Phase 3 Study Comparing New Oral Cladribine Formulation to Placebo in Adults With Generalized Myasthenia Gravis

What this Trial Is About

Researchers are evaluating the effectiveness and safety of a new oral cladribine formulation in adults with Generalized Myasthenia Gravis gMG. This phase 3 clinical trial compares cladribine to a placebo to understand its sustained benefits, the need for retreatment, and long-term safety. The study also includes a component to analyze how the drug is processed in the body and features interviews to learn about participants experiences with cladribine treatment. The trial is organized into three periods a double-blind placebo-controlled DBPC period where participants receive either placebo or cladribine in two separate oral courses a blinded extension BE period where initial placebo recipients switch to cladribine doses and some receive retreatment if needed and a retreatment RT period where participants may receive additional cladribine or placebo based on clinical need. Doses include low and high oral cladribine, administered as scheduled treatment courses. Participants will undergo various assessments including evaluations of muscle weakness and daily living activities using specific scales like the MG-ADL and QMG. Researchers will monitor safety through tracking adverse events, laboratory tests, and heart monitoring. Pharmacokinetic blood sampling will measure cladribine levels at multiple time points. The study duration extends up to week 144, during which participants responses and safety are closely observed to understand the drugs effects over time.

Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)