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Piclidenoson Study of Efficacy and Safety in Patients with Lowe Syndrome

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

The study examines people with Lowe Syndrome, a rare genetic disorder that can affect the kidneys, eyes, and brain. The medication being tested is an oral tablet called Piclidenoson, taken in a small daily dose.

The purpose of the study is to determine whether the medication can improve the kidney’s ability to take up a special imaging agent called 99mTc-DMSA, which helps doctors measure how well the kidney’s tiny filtering tubes work, while also monitoring safety.

Participants will receive the tablet for about six months and will attend regular clinic visits where urine and blood are checked for signs such as LMW proteinuria (small proteins appearing in the urine) and for signs of Fanconi syndrome (a condition where the kidneys leak many substances). A simple heart test, known as an ECG, will be performed to monitor heart health, and the imaging agent will be used at the start and after treatment to compare results. The schedule includes routine visits and tests, and any side effects are recorded throughout the study.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Baseline Visit

    You attend the first study visit after enrollment. During this visit, the research team measures your kidney function using a scan called 99mTc-DMSA, which shows how well your kidneys re‑absorb small proteins. Blood samples are taken to check general health, and urine is collected to assess protein levels. Your vital signs, such as blood pressure and heart rate, are recorded, and an electrocardiogram (ECG) is performed to evaluate heart rhythm.

  2. Step 2

    Start of Medication

    You begin taking the study drug, piclidenoson. Each dose is a 6 mg tablet taken by mouth, typically once each day. You are instructed to take the tablet at the same time each day for a total treatment period of six months (approximately 26 weeks).

  3. Step 3

    Monthly Safety Checks

    Throughout the treatment period, you have regular safety check‑ins, usually once a month. During these visits, the staff measures your vital signs, asks about any new symptoms or side effects, and may draw a small amount of blood for laboratory tests. These checks help ensure that the medication is well tolerated.

  4. Step 4

    Three‑Month Assessment

    At the three‑month mark, you return for a detailed assessment. Urine is collected again to measure low‑molecular‑weight protein levels, including retinol‑binding protein and beta‑2 microglobulin. Additional tests evaluate aspects of Fanconi syndrome, such as 24‑hour urine volume and the amounts of sodium, glucose, phosphate, and amino acids in the urine, as well as serum bicarbonate in the blood. Safety evaluations, including vital signs, physical exam, laboratory tests, and ECG, are also repeated.

  5. Step 5

    Six‑Month Assessment and End of Treatment

    After six months of taking piclidenoson, you undergo a final set of evaluations. The same kidney scan (99mTc-DMSA) is repeated to compare the renal uptake with the baseline result. Urine and blood tests are performed again to assess protein loss, Fanconi syndrome parameters, and overall safety. All vital signs, physical examinations, laboratory results, and ECG findings are recorded. This visit marks the end of the medication period.

  6. Step 6

    Study Completion

    After the six‑month visit, the study team reviews all collected data to determine the effectiveness and safety of piclidenoson for treating Lowe syndrome. No further study‑related procedures are required, and you are free to continue any routine medical care as advised by your regular physician.

Who can join the trial?

6 criteria

  • Be a male who is at least 18 years old.
  • Have a confirmed diagnosis of Lowe Syndrome that is proven by genetic testing.
  • Have a kidney function measurement called estimated glomerular filtration rate (eGFR) that is 40 or higher; this number is calculated using a method called the CKD‑EPI equation.
  • Agree not to donate sperm while taking the study medicine and for at least one month after the last dose, and agree to use condoms during the study and for one month after the last dose.
  • Be able to follow the study schedule and complete all required visits and tests.
  • Be able to understand the study information and sign a written informed consent form, either personally or through a legal guardian.

Who cannot join the trial?

11 criteria

  • Taking long‑term medicines that are not for Lowe syndrome (chronic therapies: medicines used continuously over a long time).
  • Having a serious current or past medical or mental health problem (acute or chronic medical or psychiatric illness: serious health issues that happen suddenly or last a long time), such as a long‑lasting infection (systemic infection) or cancer (malignancy), that the doctor thinks could make the study unsafe or hard to finish.
  • Being in another drug or vaccine study at the same time, or having been in one within the last 30 days (participation in another investigational trial).
  • Having a kidney function test that is too low (estimated glomerular filtration rate (eGFR) < 40 mL/min/1.73 m²: a measure of how well the kidneys filter waste; a low number means poor kidney function).
  • Having liver enzyme levels that are too high (liver aminotransferase levels > 1.5 times the normal limit: blood tests that show possible liver damage).
  • Having a heart test that shows a long interval (QTcF interval > 450 milliseconds on ECG: a measurement on an electrocardiogram that can indicate risk of abnormal heart rhythms), unless the long result is caused by a known heart‑block condition (right or left bundle branch block) or a heart device (cardiac pacemaker).
  • Having any condition that raises the risk of dangerous heart rhythm problems (proarrhythmic risk), such as low potassium (hypokalemia), low magnesium (hypomagnesemia), or a genetic condition called Long QT Syndrome (congenital Long QT Syndrome).
  • Planning to take, or currently taking, any medication on the CredibleMeds list that can cause a serious heart rhythm problem called Torsades de Pointes (CredibleMeds list of drugs known to cause Torsades de Pointes).
  • Having an active stomach or intestinal disease that could stop the oral medication from being absorbed properly (active gastrointestinal disease).
  • Having an active problem with drug or alcohol dependence (active drug or alcohol dependence).
  • Taking strong medicines that speed up the body’s drug‑processing system (strong cytochrome P450 inducers), such as rifampin, phenobarbital, phenytoin, or carbamazepine.
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Investigated drugs

Piclidenoson is an oral tablet taken by patients in this study. It is being tested to see if it can help the kidneys work better at reabsorbing small proteins. The researchers will give the medicine for six months and then measure how much of a special imaging agent the kidneys take up, compared with the amount taken up before treatment. This will show whether Piclidenoson improves the kidney’s ability to handle low‑molecular‑weight proteins in people with Lowe syndrome.

What is already known about the treatment

Piclidenoson - Piclidenoson is taken as a 6 mg oral tablet that patients swallow. It is an experimental drug currently in phase 2 clinical trials and is not yet approved for general medical use. The medicine is being tested mainly for Lowe syndrome, a rare kidney disorder, and has also been studied for skin and joint inflammation. It works by activating the A3 adenosine receptor, which helps reduce inflammation and may improve kidney function, placing it in the class of A3 adenosine receptor agonists.

Investigated diseases

Lowe syndrome - Lowe syndrome is a rare genetic disorder that is present from birth and affects the eyes, kidneys, and brain. Children often develop cataracts early in life, which can cause vision problems. The kidneys show a defect in the tiny tubes that re‑absorb substances, leading to loss of nutrients and minerals in the urine. Over time this tubular dysfunction can cause a condition called Fanconi syndrome, with increased urine volume and low blood bicarbonate. Neurological development may be delayed, and some individuals have learning difficulties. The disease tends to progress slowly, with kidney and eye problems becoming more noticeable as the person grows.
Trial detailsLast updated 21 Oct 2026
Age18+ yearsPhasePhase IITrial ID2026-525495-26-00Protocol codePICLILOWEEstimated enrolment5 patientsSponsorCan-Fite Biopharma Ltd.

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