Open Label Study in Adolescents and Children With Myotonic Disorders

PHASE3CompletedINTERVENTIONAL
Enrollment

12

Participants

Timeline

Start Date

September 3, 2021

Primary Completion Date

June 14, 2024

Study Completion Date

August 8, 2024

Conditions
Myotonic Dystrophy
Interventions
DRUG

Mexiletine

"Patients will be enrolled sequentially into 2 cohorts. Cohort 1 - (patients aged 12 to \< 18 years): approximately 8 weeks - 4 weeks of dose titration period + 4 weeks of maintenance period.~Cohort 2 - (patients aged 6 to \< 12 years,): approximately 8 weeks - 4 weeks of dose titration period + 4 weeks of maintenance period.~Enrolment for Cohort 2 will begin after initial safety assessment of patients in Cohort 1 by the DSMB and no safety concerns are observed. The dose level for cohort 2 will be confirmed by PK modelling study."

Trial Locations (1)

Unknown

Hôpital Necker-Enfants-Malades, Paris

All Listed Sponsors
lead

Lupin Ltd.

INDUSTRY

NCT04624750 - Open Label Study in Adolescents and Children With Myotonic Disorders | Biotech Hunter | Biotech Hunter