MS700568_0183 - MyClad
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, 3-Arm, 3-Period Study to Assess the Efficacy and Safety of a New Formulation of Oral Cladribine Compared with Placebo in Participants with Generalized Myasthenia Gravis
IRAS ID
1009423
Contact name
Communication Centre Merck KGaA Communication Centre Merck KGaA
Contact email
Sponsor organisation
Merck Healthcare KGaA
Eudract number
2023-507746-83
Research summary
This study assessing a new formulation of oral Cladribine (the study drug) compared with placebo (an inactive drug) in participants with Generalized Myasthenia Gravis (gMG). gMG is a long-lasting condition where the immune system mistakenly attacks and affects the muscles, causing muscle weakness and tiredness or exhaustion.
This study aims to find out:
• How well and for how long the study drug controls the disease compared to placebo.
• How well participants tolerate the study drug.
• What the body does to the study drug to break it down and remove it (called pharmacokinetics [PK]).
• How substances in the body (called biomarkers) are changed by the study drug, and how they relate to gMG.The study is divided into 3 periods:
• Double-Blind Placebo-Controlled (DBPC) Period (Period 1): Participants will be randomised 1:1:1 to receive either cladribine high dose, cladribine low dose, or placebo.
• Blinded Extension (BE) Period (Period 2): Participants initially randomised to placebo will be re-randomised to receive cladribine (high or low dose); participants who received cladribine in the DBPC period will be allocated to receive placebo (to maintain patients’ and investigators’ blinding) and may be eligible to receive a supplemental dose of cladribine, based on clinical need.
• Retreatment (RT) Period (Period 3): Participants may be retreated with cladribine (supplemental or full dose), if clinically justified.Visit frequency:
• Period 1 and 2: Site visits every 4 weeks, with 1 video/phone call in between.
• Period 3: Site visit every 12 weeks with 2 monthly video/phone calls in between.240 participants will take part in the study for approximately 144 weeks.
REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
24/EM/0037
Date of REC Opinion
30 Apr 2024
REC opinion
Further Information Favourable Opinion