Leniolisib for APDS

Recruiting in Palo Alto (17 mi)

+15 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Pharming Technologies B.V.

Must not be taking: Immunosuppressants, CYP3A inhibitors, others

Disqualifiers: ECG abnormalities, Liver disease, others

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 1 jurisdiction

Trial Summary

What is the purpose of this trial?This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 1 to 6 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)

Will I have to stop taking my current medications?

The trial requires stopping certain medications before starting, such as immunosuppressive drugs and some heart medications. If you're taking any of these, you may need to stop them for a specific time before joining the study.

Eligibility Criteria

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.

Inclusion Criteria

Institutional review board- or IEC-approved written informed consent or assent and privacy language as per national and local regulations must be obtained from the patient and/or parent or legal guardian prior to any study-related procedures

I can take pills without any trouble.

Patient parent or legal guardian is willing and able to complete the informed consent or assent process and comply with study procedures and visit schedule

+7 more

Exclusion Criteria

I have had cancer before.

My diabetes is not well-managed currently.

I, or my guardian, have not refused to sign the consent form.

+12 more

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive leniolisib orally based on weight for 12 weeks to assess safety and efficacy

12 weeks

Regular visits for monitoring and assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive leniolisib for long-term safety follow-up

1 year

Participant Groups

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.

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.

Leniolisib is already approved in United States for the following indications:

🇺🇸 Approved in United States as Joenja for: