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PI3K Inhibitor

Leniolisib for Activated PI3K Delta Syndrome

Phase 3
Recruiting
Research Sponsored by Pharming Technologies B.V.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patient weighs ≥13 kg and <45 kg at baseline.
Patient has at least 1 measurable nodal lesion on magnetic resonance imaging/low-dose computed tomography within 6 months of screening.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline to end of 12 weeks, & from baseline to through study completion, an average of 1 year
Awards & highlights

Study Summary

This trial will study the safety and effectiveness of a new drug called leniolisib in kids with a rare condition called activated phosphoinositide 3-kinase delta syndrome.

Who is the study for?
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.Check my eligibility
What is being tested?
The 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.See study design
What are the potential side effects?
Potential side effects of Leniolisib aren't specified here but may include reactions related to immune system suppression given its pharmacological target. Side effects could also relate to drug components like lactose monohydrate or hypromellose.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I weigh between 13 kg and 45 kg.
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I have a measurable cancerous node identified by MRI or CT scan in the last 6 months.
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I can take pills without any trouble.
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Your heart rate is within a normal range.
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My heart rate is between 60 and 140 beats per minute.
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I am 10 years or older with a heart rate between 50 to 100 bpm.
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I have given my written consent for the study, as approved by an ethics committee.
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I am a woman not able to have children or a man willing to use contraception.
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I, or my child, will follow study rules about menstruation if it starts after screening.
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I will not engage in heterosexual activity for the study duration and 6 months after.
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I will use effective birth control for 30 days after my last study procedure.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline to end of 12 weeks, & from baseline to through study completion, an average of 1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to end of 12 weeks, & from baseline to through study completion, an average of 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Part I & II: Change from baseline in clinical laboratory test results
Part I & II: Change from baseline in electrocardiograms (ECGs)
Part I & II: Change from baseline in growth and physical development
+5 more
Secondary outcome measures
Part I and II: Key secondary efficacy outcomes for Part I include incidence of infections and use of antibiotics.
Part I and II: Pediatric Quality of Life Inventory (PedsQLTM) Parent Report for Children Questionnaire 4.0 Generic Core Scales
Part I: To assess the maximum concentration (Cmax) of leniolisib in pediatric patients (aged 4 to 11 years) with APDS
+6 more

Trial Design

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 Location

Who is running the clinical trial?

University of California, Los AngelesOTHER
1,525 Previous Clinical Trials
10,279,723 Total Patients Enrolled
Pharming Technologies B.V.Lead Sponsor
20 Previous Clinical Trials
1,678 Total Patients Enrolled
CMIC Co, Ltd. JapanIndustry Sponsor
10 Previous Clinical Trials
3,788 Total Patients Enrolled

Media Library

Leniolisib (PI3K Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05438407 — Phase 3
Acute Promyelocytic Leukemia Research Study Groups: Leniolisib
Acute Promyelocytic Leukemia Clinical Trial 2023: Leniolisib Highlights & Side Effects. Trial Name: NCT05438407 — Phase 3
Leniolisib (PI3K Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05438407 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Could you please elaborate on the age requirements for participants in this trial?

"This clinical trial is looking for pediatric patients aged 4-11 years old."

Answered by AI

Could I qualify to be a test subject for this experiment?

"This study is looking for 15 participants who have APDS and are between the ages of 4 and 11. The most important criteria that candidates must meet are as follows: Patient weighs ≥13 kg and <45 kg at baseline., Patient is male or female and between the age of 4 to 11 years old at time of the first study procedure. Females should be of nonchildbearing potential at screening., Patient has at least 1 measurable nodal lesion on magnetic resonance imaging (MRI)/low-dose computed tomography (CT)., Patient has nodal or extranodal lymphoproliferation and clinical findings consistent with APDS"

Answered by AI

What have been the findings of studies on Leniolisib's safety profile?

"Leniolisib's safety is well documented, as this drug has progressed to Phase 3 clinical trials. In our estimation, Leniolisib carries a risk level of 3."

Answered by AI

Can new patients still enroll in this clinical trial?

"According to the website clinicaltrials.gov, this study is no longer recruiting patients for participation. The trial was originally posted on 8/31/2022 but was most recently edited on 7/8/2022. 1 other trials are enrolling participants currently."

Answered by AI
~7 spots leftby Feb 2025