An open-label, single arm study to investigate the safety, pharmacokinetics and pharmacodynamics of repeat doses of inhaled nemiralisib in patients with APDS/PASLI

GlaxoSmithKline Research & Development Ltd0 sites20 target enrollmentNovember 30, 2015

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Not specified
Sponsor: GlaxoSmithKline Research & Development Ltd
Enrollment: 20
Status: Active, not recruiting
Last Updated: 5 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

November 30, 2015

End Date

TBD

Last Updated

5 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

GlaxoSmithKline Research & Development Ltd

Eligibility Criteria

Inclusion Criteria

•A subject will be eligible for inclusion in this study only if all of the following criteria apply:
•1\. Male and female subjects aged 18 or older at the time of signing the informed consent.
•TYPE OF SUBJECT AND DIAGNOSIS INCLUDING DISEASE SEVERITY
•2\. Patients with a clinical phenotype consistent with APDS, including a history of recurrent (frequency greater than would be expected in an immunocompetent individual) ear, sinus or pulmonary infections, and who have a known type 1 APDS associated genetic PI3Kdelta mutation (e.g. E1021K, N334K, E525K and C416R) or type 2 APDS\-associated mutation
•3\. Body weight \=40 kg and body mass index (BMI) \=17kg/m2 (inclusive)
•4\. Female subject. A female subject is eligible to participate if she is not pregnant (as confirmed by a negative urine human chorionic gonadotrophin (hCG) test), not lactating, and at least one of the following conditions applies:
•\- Non\-reproductive potential defined as:
•o Pre\-menopausal females with one of the following:
•– Documented tubal ligation
•– Documented hysteroscopic tubal occlusion procedure with followup confirmation of bilateral tubal occlusion

Exclusion Criteria

•A subject will not be eligible for inclusion in this study if any of the following criteria apply:
•CONCURRENT CONDITIONS/MEDICAL HISTORY (INCLUDES LIVER
•FUNCTION AND QTc INTERVAL)
•1\. Alanine aminotransferase (ALT) \>2xULN and bilirubin \>1\.5xULN (isolated bilirubin \>1\.5xULN is acceptable if bilirubin is fractionated and direct bilirubin \<35%).
•2\. Current or chronic history of liver disease except where hepatomegaly is identified by their clinician to be secondary to APDS, or known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones).
•3\. QTc \> 450 millisecond (msec) or QTc \> 480 msec in subjects with Bundle Branch Block
•NOTES: The QTc is the QT interval corrected for heart rate (HR) according to Bazett’s formula (QTcB), Fridericia’s formula (QTcF), and/or another method, machineread or manually over\-read.
•The specific formula that will be used to determine eligibility and discontinuation for an individual subject should be determined prior to initiation of the study. In other words, several different formulae cannot be used to calculate the QTc for an individual subject and then the lowest QTc value used to include or discontinue the subject from the trial.
•For purposes of data analysis, QTcB, QTcF, another QT correction formula, or a composite of available values of QTc will be used as specified in the Reporting and Analysis Plan (RAP).
•4\. A subject with a clinical abnormality or laboratory parameter(s) which is/are not specifically listed in the inclusion or exclusion criteria, outside the reference range for the population being studied may be included only if the investigator, in consultation with the Medical Monitor if required, agree and document that the finding is unlikely to introduce additional risk factors and will not interfere with the study procedures.