NIH Clinical Center Search the Studies: Study Number, Study Title

Protocol Details

An Open-Label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 4 to 11 Years) With APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed by an Open-Label Long-Term Extension

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number

001573-I

Sponsoring Institute

National Institute of Allergy and Infectious Diseases (NIAID)

Recruitment Detail

Type: No longer recruiting/follow-up only
Gender: Male & Female
Min Age: 4 Years
Max Age: 11 Years

Referral Letter Required

Yes

Population Exclusion(s)

None

Keywords

Leniolisib

Recruitment Keyword(s)

None

Condition(s)

Activated Phosphoinositide 3-Kinase Delta Syndrome

Investigational Drug(s)

CDZ 173 (leniolisib)

Investigational Device(s)

None

Intervention(s)

Drug: Leniolisib

Supporting Site

National Institute of Allergy and Infectious Diseases

Background:

Activated phosphoinositide 3-kinase delta syndrome (APDS) is a rare, inherited disease that affects how the immune system works. Symptoms often first appear in children younger than 3 years. Symptoms can include frequent respiratory infections, severe viral illnesses, and slowed growth and development.

Objective:

To test a drug (leniolisib) in children with APDS. (This drug is approved to treat APDS in people older than 12 years.)

Eligibility:

People aged 4 to 11 years with APDS.

Design:

Participants will have 11 study visits in 15 to 18 months.

Screening will take up to 44 days. Participants will have a physical exam, with tests of the blood, urine, and stool. They will have imaging scans and tests of their heart function. They will be tested for HIV, tuberculosis, COVID-19, and hepatitis. Their body will be examined to determine their level of sexual maturity.

Leniolisib is a tablet taken by mouth. It must be swallowed whole. Participants will take the tablet twice a day, every day, for about 15 months. Their caregiver will record doses in a diary; they will also record any changes in their child s health.

Blood and urine tests will be repeated at study visits; heart tests and imaging scans will be repeated on some visits. Participants (and/or their caregivers) will fill in questionnaires. They will answer questions about their symptoms and any changes since they began taking the drug.

Participants will have a follow-up visit about 28 days after they stop taking the drug.

--Back to Top--

Eligibility

INCLUSION CRITERIA:

Participants seen at the NIH must be co-enrolled on NIH IRB-reviewed protocol #14I0206 (Novel Genetic Disorders of the Immune System/Omnibus).


--Back to Top--

Citations:

Not Provided

--Back to Top--

Contacts:

Principal Investigator

Referral Contact

For more information:

V. Koneti Rao, M.D.
National Institute of Allergy and Infectious Diseases (NIAID)
BG 10 RM 12C106
10 CENTER DR
BETHESDA MD 20892
(301) 496-6502
kr191c@nih.gov

Alanvin D. Orpia, R.N.
National Institute of Allergy and Infectious Diseases (NIAID)
National Institutes of Health
Building 10
Room 12C106
10 Center Drive
Bethesda, Maryland 20892
(240) 669-2935
alanvin.orpia@nih.gov

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: TTY Users Dial 7-1-1
ccopr@nih.gov

Clinical Trials Number:

NCT05438407

--Back to Top--