[CIS PIDD] [cis-pidd] PIK3CD/p110delta study

[Rosenzweig, Sergio (NIH/CC/DLM) [E]]

Dear All,
Please see below info regarding a PIK3CD inhibitor protocol starting at
NIH. Please contact Koneti Rao or Sharon Webster if you have PIK3CD pts
interested in participating.
Thanks,
Sergio

Sergio D. Rosenzweig, MD, PhD
Immunology Service, DLM, CC, NIH




Recruiting APDS/PASLI Patients for Phase 2/3 Study of CDZ173

Investigators at NIAID/NIH Clinical Center are conducting an open-label
research study in
patients with APDS/PASLI (activated phosphoinositide 3-kinase delta
syndrome/p110δ-activating mutation causing senescent T cells,
Lymphadenopathy and immuno­deficiency) to assess the safety and efficacy
of CDZ173, a
proprietary PI3Kδ inhibitor from Novartis. This study follows the
discovery by NIAID that PASLI patients harbor gain of function mutations
in the PIK3CD
Gene (Lucas et al 2014; Angulo et al 2013; Crank et al 2014) and is
recruiting
patients with a confirmed mutation in PIK3CD. Given the specificity of
CDZ173 to selectively inhibit the p110δ subunit of PI3Kδ, this study will
test if
CDZ173 can be used safely to provide effective treatment for PASLI, a newly
described disease with a significant unmet medical need.

How does APDS/PASLI present?
Generally, the majority of patients with APDS present with an unexplained
Common Variable 
Immunodeficiency or Hyper IgM Syndrome, associated with massive
lymphoproliferation (adenopathy and
splenomegaly), autoimmune manifestations and recurrent oto-sino-pulmonary
infections from early infancy; these infections are often severe, leading
to lung damage, particularly bronchiectasis and hearing loss.
We are recruiting for an open-label, non-randomized, within-patient
dose-finding study to assess
the safety and efficacy of CDZ173 in patients with APDS/PASLI·

12-week study with weekly visits.
·      
Inclusion Criteria:
o  
Ages: 16 years to 75 years
o  
Genders: both males and females
o  
Patients who have a documented
APDS/PASLI-associated mutation in PI3K delta gene.
·      
Study patients must have nodal and/or extranodal
lymphoproliferation and corresponding clinical findings and manifestations
compatible with APDS/PASLI.
·      
Exclusion Criteria:
o  
Any medically significant disease or condition
that is unrelated to APDS/PASLI
o  
Other protocol-defined inclusion/exclusion
criteria may apply.
Contact information at NIH

Sharon Webster, RN, BSN, CCRC
LCID, NIAID, NIH
Bldg. 10, RM 11N222
Bethesda, MD  20892 ­ 1888
E-Mail: sharon.webster at nih.gov
Phone: 301-396-2771

V. Koneti Rao, MD, LI/NIAID
Building 10, Rm 12C103
Bethesda, MD 20892
Email: korao at niaid.nih.gov
Phone: 301-496-6502






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