Ignite Creation Date:
2024-10-26 @ 3:37 PM
Last Modification Date:
2024-10-26 @ 3:37 PM
Study NCT ID:
NCT06549114
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-07-17
Brief Title:
Leniolisib for Immune Dysregulation in PIDs
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Study to Assess Safety and Tolerability and Explore Efficacy of Leniolisib for Immune Dysregulation in Primary Immunodeficiency Disorders
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study is an exploratory non-randomized open-label within-patient dose escalation study The primary objective is to assess safety and tolerability of leniolisib Secondary objectives include assessments of PKPD and to explore clinical efficacy measures with administration of three different dose levels of leniolisib
Detailed Description:
Patients ages 12-75 diagnosed with genetically defined PID disorders linked to PI3K signaling This includes disorders caused by pathogenic variants in SOCS1 PTEN CTLA4 NFKB1 variants leading to NFKB pathway activation FAS germline or somatic or RAS-associated leukoproliferative disorder caused by somatic variants in NRAS or KRAS not juvenile myelomonocytic leukemia JMML All subjects participating will receive leniolisib film-coated tablets FCTs with a planned dose regimen consisting of a starting dose of 10 mg twice daily BID for 4 weeks followed by 30 mg BID for 4 weeks and then 70 mg BID for 12 weeks Leniolisib dose increase at the individual subject level will occur if no safety or tolerability issues have been identified by the Investigator that precludes the planned dose escalation If subjects experience dose-limiting toxicities or intolerance the dose may be temporarily discontinued for up to 2 weeks andor the dose may be de-escalated and continued If treatment is temporarily discontinued the previous dose or a de-escalated dose may be restarted based on clinician preference
Subjects not continuing leniolisib treatment outside of the current protocol will be followed up with the EOS visit planned to occur approximately 28 days after last dose of leniolisib
Subjects not continuing leniolisib treatment outside of the current protocol will be followed up with the EOS visit planned to occur approximately 28 days after last dose of leniolisib
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None