Lowe SyndromePhase 2NCT07410455
An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome
A Phase 2 study testing Piclidenoson in people with Lowe Syndrome, run by Can-Fite BioPharma at 1 site in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Lowe Syndrome
- What is being tested?
- Piclidenoson
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteItaly
- Study dates
- 1 Jun 2026to 31 Aug 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site
IRCCS Ospedale Pediatrico Bambino Gesù
Roma, 00165
Clinical Trials Office, +39 06 68591
About this study
The primary objective of this trial is to:
- 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:
- Evaluate changes in urinary excretion of LMWPs and other clinical parameters of renal Fanconi syndrome
- Evaluate safety of piclidenoson in patients with Lowe syndrome
Read the detailed description
Objectives
- 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.
- 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.
- Conditions
- Lowe Syndrome
- Being tested
- Piclidenoson
- Keywords
- Lowe Syndrome, Piclidenoson, CF101, Can-Fite
- Medical subject headings
- Oculocerebrorenal Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PiclidenosonDrug
Piclidenoson will be administered orally at a dose of 3 mg twice per day for 6 months
| Study group | Role | What they receive |
|---|---|---|
| Piclidenoson (CF101) open-label treated patients | Experimental | Piclidenoson will be administered orally at a dose of 3 mg twice per day for 6 months Receives: Drug: Piclidenoson |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 6 months
Secondary outcomes (6)
beta-2 microglobulin
Change in low-molecular weight 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
Measured: 3 and 6 months of treatment
urinary excretion of sodium
Change in the Fanconi syndrome as assessed by 24-hour urine volume, as compared to baseline; urinary excretion of sodium, glucose, phosphate, and amino acids; and changes in serum bicarbonate, after 3 and 6 months of treatment
Measured: 3 and 6 months
urinary excretion glucose
Change in 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
Measured: 3 and 6 months
urinary excretion of phosphate
Change in 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
Measured: 3 and 6 months
urinary excretion of amino acids
Change in 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
Measured: 3 and 6 months
serum bicarbonate
Change in 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
Measured: 3 and 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2026
- Study start1 Jun 2026 (estimated)
- Primary completion31 Aug 2027 (estimated)
- Study completion31 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lowe SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Can-Fite BioPharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Francesco, MD, Principal investigator, Emma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07410455
- Sponsor's protocol ID
- PICLILOWE
- Status last verified
- Feb 2026
- First submitted
- 8 Feb 2026
- Last updated
- 18 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome
- ClinicalTrials.gov ID
- NCT07410455
- Acronym / sponsor ID
- PICLILOWE
- Conditions
- Lowe Syndrome
- Keywords
- Lowe Syndrome, Piclidenoson, CF101, Can-Fite
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Can-Fite BioPharmaIndustry (pharma, biotech, devices)
- Study start
- 1 Jun 2026 (estimated)
- Primary completion
- 31 Aug 2027 (estimated)
- Study completion
- 31 Aug 2027 (estimated)
- First posted
- 13 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this trial is to:
- 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:
- Evaluate changes in urinary excretion of LMWPs and other clinical parameters of renal Fanconi syndrome
- Evaluate safety of piclidenoson in patients with Lowe syndrome
Detailed description
Objectives
- 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.
- 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Piclidenoson (CF101) open-label treated patients | Experimental | Drug: PiclidenosonPiclidenoson will be administered orally at a dose of 3 mg twice per day for 6 months |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (10 listed)
- 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/;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PiclidenosonPiclidenoson will be administered orally at a dose of 3 mg twice per day for 6 months | Drug | Piclidenoson (CF101) open-label treated patients |
Primary outcome
99mTc-DMSA
Time frame: 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
6 secondary outcomes
beta-2 microglobulin
Time frame: 3 and 6 months of treatment
urinary excretion of sodium
Time frame: 3 and 6 months
urinary excretion glucose
Time frame: 3 and 6 months
urinary excretion of phosphate
Time frame: 3 and 6 months
urinary excretion of amino acids
Time frame: 3 and 6 months
serum bicarbonate
Time frame: 3 and 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Francesco, MD principal investigator
Emma
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Piclidenoson in people with Lowe Syndrome, run by Can-Fite BioPharma at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 18 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Zivit Harpaz (+972 3 924 1114, Zivit@canfite.co.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
99mTc-DMSA (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Feb 2026.