Recruiting soon

Piclidenoson for Improved Renal Function in Lowe Syndrome Patients

Study Aim

This study aims to evaluate if 6 months of treatment with Piclidenoson can improve the functioning of renal proximal tubular cells (PTCs) in patients with Lowe Syndrome, as measured by an increase in 99mTc-DMSA uptake compared to the beginning of the study.

What is being tested

Piclidenoson

Drug

Who is being recruted

Congenital Abnormalities+19

+ Abnormalities, Multiple

+ Urogenital Diseases

Over 18 Years

+16 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 2

Interventional

Study Start: June 2026

See protocol details

Summary

Principal SponsorCan-Fite BioPharma

Study ContactZivit Harpaz

Last updated: February 14, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: June 1, 2026

Actual date on which the first participant was enrolled.

This study focuses on a drug called piclidenoson and its potential benefits for patients with Lowe Syndrome, a rare genetic disorder affecting the kidneys, eyes, and brain. The main goal is to see if piclidenoson can improve the kidneys' ability to reabsorb certain proteins, which is often impaired in Lowe Syndrome. This research is important as it could lead to a new treatment option for this condition, potentially improving the quality of life for those affected. During the study, participants will receive piclidenoson for a period of 6 months. The effectiveness of the drug will be measured by observing changes in the kidneys' ability to reabsorb proteins. This will be done using a special type of scan called a 99mTc-DMSA scan. The study will also monitor any changes in the symptoms of Lowe Syndrome and will keep track of any potential side effects of piclidenoson. The safety of the drug is another important aspect being evaluated in this study.

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

NCT07410455

Principal SponsorCan-Fite BioPharma

Study ContactZivit Harpaz

Last updated: February 14, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

5 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Male

Biological sex of participants that are eligible to enroll.

Over 18 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Congenital AbnormalitiesAbnormalities, MultipleUrogenital DiseasesBrain DiseasesBrain Diseases, MetabolicCentral Nervous System DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsKidney DiseasesMetabolic DiseasesMetabolism, Inborn ErrorsCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNutritional and Metabolic DiseasesOculocerebrorenal SyndromeUrologic DiseasesRenal Tubular Transport, Inborn ErrorsAmino Acid Transport Disorders, InbornBrain Diseases, Metabolic, InbornGenetic Diseases, InbornGenetic Diseases, X-LinkedFemale Urogenital DiseasesMale Urogenital Diseases

Criteria

6 inclusion criteria required to participate

Males 18 years and above;

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 understand and provide written informed consent (subject or legal guardian).

Estimated glomerular filtration rate (eGFR) ≥ 40 mL/min/1.73m2, as calculated by the CKD-EPI equation;

Show More Criteria

10 exclusion criteria prevent from participating

Active gastrointestinal disease which could interfere with the absorption of oral medication;

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);

Liver aminotransferase levels greater than 1.5 times the laboratory's upper limit of normal;

Subjects receiving chronic therapies not related to Lowe syndrome; Estimated glomerular filtration rate (eGFR) <40 mL/min/1.73m2 by the CKD-EPI equation;

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

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

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Suspended

IRCCS Ospedale Pediatrico Bambino Gesù

Roma, ItalyOpen IRCCS Ospedale Pediatrico Bambino Gesù in Google Maps

Recruiting soonOne Study Center