An Open-Label Study to Assess the Safety & Efficacy of Leniolisib in Japanese Patients With APDS

PHASE3RecruitingINTERVENTIONAL
Enrollment

3

Participants

Timeline

Start Date

August 3, 2023

Primary Completion Date

January 31, 2025

Study Completion Date

March 31, 2025

Conditions
APDS Gene Mutation
Interventions
DRUG

Leniolisib

The doses selected range from 40 to 70 mg BID (based on body weight, resulting in total daily doses ranging from 80 to 140 mg a day for 12 weeks in Part I and 1 year in Part II, or until marketing approval in Japan, whichever is longer.

Trial Locations (2)

113-8510

RECRUITING

Tokyo Medical And Dental University Hospital, Tokyo

734-8551

RECRUITING

Hiroshima University Hospital, Hiroshima

Sponsors
All Listed Sponsors
collaborator

Laboratory Corporation of America

INDUSTRY

collaborator

Axial Biotech, Inc

INDUSTRY

collaborator

CMIC Co, Ltd. Japan

INDUSTRY

lead

Pharming Technologies B.V.

INDUSTRY

NCT06249997 - An Open-Label Study to Assess the Safety & Efficacy of Leniolisib in Japanese Patients With APDS | Biotech Hunter | Biotech Hunter