CompletedNot ApplicableInterventional

A trial to test the use of HIV drugs to treat neurofibromatosis type 2 (NF2) related tumours

Neurofibromatosis type 2 (NF2-related schwannomatosis) is a genetic condition that causes non-cancerous tumours to grow along nerves, including bumps under the skin called schwannomas. Standard treatments like surgery can be difficult and carry risks, so researchers are searching for effective tablet treatments. This trial investigates whether two existing antiviral medicines, lopinavir and ritonavir (usually used to treat HIV), can help. Laboratory tests have shown these drugs might stop tumour cells from growing. In this study, adult participants take the tablets for 30 days. Doctors will take small skin tissue samples (biopsies) and blood samples before and after the treatment to see how well the medicine reaches the tumours and how the body responds. The trial aims to find out if these medications are worth testing in larger clinical trials in the future.

At a glance

Status

Completed

Phase

Not Applicable

Sponsor

University Hospitals Plymouth NHS Trust

Enrolment target

Start

01 Apr 2024

Estimated completion

31 Mar 2026

What is this study about?

NF2-related schwannomatosis is a rare genetic condition that causes non-cancerous tumours to grow along nerves in the body. Many people with this condition develop small, benign lumps just under their skin, known as schwannomas. While they are not cancerous, they can cause pain, numbness, and other complications. At present, main treatments include surgery or radiation therapy, but these can be difficult to perform repeatedly and may damage healthy nerves.

To find better alternatives, researchers are exploring 'drug repurposing'—testing medicines that are already approved for other health conditions. In this trial, scientists are looking at lopinavir and ritonavir, two tablets widely used for many years to treat HIV. In laboratory tests, these medicines showed an ability to slow down the growth and survival of schwannoma cells. Because these drugs are already well understood and have known safety records, they are promising candidates to test in people with NF2.

The main goal of this early-stage study is to discover whether the drugs actually reach the skin tumours in adequate amounts and whether they turn off the chemical signals that make tumours grow. By understanding how the body absorbs and processes the medicine inside tumour tissue, doctors will be able to decide whether to launch larger studies to treat NF2 tumours in the future.

Key takeaways

Tests whether two existing antiviral tablets can reach and affect NF2 skin tumours.
Involves taking daily tablets for 30 days, along with two small skin biopsies and blood tests.
This is an early scientific study and is not expected to cure or shrink tumours immediately.
Participants must be 18 or older with NF2 and have a skin tumour suitable for a biopsy.
Strict birth control rules apply because the study medication interferes with hormonal contraceptives.

Who may be eligible?

This trial is open to adults aged 18 and over who have been diagnosed with NF2-related schwannomatosis and have tumours visible on scans, as well as at least one tumour under the skin that is suitable for a small tissue sample (biopsy). Participants must be generally well in their day-to-day activities, able to swallow tablets, and have reasonably healthy liver, kidney, and blood test results.

You will not be able to take part if you are allergic to lopinavir or ritonavir, or if you take certain medicines that strongly interfere with how these drugs work in the liver. People with specific serious heart rhythm problems may also be excluded for safety reasons.

Because the study drugs can stop hormonal birth control (such as the pill or implants) from working properly, women who could become pregnant must use two reliable barrier methods of contraception (such as condoms and a diaphragm) throughout the trial and for three months afterwards. A negative pregnancy test is required before starting.

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.

I am aged 18 or older.
I have a confirmed diagnosis of NF2-related schwannomatosis.
I have at least one bump or tumour just under the skin that could be sampled.
I am able to swallow tablets whole.
I am willing to follow the required birth control measures during the study.
I do not have a known allergy to lopinavir or ritonavir.

Answer every question to see your result.

What does participation involve?

Taking part in the trial lasts around two months (60 days) in total. At the start, you will visit the hospital for health checks, a blood test, and a small skin biopsy, which involves numbing the skin and taking a tiny piece of tumour tissue for lab analysis.

You will then take the trial tablets (lopinavir and ritonavir) by mouth at home every day for 30 days. On day 30, you will return to the clinic for a second skin biopsy and blood test so the team can measure drug levels in your body. Finally, you will attend a follow-up safety visit around day 60 to make sure you are feeling well and that any biopsy sites have healed properly.

Potential risks and benefits

This early-stage study is designed to gather scientific information rather than shrink your tumours, so you may not experience any direct health benefits from taking part. The main risks include temporary soreness, bruising, bleeding, or small scars from the skin biopsies and blood tests. As with any medication, the trial tablets can cause mild side effects like an upset stomach, though your health will be watched closely by the clinical team throughout. Taking part is completely voluntary, and you are free to leave the study at any time without it affecting your usual NHS care.

Locations (2)

Derriford Hospital
Approximate
Plymouth, England
Manchester Centre for Clinical Neurosciences
City only
Salford, England

Common questions

Does taking these drugs mean I have HIV?

No. While these tablets were created to treat HIV, researchers are testing them here entirely for their separate ability to block tumour growth in NF2.

Will this trial make my tumours disappear?

Probably not. This is an early exploratory trial lasting only 30 days, designed to check if the medicine reaches the tumours, rather than to provide a cure.

Does a skin tumour biopsy hurt?

The area is numbed with a local anaesthetic injection first so you should not feel pain during the procedure, although the site may feel tender or bruised for a few days afterwards.

Why do I need to use barrier contraception?

The study drugs can stop hormonal contraceptives (like the pill, patch, or injection) from working correctly. Using two barrier methods (like condoms and a cap) prevents accidental pregnancy during the trial.

What happens if I change my mind during the study?

You can leave the trial at any point without giving a reason. Your doctors will continue to look after you using your normal treatment plan.

How to find out more

Katherine Beaney

katherine.beaney@plymouth.ac.uk - Official trial record

Always speak to your GP or specialist before deciding to take part in a study.