A Phase 2 Randomized, Double-blind, Placebo-Controlled Study to Assess the Safety and Efficacy of MTX-474 in the Treatment of Participants with Diffuse Cutaneous Systemic Sclerosis (dcSSc)
Diffuse cutaneous systemic sclerosis is a long-term condition where the immune system causes skin on many parts of the body to become tight, thick, and hard. It can also affect internal organs. Currently, treatments are limited. This Phase 2 study looks at an experimental medicine called MTX-474 to see how well it works and how safe it is. Adults taking part will receive either the study medicine or an inactive drip (a dummy treatment called a placebo). Doctors will check participants' skin thickness over 24 weeks using a standard physical examination. They will also run blood tests and take small skin samples to see how the medicine behaves in the body and whether it improves symptoms. The goal is to discover whether MTX-474 could become a helpful new treatment option.
At a glance
What is this study about?
Systemic sclerosis, sometimes called scleroderma, is a rare autoimmune condition. In the 'diffuse' type, the body makes too much collagen, causing skin over the arms, legs, and chest to become thick, hard, and tight. This can make moving joints difficult and uncomfortable, and it can sometimes involve internal organs like the lungs, heart, or kidneys. Finding treatments that can stop or reverse this skin hardening is an important goal for medical researchers.
This clinical trial is testing a new drug called MTX-474. Scientists want to find out whether MTX-474 can reduce skin tightness and whether it is safe for people to take. Because this is a Phase 2 study, the main goal is to explore how effective the drug is and to look closely for any unwanted side effects.
To make sure the results are fair and reliable, the study is 'double-blind' and 'placebo-controlled'. This means half of the participants receive MTX-474, while the other half receive a dummy drip containing simple salt or sugar water. Neither you nor your study doctor will know which treatment you are receiving until the study is finished, ensuring the test is completely unbiased.
Taking part helps researchers understand whether MTX-474 could become a useful prescription medicine in the future. Even if the medicine does not directly help every person who joins, the findings provide vital clues for treating this challenging condition.
Key takeaways
- Testing a new medicine called MTX-474 for diffuse systemic sclerosis.
- Aimed at adults aged 18 and older with active skin thickening.
- Compares the active drug to an inactive dummy drip (placebo) over 24 weeks.
- Involves regular clinic visits, skin checks, blood tests, and skin biopsies.
- Participation is completely voluntary and you can withdraw at any time.
Who may be eligible?
This study is open to adults aged 18 and over who have been diagnosed with diffuse cutaneous systemic sclerosis. Both men and women can take part.
To join, participants must have active skin changes linked to the condition. Because the study measures whether the medicine softens thickened skin, doctors need to confirm that your skin changes can be measured accurately using standard clinical checks.
People with certain other severe medical conditions, active infections, or those taking specific medicines that could interfere with the study drug may not be able to join. The study team will review your full medical history and perform safety tests before you are accepted.
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 18 years of age or older.
- I have been diagnosed with diffuse cutaneous systemic sclerosis (dcSSc).
- I currently have thickened or tight skin on my arms, legs, or torso.
- I am able and willing to attend regular hospital appointments for infusions and tests.
- I am willing to undergo skin checks, blood tests, and minor skin biopsies.
What does participation involve?
If you take part, you will receive the study treatment through a drip into a vein (an intravenous infusion). You will be randomly assigned by a computer to receive either MTX-474 or a placebo.
You will need to attend regular hospital clinic visits over at least 24 weeks. During these visits, doctors will measure your skin thickness by gently pinching skin at different spots on your body. You will also have routine safety checks, including blood tests, urine tests, blood pressure measurements, and general physical examinations.
The study also involves taking small skin samples (biopsies) under local anaesthetic at around week 12, as well as several blood tests to see how quickly the medicine enters and leaves your body. The research team will monitor you closely throughout the trial to check on your wellbeing.
Potential risks and benefits
Locations (6)
- —UnverifiedNetherlands
- —UnverifiedRomania
- —UnverifiedPoland
- —UnverifiedFrance
- —UnverifiedItaly
- —UnverifiedSpain
Common questions
What is a placebo?
A placebo is a dummy treatment that looks just like the real medicine but contains no active drug. In this trial, it is a simple salt or sugar water drip used to compare results fairly.
Will I have to pay to take part?
No. Clinical trials in the UK do not charge you for the study medication or tests. Travel expenses to and from clinic visits are often reimbursed.
What is a skin biopsy?
A skin biopsy involves numbing a tiny patch of skin with an injection and taking a tiny sample for laboratory testing. It is a quick and routine procedure.
Can I carry on taking my usual medicines?
Some medicines are permitted, while others may interfere with the trial. The study doctor will review all your current prescriptions before you begin.
Can I leave the study if I change my mind?
Yes. You can leave the trial at any point, for any reason, and it will not affect the standard medical care you receive.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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