This is an open-label study conducted to investigate safety, pharmacokinetics and pharmacodynamics of repeat doses of inhaled nemiralisib (NEMI) in participants with activated phosphoinositide 3-kinase (PI3K) delta syndrome /p110 delta-activating mutation causing senescent T Cells, lymphadenopathy and immunodeficiency (APDS/PASLI)
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Number of Participants With Any Serious Adverse Events (SAEs) and Any Non-serious Adverse Events (Non-SAEs)
Timeframe: Upto 7.5 months
Change From Baseline in Diastolic Blood Pressure (DBP) and Systolic Blood Pressure (SBP)
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
Change From Baseline in Pulse Rate
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
Change From Baseline in Respiratory Rate
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
Change From Baseline in Body Temperature
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
Change From Baseline in Electrocardiogram (ECG) Mean Heart Rate
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Change From Baseline in PR Interval, QRS Duration, Uncorrected QT Interval, QT Corrected Interval-Fredericia Interval (QTcF) and QTc Corrected by Bazett's Formula (QTcB)
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Change From Baseline in Clinical Chemistry Parameters: Alanine Aminotransferase (ALT), Alkaline Phosphatase (ALP) and Aspartate Aminotransferase (AST)
Timeframe: Baseline (Day -1) and at Days 14, 28, 56 and 83
Change From Baseline in Clinical Chemistry Parameters : Albumin and Total Protein
Timeframe: Baseline (Day -1) and at Days 14, 28, 56 and 83
Change From Baseline Values in Clinical Chemistry Parameters: Sodium, Potassium, Calcium, Glucose and Urea
Timeframe: Baseline (Day -1) and at Days 14, 28, 56 and 83
Change From Baseline Values in Clinical Chemistry Parameters: Direct Bilirubin, Total Bilirubin and Creatinine
Timeframe: Baseline (Day -1) and at Days 14, 28, 56 and 83
Change From Baseline Values in Clinical Chemistry Parameter: C-Reactive Protein
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Change From Baseline for Hematology Parameters: Basophil, Eosinophils, White Blood Cells (WBC), Lymphocytes, Neutrophils, Monocytes and Platelets
Timeframe: Baseline (Day -1) and at Days 14, 28, 56 and 83
Change From Baseline for Hematology Parameter: Hemoglobin
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Change From Baseline for Hematology Parameter: Hematocrit
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Change From Baseline in Hematology Parameter: Mean Corpuscular Volume (MCV)
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Change From Baseline in Hematology Parameter: Mean Corpuscular Hemoglobin (MCH)
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Change From Baseline for Hematology Parameter: Red Blood Cell Count
Timeframe: Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1)
Timeframe: Baseline (Day 1: pre-dose) and at Day 1: 1 Hour post-dose; Day 2: 1 Hour post-dose; Day 14: Pre-dose; Day 14: 1 Hour post-dose and Day 83: Pre-dose