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PI3K Inhibitor

Leniolisib for APDS

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
Be younger than 18 years old
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 studies the safety & effectiveness of a drug in 15 kids with APDS, a rare genetic condition.

Who is the study for?
This trial is for children aged 1 to 6 with Activated PI3K Delta Syndrome (APDS), who can take oral medication and have a confirmed genetic mutation related to APDS. They should weigh between 8 and 37 kg, have normal blood pressure and heart rate, and not be on certain immunosuppressants or medications that affect the immune system.Check my eligibility
What is being tested?
The study tests Leniolisib's safety, tolerability, how it affects the body (pharmacokinetics/pharmacodynamics), and effectiveness in at least 15 pediatric patients with APDS. It's an open-label trial where everyone gets the same treatment without any comparison group.See study design
What are the potential side effects?
While specific side effects of Leniolisib are not listed here, common ones may include digestive issues, changes in liver function tests, potential allergic reactions or infections due to immune system impact. Close monitoring will identify any adverse effects during the trial.

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
+4 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: LeniolisibExperimental Treatment1 Intervention
Leniolisib film-coated granules in 10, 15 and 20 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?

Labcorp Drug Development IncIndustry Sponsor
16 Previous Clinical Trials
3,171 Total Patients Enrolled
University of California, Los AngelesOTHER
1,532 Previous Clinical Trials
10,265,253 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: NCT05693129 — Phase 3
Acute Promyelocytic Leukemia Research Study Groups: Leniolisib
Acute Promyelocytic Leukemia Clinical Trial 2023: Leniolisib Highlights & Side Effects. Trial Name: NCT05693129 — Phase 3
Leniolisib (PI3K Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05693129 — Phase 3

Frequently Asked Questions

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

Are there any age limitations for participating in this trial?

"The trial seeks to enrol patients between 1 and 6 years of age."

Answered by AI

How many medical centers are currently carrying out this trial?

"This clinical study is taking place at the National Institutes of Health in Bethesda, Maryland; The University of North carolina in Chapel Hill, North Carolina and Rainbow Childrens Hospital in Shaker Heights, Ohio with 4 additional sites."

Answered by AI

What precautionary measures should be taken when administering Leniolisib to patients?

"Previous clinical evidence suggests that Leniolisib is safe, thus it was given a rating of 3 on our scale. The Phase 3 trial demonstrates its efficacy and multiple data points reinforce the drug's safety."

Answered by AI

Are there still opportunities to partake in this experiment?

"Data hosted on clinicialtrials.gov indicates that, despite initially being listed on March 30th 2023 and later updated in January 12th 2023, this clinical trial has stopped recruiting patients at the moment. Nevertheless, there are currently two other trials actively registering candidates for participation."

Answered by AI

Who meets the criteria for taking part in this research endeavor?

"This trial is seeking 15 children aged 1 to 6 years old that have been diagnosed with acute promyelocytic leukemia (apl). Furthermore, these participants must fulfill the following criteria: They can be male or female; their body weight should range from 8 kg to 37Kg; they possess a PIK3CD (APDS1) or PIK3R1 mutation; MRI or CT scans reveal at least one measurable nodal lesion in the last six months; other clinical findings point towards APDS as well. Additionally, it's important that they are able to ingest medications without difficulty and their blood pressure falls within acceptable"

Answered by AI

What goals is this clinical trial seeking to attain?

"The clinical trial is assessing outcomes across a one-year plus thirty day timeframe. The primary goal of the study is to measure changes in laboratory test results from baseline, and secondary objectives include evaluating leniolisib's maximum concentration (Cmax) and time to maximum concentration (Tmax), as well as reductions in lymphadenopathy measured via MRI or low-dose CT scans. Summary statistics will be provided by visit, with arithmetic mean with SD of absolute values plotted against change from baseline values over time."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Leniolisib for Activated PI3K Delta Syndrome (APDS)
2023. "Leniolisib for Activated PI3K Delta Syndrome (APDS)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05693129.
~10 spots leftby Sep 2025
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