A phase 2 study with MT-7117 (dersimelagon) in patients with dcSSc
Research type
Research Study
Full title
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects with Diffuse Cutaneous Systemic Sclerosis
IRAS ID
282445
Contact name
Christopher Denton
Contact email
Sponsor organisation
Mitsubishi Tanabe Pharma Development America, Inc. (MTDA)
Eudract number
2020-000134-17
Duration of Study in the UK
1 years, 8 months, 9 days
Research summary
Summary of Research
Diffuse Cutaneous Systemic Sclerosis (referred to as “dcSSc”) is a sub-type of systemic sclerosis which is a complex genetic, autoimmune and chronic medical condition caracterised by excessive production of collagen (fibrosis), microvascular damage and fibrosis in the skin, joints, lungs, oesophagus, gastrointestianl tract, kidneys, heart and other internal organs.
The study drug MT-7117 (dersimelagon) is an experimental therapy being developed for the treatment of dsCCs by reducing the inflamatory processes. The purpose of this study is to evaluate the efficacy, the safety and tolerability of this study drug in participants with dcSSc.
The study is divided into three parts: screening, double blind treatment period and follow up period. During the study, patients will have 9 sites visits during a 60 weeks period. This phase 2 study is double blind, meaning that the participants will receive either the study drug, MT-7117 or the placebo, and neither the participants nor the study staff will know which one the participant will receive. The study is also randomised, meaning that the participants will be allocated by change to receive the study drug or the inactive form of the study drug, called placebo. In this research study, 1 out of every 2 participants will receive active medicine (50% chance) and 1 out of 2 will receive placebo (50% chance).
Approximately 72 participants worldwide will take part in this study.Summary of Results
The primary efficacy endpoint of ACR CRISS composite score (0-1) did not demonstrate a statistically significant difference between the placebo and MT-7117 groups after 52 weeks of treatment. There were no statistical changes in the mRSS, a measure of skin thickness, and HAD-QI.
However, the secondary endpoint of %pFVC demonstrated greater numerical improvement from baseline in the MT-7117 group compared to the placebo group, which indicates that MT-7117 may have the potential to improve pulmonary function in subjects with dcSSc. Importantly, compared to the placebo group, the subset of subjects who completed 52 weeks of MT-7117 300 mg demonstrated a nominally statistically significant difference at Week 52 in change in %pFVC from baseline, while the subjects whose dose was reduced to 200 mg due to AEs did not.
These findings may indicate that administration of 300 mg for 52 weeks may benefit pulmonary dysfunction in SSc. Although the use of MT-7117 was well tolerated and has a safety profile that supports use in patients with dcSSc, skin hyperpigmentation was the most frequently reported TEAE that led to dose reduction or discontinuation.
Has the registry been updated to include summary results?: Yes
If yes - please enter the URL to summary results: https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Fclick.pstmrk.it%2F3ts%2Fwww.clinicaltrialsregister.eu%252Fctr-search%252Ftrial%252F2020-000134-17%252Fresults%2FNBTI%2FbIW6AQ%2FAQ%2F95035186-9155-45b4-a526-6cc1debe2d59%2F1%2FYu1p4QFYUb&data=05%7C02%7Clondoncentral.rec%40hra.nhs.uk%7C210178f7f67b415f159008dd2e63db3c%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638717728587639019%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=zWJwHRU%2BdbZMorY1hvFHSy4cxPv9z%2BPEUnEU1NYl%2BtU%3D&reserved=0REC name
London - Central Research Ethics Committee
REC reference
20/LO/1077
Date of REC Opinion
23 Dec 2020
REC opinion
Further Information Favourable Opinion