RECRUITING

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

Conditions

Generalized Myasthenia Gravis Anti-AChR antibody positive anti-MuSK antibody positive anti-LRP4 antibody positive seronegative gMG

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The purpose of this clinical study is to determine the efficacy and safety of a new oral cladribine formulation in participants with Generalized Myasthenia Gravis (gMG) in comparison to placebo. It will also investigate the sustained efficacy, the need for retreatment, and the long-term safety of oral cladribine in gMG. An additional component is included to characterize the Pharmacokinetics (PK) of the new cladribine formulation in gMG participants. This study is divided into 3 periods: the double-blind placebo control (DBPC) pivotal period, and 2 extensions, the blinded extension (BE) and the retreatment (RT) period.

Official 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(MyClad)

Quick Facts

Study Start:2024-06-25

Study Completion:2030-07-23

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06463587

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Adults of ≥ 18 years of age at the time of signing the informed consent. * Diagnosis of Myasthenia Gravis with generalized muscle weakness, meeting clinical criteria for Myasthenia Gravis Foundation of America Class II to IVa classification. * In participants positive for Acetylcholine receptor antibody (anti-AChR) or muscle-specific kinase antibody(anti-MuSK) * In participants that are autoantibody seronegative and participants who are positive for anti-low-density lipoprotein receptor-related protein 4 antibodies (anti-LRP4) * Has a Screening and Baseline MG-ADL score more than or equal to (\>=) 6 with at least 50 percentage (%) of the total score due to non-ocular symptoms * If treated with oral corticosteroids: should be on a stable daily dose for at least 4 weeks before randomization. In such case, the daily dose of oral steroids should not exceed 20 milligrams(mg)/day for prednisone/ prednisolone or 16 mg/day for methylprednisolone * If treated with acetylcholinesterase inhibitor should be on a stable daily dose for at least 4 weeks before randomization * Have a body weight \>= 40 kilograms * Other protocol defined inclusion criteria could apply	* Immunologic disorder other than MG or any other condition requiring chronic oral, intravenous, intramuscular, or intraarticular corticosteroid therapy. Well-controlled thyroid disease, as per the Treating Investigator or the participants regular treating physician recorded in the source documents, is not exclusionary * Molecularly characterized or suspected congenital myasthenic syndrome, Lambert-Eaton myasthenic syndrome, inherited myopathy, muscular dystrophy, acquired myopathy or any other neurologic or systematic disease that mimics MG muscular weakness * Active, clinically significant viral, bacterial, or fungal infection, including brain MRI findings consistent with signs of infection such as PML, or any major episode of infection requiring hospitalization or treatment with parenteral anti-infectives within 4 weeks before or during Screening, or completion of oral antiinfectives within 2 weeks before or during Screening, or a history of recurrent infections (i.e. 3 or more of the same type of infection in a 12-month rolling period). Vaginal candidiasis, onychomycosis, and genital or oral herpes simplex virus considered by the Investigator to be sufficiently controlled would not be exclusionary. * Has a history of or current diagnosis of active tuberculosis (TB) * Active malignancy, or history of cancer * Treatment with nonsteroidal immunosuppressants, used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus within 4 weeks prior to randomization * Treatment with eculizumab, rozanolixizumab efgartigimod, ravulizumab, or zilucoplan within 8 weeks prior to randomization * History of thymectomy within 6 months prior to Screening. * History of generalized seizures (except for history of infantile febrile seizures). * Negative for Varicella Zoster Virus antibodies at screening. * Other protocol defined exclusion criteria could apply

Inclusion Criteria

Exclusion Criteria

* Adults of ≥ 18 years of age at the time of signing the informed consent.
* Diagnosis of Myasthenia Gravis with generalized muscle weakness, meeting clinical criteria for Myasthenia Gravis Foundation of America Class II to IVa classification.
* In participants positive for Acetylcholine receptor antibody (anti-AChR) or muscle-specific kinase antibody(anti-MuSK)
* In participants that are autoantibody seronegative and participants who are positive for anti-low-density lipoprotein receptor-related protein 4 antibodies (anti-LRP4)
* Has a Screening and Baseline MG-ADL score more than or equal to (\>=) 6 with at least 50 percentage (%) of the total score due to non-ocular symptoms
* If treated with oral corticosteroids: should be on a stable daily dose for at least 4 weeks before randomization. In such case, the daily dose of oral steroids should not exceed 20 milligrams(mg)/day for prednisone/ prednisolone or 16 mg/day for methylprednisolone
* If treated with acetylcholinesterase inhibitor should be on a stable daily dose for at least 4 weeks before randomization
* Have a body weight \>= 40 kilograms
* Other protocol defined inclusion criteria could apply

* Immunologic disorder other than MG or any other condition requiring chronic oral, intravenous, intramuscular, or intraarticular corticosteroid therapy. Well-controlled thyroid disease, as per the Treating Investigator or the participants regular treating physician recorded in the source documents, is not exclusionary
* Molecularly characterized or suspected congenital myasthenic syndrome, Lambert-Eaton myasthenic syndrome, inherited myopathy, muscular dystrophy, acquired myopathy or any other neurologic or systematic disease that mimics MG muscular weakness
* Active, clinically significant viral, bacterial, or fungal infection, including brain MRI findings consistent with signs of infection such as PML, or any major episode of infection requiring hospitalization or treatment with parenteral anti-infectives within 4 weeks before or during Screening, or completion of oral antiinfectives within 2 weeks before or during Screening, or a history of recurrent infections (i.e. 3 or more of the same type of infection in a 12-month rolling period). Vaginal candidiasis, onychomycosis, and genital or oral herpes simplex virus considered by the Investigator to be sufficiently controlled would not be exclusionary.
* Has a history of or current diagnosis of active tuberculosis (TB)
* Active malignancy, or history of cancer
* Treatment with nonsteroidal immunosuppressants, used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus within 4 weeks prior to randomization
* Treatment with eculizumab, rozanolixizumab efgartigimod, ravulizumab, or zilucoplan within 8 weeks prior to randomization
* History of thymectomy within 6 months prior to Screening.
* History of generalized seizures (except for history of infantile febrile seizures).
* Negative for Varicella Zoster Virus antibodies at screening.
* Other protocol defined exclusion criteria could apply

Contacts and Locations

Study Contact

US Medical Information

CONTACT

888-275-7376

eMediUSA@emdserono.com

Communication Center

CONTACT

+49 6151 72 5200

service@emdgroup.com

Principal Investigator

Medical Responsible

STUDY_DIRECTOR

EMD Serono Research & Development Institute, Inc.

Study Locations (Sites)

Neurology of Central Florida Research Center, LLC

Altamonte Springs, Florida, 32714

United States

SFM Clinical Research, LLC

Boca Raton, Florida, 33487

United States

Atrium Health Wake Forest Baptist

Winston-Salem, North Carolina, 27157-1078

United States

Clinical Trials of South Carolina - Charleston

Charleston, South Carolina, 29406

United States

Collaborators and Investigators

Sponsor: Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany

Medical Responsible, STUDY_DIRECTOR, EMD Serono Research & Development Institute, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-06-25

Study Completion Date2030-07-23

Study Record Updates

Study Start Date2024-06-25

Study Completion Date2030-07-23

Terms related to this study

Keywords Provided by Researchers

Anti-AChR antibody positive
anti-MuSK antibody positive
anti-LRP4 antibody positive
seronegative gMG

Additional Relevant MeSH Terms

Generalized Myasthenia Gravis