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Cladribine Low Dose

Phase 3

Generalized Myasthenia Gravis | Small molecule | Neurology |Merck & Company, Inc.|Last Updated: May 22, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment264
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06463587Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)PHASE3 RECRUITING 264Jun 25, 2024Nov 12, 2030May 22, 2026138 United States, Argentina +21
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Study Endpoints
Primary Endpoints
Change from Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
Baseline, Week 24
Secondary Endpoints
Change from Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
Baseline, Week 24
Percentage of MG-ADL Responders at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
At Week 24
Change from Baseline in Myasthenia Gravis Composite (MGC) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
Baseline, Week 24
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PlaceboPLACEBO_COMPARATORDBPC 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: If clinically justified, participants requiring retreatment with cladribine Low Dose or High Dose or cladribine supplemental dose will receive the selected dose of cladribine or matching placebo regimen.
Cladribine Low DoseEXPERIMENTALDBPC 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: If clinically justified, participants requiring retreatment with cladribine Low Dose regimen and/or supplemental dose will receive the selected dose of cladribine or matching placebo regimen.
Cladribine High DoseEXPERIMENTALDBPC 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: If clinically justified, participants requiring retreatment with cladribine High Dose regimen and/or supplemental dose will receive the selected dose of cladribine or matching placebo regimen.
Interventions
NameTypeDescription
PlaceboOTHERParticipants will receive placebo matched to cladribine in two courses separated by 4 weeks.
Cladribine Low DoseDRUGParticipants will receive cladribine low dose in two courses separated by 4 weeks.
Cladribine High DoseDRUGParticipants will receive cladribine high dose in two courses separated by 4 weeks.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites138

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 A...

Countries:United StatesArgentinaAustraliaBelgiumBulgariaChinaFranceGeorgiaGermanyGreeceHungaryIndiaItalyJapanPolandRomaniaSouth KoreaSpainSwedenSwitzerlandTaiwanTurkey (Türkiye)United Kingdom
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