Safety, Pharmacokinetic (PK) and Pharmacodynamic (PD) Study of Repeat Doses of Inhaled GSK2269557 in Patients With APDS/PASLI
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02593539|
Recruitment Status : Recruiting
First Posted : November 2, 2015
Last Update Posted : August 9, 2019
|Condition or disease||Intervention/treatment||Phase|
|Activated PI3K-delta Syndrome||Drug: GSK2269557 DPI 500 mcg||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-label, Single Arm Study to Investigate the Safety, Pharmacokinetics and Pharmacodynamics of Repeat Doses of Inhaled GSK2269557 in Patients With APDS/PASLI|
|Actual Study Start Date :||July 22, 2016|
|Estimated Primary Completion Date :||May 13, 2020|
|Estimated Study Completion Date :||May 13, 2020|
Experimental: GSK2269557 DPI 1000 mcg
Subjects will receive GSK2269557 1000 mcg once daily via dry powder inhaler (DPI) for 84 days
Drug: GSK2269557 DPI 500 mcg
Subjects will receive 2 inhalations of GSK2269557 500 mcg in DPI (total dose: 1000 mcg) to be taken every day before breakfast (with the exception of days when the subjects have a planned visit to the study clinic. On those days, they will be dosed at the clinic). The subject should hold their breath for 10 seconds before exhaling. Inhalations should be taken approximately 30 seconds apart.
- Number of subjects with any adverse events (AE) [ Time Frame: Up to 6 months ]An AE is any untoward medical occurrence in a patient or clinical investigation subject, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
- Systolic and diastolic blood pressure as a measure of safety [ Time Frame: Up to 7.5 months ]Systolic and diastolic blood pressure will be measured in semi-supine position after 5 minutes rest.
- Temperature as a measure of safety [ Time Frame: Up to 7.5 months ]Temperature will be measured in semi-supine position after 5 minutes rest.
- Pulse rate as a measure of safety [ Time Frame: Up to 7.5 months ]Pulse rate will be measured in semi-supine position after 5 minutes rest.
- Respiratory rate as a measure of safety [ Time Frame: Up to 7.5 months ]Respiratory rate will be measured in semi-supine position after 5 minutes rest.
- 12-lead electrocardiogram (ECG) as a measure of safety [ Time Frame: Up to 7.5 months ]Single (triplicate at screening) 12-lead ECGs will be obtained at each time point during the study using an ECG machine that automatically calculates the heart rate and measures PR, QRS, QT, and QTc intervals.
- Composite of hematology parameters as a measure of safety [ Time Frame: Up to 7.5 months ]The following hematology parameters will be measured: Platelet Count, red blood cell (RBC) Count, Hemoglobin, Hematocrit, mean corpuscular volume (MCV), mean corpuscular hemoglobin (MCH), white blood cells (WBC) count, Neutrophils, Lymphocytes, Monocytes, Eosinophils, and Basophils
- Composite of clinical chemistry parameters as a measure of safety [ Time Frame: Up to 7.5 months ]The following clinical chemistry parameters will be measured: blood urea nitrogen (BUN)/Urea, Creatinine, Glucose, C-reactive protein (CRP), Potassium, Sodium, Calcium, aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase, Total and direct bilirubin, Total Protein, and Albumin
- Composite of urinalysis parameters as a measure of safety [ Time Frame: Up to 7.5 months ]Specific gravity, power of hydrogen (pH), glucose, protein, blood and ketones measured by dipstick; and microscopic examination (if blood or protein is abnormal)
- Spirometry: Forced expiratory volume in one second (FEV1) [ Time Frame: Up to 7.5 months ]FEV1 is the maximal amount of air that can be forcefully exhaled in one second.
- GSK2269557 trough plasma concentration following single and repeated treatment [ Time Frame: Pre-dose, 5 minutes, 3 hours and 24 hours post-dose on Day 1 and pre-dose on Days 2, 14, and 83 and early withdrawal ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02593539
|Contact: US GSK Clinical Trials Call Center||877-379-3718||GSKClinicalSupportHD@gsk.com|
|Contact: EU GSK Clinical Trials Call Center||+44 (0) 20 89904466||GSKClinicalSupportHD@gsk.com|
|GSK Investigational Site||Recruiting|
|Cambridge, United Kingdom, CB2 0GG|
|Contact: US GSK Clinical Trials Call Center 877-379-3718 GSKClinicalSupportHD@gsk.com|
|Contact: EU GSK Clinical Trials Call Centre +44 (0) 20 8990 4466 GSKClinicalSupportHD@gsk.com|
|Principal Investigator: Edward Banham-Hall|
|Study Director:||GSK Clinical Trials||GlaxoSmithKline|