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)

Status: Recruiting
Location: See all (35) locations...
Intervention Type: Drug, Other
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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 \>= 50 percentage (%) of the total score due to non-ocular symptoms. Screening and Baseline MG-ADL scores must be stable. The difference between the Screening and Baseline scores should not be more than 2 and there should be no reported MG exacerbation during the Screening period

• If treated with oral corticosteroids: should be on a stable daily dose for at least 3 months prior to and during screening. 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 (pyridostigmine dose ≤ 480 mg/day) for at least 3 months prior to and during screening

• Have a body weight \>= 40 kilograms

• Other protocol defined inclusion criteria could apply

Locations
United States
Florida
Neurology of Central Florida Research Center, LLC
RECRUITING
Altamonte Springs
SFM Clinical Research, LLC
RECRUITING
Boca Raton
North Carolina
Atrium Health Wake Forest Baptist
RECRUITING
Winston-salem
South Carolina
Clinical Trials of South Carolina - Charleston
RECRUITING
Charleston
Vermont
The University of Vermont Medical Center
RECRUITING
Burlington
Other Locations
Argentina
Expertia S.A- Mautalen Salud e Investigación
RECRUITING
Ciudad Autonoma Buenos Aires
Instituto de Investigaciones Metabolicas (IDIM)
RECRUITING
Ciudad Autonoma Buenos Aires
Instituto de Investigaciones Neurologicas Raul Carrea, FLENI - de Investigaciones Clinicas del área de Cardiología y enfermedades metabólicas asociadas
RECRUITING
Ciudad Autonoma Buenos Aires
Hospital Cordoba
RECRUITING
Córdoba
Fundacion Rosarina de Neurorehabilitacion
RECRUITING
Rosario
INECO Neurociencias Oroño
RECRUITING
Rosario
CER San Juan Centro Polivalente de Asistencia e Inv. Clinica
RECRUITING
San Juan
Centro de Investigaciones Medicas Tucuman
RECRUITING
San Miguel De Tucumán
Australia
Gold Coast University Hospital - PARENT
RECRUITING
Southport
Georgia
Aversi Clinic Ltd
RECRUITING
Tbilisi
Ltd. Pineo Medical Ecosystem
RECRUITING
Tbilisi
New Hospitals LLC
RECRUITING
Tbilisi
Japan
Hakodate Municipal Hospital - Dept of Neurology
RECRUITING
Hakodate-shi
General Hanamaki Hospital - Dept of Neurology
RECRUITING
Hanamaki-shi
Kagawa University Hospital - Dept of Neurology
RECRUITING
Kita-gun
IUHW Narita Hospital - Dept of Neurology
RECRUITING
Narita-shi
Tokyo Medical University Hospital - Dept of Neurology
RECRUITING
Shinjuku-ku
Osaka University Hospital - Dept of Neurology/Stroke Care Unit
RECRUITING
Suita-shi
Poland
Wielospecjalistyczne Centrum Medyczne Ibismed - Dept of Neurology
RECRUITING
Zabrze
Republic of Korea
Kyungpook National University Chilgok Hospital
RECRUITING
Daegu
Asan Medical Center
RECRUITING
Seoul
Keimyung University Dongsan Hospital
RECRUITING
Seoul
Korea University Anam Hospital
RECRUITING
Seoul
Samsung Medical Center
RECRUITING
Seoul
Seoul Metropolitan Government Seoul National University Boramae Medical Center
RECRUITING
Seoul
Taiwan
Kaohsiung Chang Gung Memorial Hospital
RECRUITING
Kaohsiung City
China Medical University Hospital
RECRUITING
Taichung
Shin Kong Wu Ho-Su Memorial Hospital
RECRUITING
Taipei
Taipei Veterans General Hospital
RECRUITING
Taipei
United Kingdom
Royal Hallamshire Hospital - Dept of Neurology
RECRUITING
Sheffield
Contact Information
Primary
US Medical Information
eMediUSA@emdserono.com
888-275-7376
Backup
Communication Center
service@emdgroup.com
+49 6151 72 5200
Time Frame
Start Date: 2024-06-25
Estimated Completion Date: 2030-08-19
Participants
Target number of participants: 264
Treatments
Placebo_comparator: Placebo
DBPC Period: Participants will be administered with Placebo, orally as 2 separate treatment courses starting on Day 1 and at the beginning of Week 5.~BE Period: Participants initially randomized to placebo matched to cladribine in DBPC period will receive cladribine Low Dose or High Dose, orally as 2 separate treatment courses starting at the beginning of Week 25 and at the beginning of Week 29 and retreated if clinically justified with placebo matched to cladribine.~RT Period: Participants requiring retreatment with cladribine Low Dose or High Dose or retreated with cladribine supplemental dose if clinically justified.
Experimental: Cladribine Low Dose
DBPC Period: Participants will be administered with cladribine Low Dose, orally as 2 separate treatment courses starting on Day 1 and at the beginning of Week 5.~BE Period: Participants initially randomized to cladribine Low Dose in DBPC period will receive placebo matched to cladribine as 2 separate treatment courses starting at the beginning of Week 25 and at the beginning of Week 29 and retreated with cladribine supplemental dose if clinically justified.~RT Period: Participants requiring retreatment with cladribine Low Dose regimen and/or supplemental dose will receive the selected dose of cladribine if clinically justified.
Experimental: Cladribine High Dose
DBPC Period: Participants will be administered cladribine High Dose, orally as 2 separate treatment courses starting on Day 1 and at the beginning of Week 5.~BE Period: Participants initially randomized to cladribine High Dose in DBPC period will receive placebo matched to cladribine as 2 separate treatment courses starting at the beginning of Week 25 and at the beginning of Week 29 and retreated with cladribine supplemental dose if clinically justified.~RT Period: Participants requiring retreatment with cladribine High Dose regimen and/or supplemental dose will receive the selected dose of cladribine if clinically justified.
Related Therapeutic Areas
Sponsors
Collaborators: EMD Serono Research & Development Institute, Inc.
Leads: Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany

This content was sourced from clinicaltrials.gov

Similar Clinical Trials