3.236.145.153
dgid:
enl:
npi:0
-Advertisement-
-Advertisement-
Genetic and Congenital

First patient enrolled in pediatric clinical trial of leniolisib for APDS

Posted on

The first patient has been enrolled in a Phase 3 clinical trial evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children with activated phosphoinositide 3-kinase delta syndrome (APDS), according to a press release by Pharming Group N.V.

The safety, tolerability, and efficacy of leniolisib will be evaluated in approximately 15 children aged 4 to 11 years with APDS at sites in the United States, Europe, and Japan.

The study’s primary efficacy endpoints are a reduction in index lymph node size and an increased proportion of naïve B cells out of total B cells from baseline at 12 weeks. Secondary endpoints include an assessment of the ability of leniolisib to modify health-related quality of life based on measures of physical, social, emotional, and school functioning using a validated patient questionnaire.

“In treating APDS, the current standard of care is to use an array of supportive therapies. While these therapies can treat some of the manifestations of APDS, they do not target the underlying cause of the disease. Pharming’s studies of leniolisib in children with APDS are important for evaluating the possibility of minimizing symptoms earlier in the disease progression,” said Manish Butte, MD, PhD, E. Richard Stiehm Endowed Chair, Professor with tenure in the Department of Pediatrics, and Division Chief of Immunology, Allergy, and Rheumatology at UCLA.

There is currently no approved treatment for APDS.

Read the full press release here.

-Advertisement-
-Advertisement-
-Advertisement-
-Advertisement-
-Advertisement-
-Advertisement-