbital, phenytoin, carbamazepine;
* Significant acute or chronic medical or psychiatric illness, including chronic systemic infection or malignancy, that, in the judgment of the Investigator, could compromise subject safety, limit the subject's ability to complete the study, and/or compromise the objectives of the study; and
* Participation in another investigational drug or vaccine trial concurrently or within 30 days prior to the Screening visit.
Primary outcome measure(s)
- 99mTc-DMSA — 6 months
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
Trial sites (1)
| Facility | City | Region | Status |
|---|
| IRCCS Ospedale Pediatrico Bambino Gesù |
Roma |
Italy |
|
