Leniolisib for Activated PI3K Delta Syndrome
Recruiting at 11 trial locations
Age: < 18
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Waitlist Available
Sponsor: Pharming Technologies B.V.
No Placebo Group
Pivotal Trial (Near Approval)
Prior Safety Data
Approved in 1 Jurisdiction
Trial Summary
What is the purpose of this trial?
This trial is testing leniolisib, an oral medicine, in children aged 4 to 11 with a genetic condition called APDS. The medicine aims to calm an overactive part of their immune system to improve their health. Leniolisib has shown benefits in patients with APDS in previous clinical trials.
Eligibility Criteria
This trial is for pediatric patients aged 4 to 11 with Activated PI3K Delta Syndrome (APDS). They must weigh between 13 kg and <45 kg, have a measurable nodal lesion, and a confirmed genetic mutation in the PIK3CD or PIK3R1 gene. Participants should not be on other trials or certain immunosuppressants, and girls who reach menarche must use contraception.Inclusion Criteria
Your heart rate is within a normal range.
- Heart rate (HR):
I am 10 years or older and my heart rate is between 50 to 100 bpm.
See 14 more
Exclusion Criteria
I haven't taken B cell depleters like rituximab in the last 6 months.
I do not have serious heart rhythm problems.
My family has a history of long QT syndrome or Torsades de Pointes.
See 11 more
Treatment Details
Interventions
- Leniolisib (PI3K Inhibitor)
Trial OverviewThe study tests Leniolisib's safety, tolerability, how it's processed by the body (pharmacokinetics), its effects on the disease (pharmacodynamics), and effectiveness in at least 15 children with APDS. It's an open-label trial where everyone gets Leniolisib; there are no comparison groups.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: LeniolisibExperimental Treatment1 Intervention
Leniolisib - Film Coated Tablets Leniolisib tablets in 10 and 30 mg strengths administered orally BID by body weight for 12 weeks for Part I and for 1 year for Part II.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Pharming Technologies B.V.
Lead Sponsor
Trials
23
Recruited
1,400+
University of California, Los Angeles
Collaborator
Trials
1,594
Recruited
10,430,000+
Fortrea
Industry Sponsor
Trials
22
Recruited
5,800+
Aixial Group
Industry Sponsor
Trials
4
Recruited
90+
CMIC Co, Ltd. Japan
Industry Sponsor
Trials
11
Recruited
3,800+
Labcorp Central Laboratory
Collaborator
Trials
2
Recruited
30+
Fortrea
Collaborator
Trials
8
Recruited
2,500+
Aixial Group
Collaborator
Trials
4
Recruited
90+
Labcorp Drug Development, Inc.
Industry Sponsor
Trials
11
Recruited
540+
Labcorp Drug Development Inc
Industry Sponsor
Trials
17
Recruited
2,100+