Active Lungs & Breathing NIHR-supported project Digestion, Kidneys & Other Organs

Platform Clinical Study for Conquering Scleroderma: A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Phase 2b Platform Clinical Study to Evaluate the Safety and Efficacy of Investigational Products in Participants with Interstitial Lung Dis...

Funder NIHR (part of DHSC)
Recipient organisation NIHR Guy's and St Thomas' Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Jan 2025 — Apr 2027

In plain English

AI plain-English summary

A clinical trial will test two experimental drugs against the lung scarring that kills most people with scleroderma, a rare autoimmune disease that hardens skin and internal organs. Around half of scleroderma patients develop interstitial lung disease (ILD), where lung tissue becomes inflamed and progressively scarred. This scarring is the leading cause of death in the condition, yet few treatments meaningfully improve how patients feel or function. The trial uses a "platform" design: roughly 430 participants globally will be randomly assigned to receive either a placebo or one of two investigational drugs—amlitelimab, a monoclonal antibody that blocks a protein involved in immune cell signalling, or BI 1015550, which inhibits a protein driving fibrosis. The study is double-blind and lasts about 60 weeks. If either drug proves safe and effective, it could become the first therapy to slow or stop lung fibrosis in scleroderma, directly extending survival and preserving quality of life for a patient group with few options. The platform design also allows future drugs to be added efficiently, accelerating the testing pipeline for this neglected disease.

View original technical description
Systemic sclerosis (or SSc) is a rare autoimmune disorder causing the tightening and hardening of the skin and internal organs. Interstitial lung disease (ILD) is characterised by inflammation and progressive fibrosis in the lung tissue. ILD is a common manifestation of SSc that tends to occur early in the disease process and is the leading cause of death in SSc.Currently, patients with SSc-ILD have a poor prognosis and there is a lack of meaningful effective therapies available that improve how the patient feels, functions and lives. Recent research has resulted in several IPs with potential use in SSc-ILD to meet this need.This is a Phase 2b, double blind, multicentre, randomised, placebo-controlled platform study to evaluate the safety and efficacy of investigational products (IP) in participants with SSc-ILD. This study will investigate multiple IPs in parallel with the capability to adapt over time. The IPs will be tested under the same general study design with participants randomly assigned to different “treatment regimens” for the different IPs. Currently, there are two treatment regimens available: • Amlitelimab – an investigational monoclonal antibody being developed for immune-mediated diseases. It works by binding to a protein involved in the function of certain cells in the immune system. By interrupting how these cells works, it is hoped that it will stop the process causing ILD in these participants.• BI 1015550 – an investigational drug being developed for the treatment of various inflammatory disorders. It works by inhibiting a protein involved in the growth of cells that cause fibrosis.Approximately 430 eligible participants will be enrolled globally. The total duration for participation is approximately 60 weeks (including screening and follow-up period).The study is sponsored by Scleroderma Research Foundation Inc.

Researchers

Philip Molyneaux (Principal Investigator)

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