AuthorisedTherapeutic exploratory (Phase II)Interventional

An open-label, phase 2 pilot study on the efficacy and safety of Piclidenoson in patients with Lowe syndrome

Lowe syndrome is a rare genetic condition that can affect the eyes, brain, and kidneys. One common problem is that the kidneys struggle to hold onto essential nutrients, causing them to leak out into the urine. This Phase 2 trial is investigating whether a new medicine called Piclidenoson can help the kidneys work better. Researchers will monitor participants over six months to see if kidney function improves and to ensure the treatment is safe. This is an 'open-label' study, meaning everyone who takes part will receive the active study medicine. No one will receive a dummy pill (placebo). Throughout the trial, doctors will track kidney health using special scans, blood tests, and urine tests. The trial is open to men aged 18 and older with Lowe syndrome. Taking part could help doctors understand whether Piclidenoson offers a new way to support kidney health in people living with this condition.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Can-Fite Biopharma Ltd.

Enrolment target

Start

19 Oct 2026

Lowe Syndrome

What is this study about?

Lowe syndrome, also known as oculocerebrorenal syndrome, is a rare genetic condition that mainly affects males. It causes symptoms in several parts of the body, particularly the eyes, brain, and kidneys. In the kidneys, it often leads to a condition where the tiny filtering tubes cannot reabsorb vital nutrients, such as proteins, sugars, and salts. Over time, losing these essential substances can weaken bones and affect overall kidney health.

This clinical trial is testing an experimental medicine called Piclidenoson. Researchers want to find out whether Piclidenoson can improve how well the kidneys filter and absorb nutrients. Because this is a Phase 2 'pilot' study, the main goals are to see if the medicine works as hoped and to check that it is safe and well tolerated by participants.

At present, there is no cure for Lowe syndrome, and treatments mostly focus on managing individual symptoms. If this study shows positive results, Piclidenoson could become a valuable new treatment option to help protect kidney function and improve quality of life for people living with the condition.

Key takeaways

The trial tests whether Piclidenoson can safely improve kidney function in Lowe syndrome.
The study lasts at least six months and involves regular check-ups, scans, and urine tests.
Every participant receives the active medicine; no dummy pills are used.
The trial is open to men aged 18 and older with Lowe syndrome.
Participation is completely voluntary and you can withdraw at any time.

Who may be eligible?

This study is open to men aged 18 and older who have a confirmed diagnosis of Lowe syndrome. Because the condition is linked to the X chromosome, it almost exclusively affects males.

To take part, participants must be able to attend regular hospital appointments and complete the required health checks. These checks include kidney scans, blood tests, and 24-hour urine collections.

The study team will review each person's medical history and current treatments to ensure that joining the study is safe for them. Certain other health conditions or medications may mean someone cannot participate.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you male?
Are you aged 18 or older?
Do you have a confirmed medical diagnosis of Lowe syndrome?
Are you able and willing to attend hospital visits for at least six months?
Are you comfortable having kidney scans, blood tests, and collecting urine samples?

Answer every question to see your result.

What does participation involve?

Taking part in this study lasts for at least six months. Participants will take the study medicine, Piclidenoson, as directed by the research team. Because this is an open-label trial, all participants receive the actual medicine rather than a dummy pill. During the study, you will visit the clinic regularly for health checks, including physical examinations, blood pressure measurements, and heart traces (ECGs). Doctors will test how well the kidneys are working using regular blood tests and 24-hour urine collections at three and six months. You will also have a specialised kidney scan (called a DMSA scan) at the start and after six months to measure kidney function. After the six-month treatment period ends, the research team will follow up to check your ongoing health.

Potential risks and benefits

Participants might see an improvement in their kidney function, and will receive close, expert medical monitoring throughout the trial. However, there is no guarantee that the study medicine will help. Like all new medicines, Piclidenoson can cause unexpected side effects, and having regular blood tests, scans, and urine collections requires time and effort. Taking part is completely voluntary. You can choose to leave the study at any time without giving a reason, and your regular NHS medical care will not be affected.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Italy

Common questions

What is an open-label study?

An open-label study means that both you and the doctors know you are taking the actual study medicine. There is no dummy pill (placebo).

What is a DMSA kidney scan?

A DMSA scan is a routine hospital scan that uses a tiny, safe amount of a radioactive tracer to produce detailed pictures showing how well the kidneys are working.

Why is the study only open to men?

Lowe syndrome is a genetic condition carried on the X chromosome. Because of how it is inherited, it almost exclusively affects males.

What is a 24-hour urine collection?

It involves collecting all the urine you pass over a full 24-hour period in a special container. This helps doctors measure exactly what nutrients the kidneys are losing.

Can I stop taking part if I change my mind?

Yes. You are free to leave the trial at any point, and your standard medical care will continue as normal.