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

NCT07410455 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: PICLILOWE
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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. 1.Evaluate changes in urinary excretion of LMWPs and other clinical parameters of renal Fanconi syndrome
2. 2.Evaluate safety of piclidenoson in patients with Lowe syndrome

Conditions

Lowe Syndrome

Keywords

Lowe Syndrome; Piclidenoson; CF101; Can-Fite

Outcome Measures

Primary Outcomes (1)

• 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

  6 months

Secondary Outcomes (6)

• beta-2 microglobulin

  3 and 6 months of treatment

• urinary excretion of sodium

  3 and 6 months

• urinary excretion glucose

  3 and 6 months

• urinary excretion of phosphate

  3 and 6 months

• urinary excretion of amino acids

  3 and 6 months

Study Arms (1)

Piclidenoson (CF101) open-label treated patients

EXPERIMENTAL

Piclidenoson will be administered orally at a dose of 3 mg twice per day for 6 months

Drug: Piclidenoson

Interventions

Piclidenoson DRUG

Piclidenoson will be administered orally at a dose of 3 mg twice per day for 6 months

Piclidenoson (CF101) open-label treated patients

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Can-Fite BioPharma: lead

Study Sites (1)

IRCCS Ospedale Pediatrico Bambino Gesù

Roma, 00165, Italy

Location

MeSH Terms

Conditions

Oculocerebrorenal Syndrome

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Renal Tubular Transport, Inborn Errors; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Amino Acid Transport Disorders, Inborn; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Francesco, MD

  Emma

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Zivit Harpaz

CONTACT

+972 3 924 1114; Zivit@canfite.co.il

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 8, 2026
• First Posted: February 13, 2026
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): August 31, 2027
• Study Completion (Estimated): August 31, 2027
• Last Updated: February 18, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)