MedTrace Logo

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

NCT06249997 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2024-02-08

No results posted yet for this study

Summary

An Open-Label, Non-Randomized Study to Assess the Safety and Efficacy of Leniolisib in Japanese Patients With Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) Followed By an Open-Label Long-Term Extension.

For the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).

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.

Sponsors & Collaborators

  • Laboratory Corporation of America

    collaborator INDUSTRY

  • Axial Biotech, Inc

    collaborator INDUSTRY

  • CMIC Co, Ltd. Japan

    collaborator INDUSTRY

  • Pharming Technologies B.V.

    lead INDUSTRY

Principal Investigators

  • Hirokazu Kanegane, Prof. · Tokyo Medical And Dental University Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-08-03
Primary Completion
2025-01-31
Completion
2025-03-31

Countries

  • Japan

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06249997 on ClinicalTrials.gov