Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation

Conditions

• PIDs Linked to PI3K

Eligibility Criteria

Sex

ALL

Age

12 Years - 75 Years

Healthy Volunteers

Not accepted

Interventions

• Leniolisib — DRUG
  All subjects will receive leniolisib film-coated tablets (FCTs) at the same dose they were receiving when they completed the preceding study (10, 30, or 70 mg twice daily \[BID\]).

Study Details

This is an open-label extension (OLE) study to extend treatment to patients with primary immunodeficiency (PID) disorders linked to phosphoinositide 3-kinase delta signaling who participated in a prior study of leniolisib, LE 7201. The primary objective is to assess long-term safety and tolerability of leniolisib. Secondary and exploratory objectives include various efficacy and immunophenotyping measures for leniolisib.

Key Dates

Start date

Apr 29, 2025

Status verified

May 2025

Primary completion

Sep 18, 2028

Completion

Apr 13, 2029

Study Design

Enrollment

12 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Treatment Arm
  All subjects will receive leniolisib film-coated tablets (FCTs)

Primary Outcome Measure

To assess the long-term safety and tolerability of leniolisib [ Time Frame: From Baseline to approximately 3 years of Treatment ]

Locations (3)

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