Trial Title:
Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)
NCT ID:
NCT06463587
Condition:
Generalized Myasthenia Gravis
Conditions: Official terms:
Myasthenia Gravis
Muscle Weakness
Cladribine
Conditions: Keywords:
Anti-AChR antibody positive
anti-MuSK antibody positive
anti-LRP4 antibody positive
seronegative gMG
Study type:
Interventional
Study phase:
Phase 3
Overall status:
Recruiting
Study design:
Allocation:
Randomized
Intervention model:
Sequential Assignment
Primary purpose:
Treatment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Intervention:
Intervention type:
Other
Intervention name:
Placebo
Description:
Participants will receive placebo matched to cladribine in two courses separated by 4
weeks.
Arm group label:
Placebo
Intervention type:
Drug
Intervention name:
Cladribine Low Dose
Description:
Participants will receive cladribine low dose in two courses separated by 4 weeks.
Arm group label:
Cladribine Low Dose
Arm group label:
Placebo
Intervention type:
Drug
Intervention name:
Cladribine High Dose
Description:
Participants will receive a total of cladribine high dose in two courses separated by 4
weeks.
Arm group label:
Cladribine High Dose
Arm group label:
Placebo
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.
Criteria for eligibility:
Criteria:
Inclusion 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
Exclusion Criteria:
- 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
Gender:
All
Minimum age:
18 Years
Maximum age:
N/A
Healthy volunteers:
No
Locations:
Facility:
Name:
SFM Clinical Research, LLC
Address:
City:
Boca Raton
Zip:
33487
Country:
United States
Status:
Recruiting
Investigator:
Last name:
Marc H Feinberg
Email:
Principal Investigator
Facility:
Name:
Fundacion Rosarina de Neurorehabilitacion
Address:
City:
Rosario
Zip:
S2000BZL
Country:
Argentina
Status:
Recruiting
Investigator:
Last name:
Carolina Mainella
Email:
Principal Investigator
Facility:
Name:
INECO Neurociencias Oroño
Address:
City:
Rosario
Zip:
S2000DTP
Country:
Argentina
Status:
Recruiting
Investigator:
Last name:
Dario Tavolini
Email:
Principal Investigator
Facility:
Name:
Centro de Investigaciones Medicas Tucuman
Address:
City:
San Miguel de Tucuman
Zip:
T4000AXL
Country:
Argentina
Status:
Recruiting
Investigator:
Last name:
Jorge Gustavo Jose
Email:
Principal Investigator
Facility:
Name:
Hospital Cordoba
Address:
City:
Cordoba
Zip:
X5000EDC
Country:
Argentina
Status:
Recruiting
Investigator:
Last name:
Susana del Valle Liwacki
Email:
Principal Investigator
Facility:
Name:
Ltd. Pineo Medical Ecosystem
Address:
City:
Tbilisi
Zip:
0114
Country:
Georgia
Status:
Recruiting
Investigator:
Last name:
Alexander Tsiskaridze
Email:
Principal Investigator
Facility:
Name:
New Hospitals LLC
Address:
City:
Tbilisi
Zip:
0114
Country:
Georgia
Status:
Recruiting
Investigator:
Last name:
Temur Margania
Email:
Principal Investigator
Facility:
Name:
Aversi Clinic Ltd
Address:
City:
Tbilisi
Zip:
0160
Country:
Georgia
Status:
Recruiting
Investigator:
Last name:
Gvantsa Giorgadze
Email:
Principal Investigator
Facility:
Name:
IUHW Narita Hospital - Dept of Neurology
Address:
City:
Narita-shi
Zip:
286-8520
Country:
Japan
Status:
Recruiting
Investigator:
Last name:
Hiroyuki Murai
Email:
Principal Investigator
Facility:
Name:
Hakodate Municipal Hospital - Dept of Neurology
Address:
City:
Hakodate-shi
Zip:
392006
Country:
Japan
Status:
Recruiting
Investigator:
Last name:
Kazuhiro Horiuchi
Email:
Principal Investigator
Facility:
Name:
General Hanamaki Hospital - Dept of Neurology
Address:
City:
Hanamaki-shi
Zip:
025-0082
Country:
Japan
Status:
Recruiting
Investigator:
Last name:
Kimiaki Utsugisawa
Email:
Principal Investigator
Facility:
Name:
Kagawa University Hospital - Dept of Neurology
Address:
City:
Kita-gun
Zip:
7610793
Country:
Japan
Status:
Recruiting
Investigator:
Last name:
Kazushi Deguchi
Email:
Principal Investigator
Facility:
Name:
Tokyo Medical University Hospital - Dept of Neurology
Address:
City:
Shinjuku-ku
Zip:
160-0023
Country:
Japan
Status:
Recruiting
Investigator:
Last name:
Masayuki Masuda
Email:
Principal Investigator
Facility:
Name:
Kyungpook National University Chilgok Hospital
Address:
City:
Daegu
Zip:
41404
Country:
Korea, Republic of
Status:
Recruiting
Investigator:
Last name:
Minsung Kang
Email:
Principal Investigator
Facility:
Name:
Keimyung University Dongsan Hospital
Address:
City:
Seoul
Zip:
02841
Country:
Korea, Republic of
Status:
Recruiting
Investigator:
Last name:
Hung Youl Seok
Email:
Principal Investigator
Facility:
Name:
Samsung Medical Center
Address:
City:
Seoul
Zip:
06351
Country:
Korea, Republic of
Status:
Recruiting
Investigator:
Last name:
Byoung Joon Kim
Email:
Principal Investigator
Facility:
Name:
Korea University Anam Hospital
Address:
City:
Seoul
Zip:
136-705
Country:
Korea, Republic of
Status:
Recruiting
Investigator:
Last name:
Byung-Jo Kim
Email:
Principal Investigator
Facility:
Name:
Asan Medical Center
Address:
City:
Seoul
Zip:
138-736
Country:
Korea, Republic of
Status:
Recruiting
Investigator:
Last name:
Young-Min Lim
Email:
Principal Investigator
Facility:
Name:
Seoul Metropolitan Government Seoul National University Boramae Medical Center
Address:
City:
Seoul
Zip:
156707
Country:
Korea, Republic of
Status:
Recruiting
Investigator:
Last name:
Yoon-Ho Hong
Email:
Principal Investigator
Facility:
Name:
China Medical University Hospital
Address:
City:
Taichung
Zip:
404327
Country:
Taiwan
Status:
Recruiting
Investigator:
Last name:
Yuh-Cherng Guo
Email:
Principal Investigator
Start date:
June 25, 2024
Completion date:
July 23, 2030
Lead sponsor:
Agency:
Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Agency class:
Industry
Collaborator:
Agency:
EMD Serono Research & Development Institute, Inc.
Agency class:
Industry
Source:
Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06463587
https://clinicaltrials.emdgroup.com/en
