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)
Age range
18 Years
Sex
ALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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