Phase 3 Trial Launched for Oral Cladribine in Generalized Myasthenia Gravis

• Merck KGaA has initiated a Phase 3 clinical trial, MyClad, to evaluate the safety and efficacy of oral cladribine capsules for treating generalized myasthenia gravis (gMG).
• The global, randomized, double-blind, placebo-controlled study will involve 240 participants and aims to assess cladribine's impact on this neurodegenerative condition.
• Cladribine selectively targets B and T lymphocytes, which are believed to be the primary cause of gMG, offering a potential at-home treatment option to reduce patient burden.
• The trial's progress could provide a new therapeutic option for gMG, addressing the challenges of nerve-muscle communication and improving patient outcomes.

Merck KGaA has announced the commencement of a Phase 3 clinical trial to investigate oral cladribine as a treatment for generalized myasthenia gravis (gMG). The MyClad study is a global, randomized, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of cladribine capsules in 240 individuals diagnosed with gMG.

gMG is a neurodegenerative condition characterized by impaired communication between nerves and muscles, leading to difficulties in moving the eyes, throat, and limbs. The current treatment landscape involves various therapies, but there remains a need for more convenient and effective options.

Cladribine's Mechanism of Action

Cladribine functions by selectively targeting B and T lymphocytes, which are implicated in the pathogenesis of gMG. By modulating these immune cells, cladribine aims to slow the progression of the disease and alleviate symptoms. Oral administration of cladribine offers the potential for at-home treatment, reducing the burden on patients and healthcare systems.

MyClad Study Design

The MyClad study will enroll 240 participants globally, randomizing them to receive either oral cladribine or a placebo. The trial's primary endpoint is to assess the safety and efficacy of cladribine in improving muscle strength and reducing fatigue associated with gMG. Secondary endpoints include evaluating the impact of cladribine on various aspects of daily living and quality of life.

Expert Commentary

"Given our extensive experience in addressing patients’ needs in immune-driven neurological conditions, we believe that cladribine capsules represent a highly differentiated potential therapeutic option for gMG," said Jan Klatt, head of development unit Neurology & Immunology for the Healthcare business of Merck KGaA.

The results of the MyClad study are anticipated to provide valuable insights into the potential of oral cladribine as a novel therapeutic option for individuals with gMG. The trial's outcomes could significantly impact the treatment paradigm for this debilitating condition, offering a more convenient and targeted approach to managing the disease.