Leniolisib for APDS

Recruiting at 15 trial locations

Overseen By Jason Bradt, MD

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Pharming Technologies B.V.

Must not be taking: Immunosuppressants, CYP3A inhibitors, others

Disqualifiers: ECG abnormalities, Liver disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

16 Participants Needed

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

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.

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

Treatment Details

Interventions

• Leniolisib

Trial Overview 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.

Participant Groups

Group I: Leniolisib Experimental Treatment 1 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:

• Activated phosphoinositide 3-kinase delta syndrome (APDS)

Who Is Running the Clinical Trial?

Pharming Technologies B.V.

Lead Sponsor

Trials

23

Recruited

1,400+

Labcorp Drug Development Inc

Industry Sponsor

Trials

17

Recruited

2,100+

University of California, Los Angeles

Collaborator

Trials

1,594

Recruited

10,430,000+

CMIC Co, Ltd. Japan

Industry Sponsor

Trials

11

Recruited

3,800+

Aixial Group

Collaborator

Trials

4

Recruited

90+

Labcorp Central Laboratory

Collaborator

Trials

2

Recruited

30+

Fortrea

Collaborator

Trials

8

Recruited

2,500+

Cmed

Collaborator

Trials

2

Recruited

30+

Labcorp Corporation of America Holdings, Inc

Industry Sponsor

Trials

22

Recruited

3,600+

Fortrea

Industry Sponsor

Trials

22

Recruited

5,800+