Study of efficacy and safety of CDZ173 in patients with APDS/ PASLI
Research type
Research Study
Full title
An open-label, non-randomized, within-patient dose-finding study followed by a randomized, subject, investigator and sponsor-blinded placebo controlled study to assess the efficacy and safety of CDZ173 in patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/ p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency)
IRAS ID
205698
Contact name
Siobhan Burns
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2014-003876-22
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 11 months, 16 days
Research summary
Research Summary
This study is designed to explore CDZ173, a selective enzyme inhibitor, in patients with a genetically activated enzyme, i.e., patients with APDS (Activated PI3K Delta Syndrome)/PASLI (p110 delta activating mutation causing senescent T cells, lymphadenopathy, and immunodeficiency). The study consists of two parts. Part I was the open label part designed to establish the safety and pharmacokinetics of CDZ173 in the target population, as well as to select the optimal dose to be tested in Part II. Part II is designed to assess efficacy and safety of CDZ173 in this population. The UK will only be taking part in Part II of the study.
The study will assess the change from baseline in the reduction of tissue damage (lesions)in the lymph nodes using MRI or CT imaging.
The study will also measure the change from baseline in percentage of naïve B cells out of total B cells.
Patients will be randomly allocated to one of the two treatment
groups in a 2:1 ratio
to receive either 70mg of the study drug or a matching placebo for a twelve week period.Summary of Results
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REC name
East of England - Cambridge South Research Ethics Committee
REC reference
18/EE/0034
Date of REC Opinion
13 Mar 2018
REC opinion
Further Information Favourable Opinion