NIHR supports UK trial to treat Scleroderma with novel drug

Scleroderma causes scarring of skin and internal organs including blood vessels 

Scleroderma or Systemic Sclerosis is an autoimmune disease. This is where the immune system attacks and damages the bodies’ own tissues. It can cause scarring of tissues and blood vessels. This results in hard, thickened areas of skin and problems with muscles, bones and internal organs. Symptoms usually start in the skin on the hands and gradually progress to other parts of the body and internal organs, including the lungs. More than 50% of people with scleroderma develop disabilities due to having limited use of their hands.

When the lungs are affected, it can lead to a lung disease known as pulmonary fibrosis and/or high blood pressure in the blood vessels that supply the lungs. This is known as pulmonary hypertension. As a result, people experience shortness of breath, tiredness and chest pain, among other life-limiting symptoms. People with scleroderma have the highest risk of death when the lungs are affected. 

Treatments for high blood pressure in the lungs in people with scleroderma do not reduce thickened skin and tissue

There are drugs used to treat and reduce the progression of high blood pressure in the lungs. However, they are limited in their ability to treat other types of scleroderma. A new drug is therefore planned for development by the pharmaceutical company, Boehringer Ingelheim (BI). The drug, Avenciguat, aims to reduce the thickening of blood vessels and tissues. This is the first time that this drug has been tested in scleroderma. The ultimate aim is to assess how effective the drug is for one or more of the clinical types of the disease. Currently, the effectiveness of Avenciguat is being tested via a Phase 2 trial at 150 sites across the world. This includes sites in Europe, US, South America and China. In 2023, BI approached Francesco Del Galdo, Professor of Experimental Medicine and Lead of the Scleroderma Research Programme at the University of Leeds, to ask for his support in trialling Avenciguat in the UK. 

MSK-TRC supports the opening of new clinical trial sites in the UK 

Professor Del Galdo proposed BI’s Phase 2 trial to members of the UK Musculoskeletal Translational Research Collaboration (MSK-TRC). The aim was to request the TRC’s support in delivering this study in the UK. TRC members completed a feasibility questionnaire to assess if their sites would qualify to participate. Professor Del Galdo praised the TRC for being able to provide him with access to a network of researchers to open more UK trial sites. 

“I suggested to BI that the network of the UK MSK-TRC could support expanding the delivery of the trial. We highlighted the importance of the unmet need for this study to TRC members. As a result, all of the NIHR infrastructure can now access this feasibility questionnaire” - Professor Del Galdo.  

The UK MSK TRC’s support has meant that BI were able to open the clinical trial within other UK regions which may not previously have been involved. This enabled the TRC to meet one of its goals: to foster collaboration and deliver experimental studies at scale through shared infrastructure resources.

Next steps

The study is actively recruiting and currently involves 200 patients from more than 20 countries globally. Those currently enrolled in the Phase 2 trial will benefit from more frequent health evaluations compared to normal NHS standards of care. Once the trial is complete, there will be an option available to those experiencing positive outcomes with Avenciguat to continue receiving the drug. The next phase of clinical trials will be to further examine the effectiveness of the drug in a larger group of patients with specific types of scleroderma.