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

Conditions

• Generalized Myasthenia Gravis

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Placebo — OTHER
  Participants will receive placebo matched to cladribine in two courses separated by 4 weeks.
• Cladribine Low Dose — DRUG
  Participants will receive cladribine low dose in two courses separated by 4 weeks.
• Cladribine High Dose — DRUG
  Participants will receive cladribine high dose in two courses separated by 4 weeks.

Study Details

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. Furthermore, in trial interviews will be conducted as a sub-study to MyClad with a sub-set of participants to gain an in depth understanding of the participant cladribine treatment and study experience.

Key Dates

Start date

Jun 25, 2024

Status verified

May 2026

Primary completion

Aug 23, 2028

Completion

Nov 12, 2030

Study Design

Enrollment

264 participants (estimated)

Allocation

RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• 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: 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.
• 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: 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.
• 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: 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.

Primary Outcome Measure

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 [ Time Frame: Baseline, Week 24 ]

Central Contacts

• US Medical Information
  888-275-7376
  [email protected]
• Communication Center
  +49 6151 72 5200
  [email protected]

Locations (21)

Find similar trials in Phoenix, AZ

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