NCT02593539 is a Phase 2 clinical trial of Nemiralisib in Activated PI3K-delta Syndrome, sponsored by GlaxoSmithKline.

Who is sponsoring NCT02593539?

NCT02593539 is sponsored by GlaxoSmithKline.

What drugs are being tested in NCT02593539?

Interventions in NCT02593539: Nemiralisib.

What condition does NCT02593539 study?

NCT02593539 studies Activated PI3K-delta Syndrome.

Is NCT02593539 still recruiting?

NCT02593539 is currently completed. Last updated 18 June 2021.

Are results published for NCT02593539?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2021-06-18 · How we verify

NCT02593539

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Safety, Pharmacokinetic (PK) and Pharmacodynamic (PD) Study of Repeat Doses of Inhaled Nemiralisib in Patients With APDS/PASLI

Completed Phase 2 Results posted Last updated 18 June 2021

What this trial tests

Phase 2 trial testing Nemiralisib in Activated PI3K-delta Syndrome in 5 participants. Completed in 4 June 2020.

Timeline

22 July 2016

Primary endpoint
4 June 2020

4 June 2020

Quick facts

Lead sponsor	GlaxoSmithKline
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	5
Start date	22 July 2016
Primary completion	4 June 2020
Estimated completion	4 June 2020
Sites	1 location across United Kingdom

Drugs / interventions tested

Nemiralisib — full drug profile →

Conditions studied

Activated PI3K-delta Syndrome — all drugs for Activated PI3K-delta Syndrome →

Sponsor

GlaxoSmithKline — full company profile →

Who can join

18 and older, any sex, with Activated PI3K-delta Syndrome. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Any Serious Adverse Events (SAEs) and Any Non-serious Adverse Events (Non-SAEs) Primary · Upto 7.5 months

An adverse event is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is defined as any untoward medical occurrence that; results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, is a congenital anomaly/birth defect, other situations as judged by physician. Number of participants with any SAE and non-SAEs are presented.

Any non-SAE

Group	Value	95% CI
All NEMI	5

Any SAE

Group	Value	95% CI
All NEMI	0

Change From Baseline in Diastolic Blood Pressure (DBP) and Systolic Blood Pressure (SBP) Primary · Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84

SBP and DBP were measured in participants in a semi-supine position after 5 minutes rest. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Not applicable (NA) indicates that standard deviation could not be calculated as a single participant was analyzed.

DBP, Day 14, n= 5

Group	Value	95% CI
All NEMI	1.0	± 6.44

DBP, Day 28, n= 4

Group	Value	95% CI
All NEMI	4.0	± 6.98

DBP, Day 56, n= 4

Group	Value	95% CI
All NEMI	8.5	± 5.80

DBP, Day 83, n= 4

Group	Value	95% CI
All NEMI	2.0	± 4.40

DBP, Day 84 n= 1

Group	Value	95% CI
All NEMI	17.0	± NA

SBP, Day 14, n= 5

Group	Value	95% CI
All NEMI	-1.0	± 3.67

SBP, Day 28, n= 4

Group	Value	95% CI
All NEMI	5.3	± 8.54

SBP, Day 56, n= 4

Group	Value	95% CI
All NEMI	10.0	± 9.31

Change From Baseline in Pulse Rate Primary · Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84

Pulse rate was measured in participants in a semi-supine position after 5 minutes rest. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. NA indicates that standard deviation could not be calculated as a single participant was analyzed.

Day 14, n= 5

Group	Value	95% CI
All NEMI	3.2	± 7.73

Day 28, n= 4

Group	Value	95% CI
All NEMI	2.0	± 10.86

Day 56, n= 4

Group	Value	95% CI
All NEMI	6.8	± 9.43

Day 83, n= 4

Group	Value	95% CI
All NEMI	4.8	± 7.76

Day 84 n= 1

Group	Value	95% CI
All NEMI	7.0	± NA

Change From Baseline in Respiratory Rate Primary · Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84

Respiratory rate was measured in participants in a semi-supine position after 5 minutes rest. . Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. NA indicates that standard deviation could not be calculated as a single participant was analyzed.

Day 14, n= 5

Group	Value	95% CI
All NEMI	0.2	± 2.28

Day 28, n= 4

Group	Value	95% CI
All NEMI	0.8	± 4.57

Day 56, n= 4

Group	Value	95% CI
All NEMI	1.5	± 5.26

Day 83, n= 4

Group	Value	95% CI
All NEMI	1.0	± 2.45

Day 84 n= 1

Group	Value	95% CI
All NEMI	2.0	± NA

Change From Baseline in Body Temperature Primary · Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84

Temperature was measured in participants in a semi-supine position after 5 minutes rest. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. NA indicates that standard deviation could not be calculated as a single participant was analyzed.

Day 14, n= 5

Group	Value	95% CI
All NEMI	0.10	± 0.158

Day 28, n= 4

Group	Value	95% CI
All NEMI	0.17	± 0.350

Day 56, n= 4

Group	Value	95% CI
All NEMI	0.07	± 0.171

Day 83, n= 4

Group	Value	95% CI
All NEMI	0.17	± 0.479

Day 84 n= 1

Group	Value	95% CI
All NEMI	0.50	± NA

Change From Baseline in Electrocardiogram (ECG) Mean Heart Rate Primary · Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83

Single 12-lead ECGs were recorded at indicated timepoints using an ECG machine that automatically calculated the heart rate. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.

Day 14, n= 5

Group	Value	95% CI
All NEMI	-1.0	± 3.94

Day 28, n= 4

Group	Value	95% CI
All NEMI	-0.8	± 2.99

Day 56, n= 4

Group	Value	95% CI
All NEMI	5.3	± 7.54

Day 83, n= 4

Group	Value	95% CI
All NEMI	1.0	± 2.31

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) Primary · Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83

Twelve lead ECGs were recorded at indicated timepoints. At each time point, ECG machine automatically measured QRS duration, uncorrected QT interval, QTcF interval and QTcB. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.

PR, Day 14, n= 5

Group	Value	95% CI
All NEMI	-2.2	± 9.23

PR, Day 28, n= 4

Group	Value	95% CI
All NEMI	-4.8	± 4.27

PR, Day 56, n= 4

Group	Value	95% CI
All NEMI	-3.3	± 11.18

PR, Day 83, n= 4

Group	Value	95% CI
All NEMI	-6.0	± 11.80

QRS Duration, Day 14, n= 5

Group	Value	95% CI
All NEMI	-5.6	± 7.33

QRS Duration, Day 28, n= 4

Group	Value	95% CI
All NEMI	-4.5	± 11.03

QRS Duration, Day 56, n= 4

Group	Value	95% CI
All NEMI	-6.5	± 8.81

QRS Duration, Day 83, n= 4

Group	Value	95% CI
All NEMI	-6.0	± 7.79

Change From Baseline in Clinical Chemistry Parameters: Alanine Aminotransferase (ALT), Alkaline Phosphatase (ALP) and Aspartate Aminotransferase (AST) Primary · Baseline (Day -1) and at Days 14, 28, 56 and 83

Blood samples were collected for the analysis of clinical parameters including ALT, ALP and AST. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.

ALT, Day 14, n= 5

Group	Value	95% CI
All NEMI	1.0	± 2.12

ALT, Day 28, n= 4

Group	Value	95% CI
All NEMI	0.8	± 1.71

ALT, Day 56, n= 4

Group	Value	95% CI
All NEMI	0.0	± 1.63

ALT, Day 83, n= 4

Group	Value	95% CI
All NEMI	1.3	± 7.50

AST, Day 14, n= 5

Group	Value	95% CI
All NEMI	-0.8	± 1.48

AST, Day 28, n= 4

Group	Value	95% CI
All NEMI	0.8	± 2.22

AST, Day 56, n= 4

Group	Value	95% CI
All NEMI	-0.8	± 4.79

AST, Day 83, n= 4

Group	Value	95% CI
All NEMI	-1.0	± 7.70

Change From Baseline in Clinical Chemistry Parameters : Albumin and Total Protein Primary · Baseline (Day -1) and at Days 14, 28, 56 and 83

Blood samples were collected for the analysis of clinical chemistry parameter-albumin and total protein. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.

Albumin, Day 14, n= 5

Group	Value	95% CI
All NEMI	2.6	± 2.07

Albumin, Day 28, n= 4

Group	Value	95% CI
All NEMI	1.3	± 3.20

Albumin, Day 56, n= 4

Group	Value	95% CI
All NEMI	2.8	± 2.36

Albumin, Day 83, n= 4

Group	Value	95% CI
All NEMI	2.0	± 3.16

Total Protein, Day 14, n= 5

Group	Value	95% CI
All NEMI	4.0	± 1.58

Total Protein, Day 28, n= 4

Group	Value	95% CI
All NEMI	1.3	± 3.59

Total Protein, Day 56, n= 4

Group	Value	95% CI
All NEMI	4.0	± 4.76

Total Protein, Day 83, n= 4

Group	Value	95% CI
All NEMI	4.3	± 2.50

Change From Baseline Values in Clinical Chemistry Parameters: Sodium, Potassium, Calcium, Glucose and Urea Primary · Baseline (Day -1) and at Days 14, 28, 56 and 83

Blood samples were collected for the analysis of clinical parameters including sodium, potassium, calcium, glucose and urea. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.

Sodium, Day 14, n= 5

Group	Value	95% CI
All NEMI	-0.2	± 2.68

Sodium, Day 28, n= 4

Group	Value	95% CI
All NEMI	0.5	± 0.58

Sodium, Day 56, n= 4

Group	Value	95% CI
All NEMI	-0.8	± 2.06

Sodium, Day 83, n= 4

Group	Value	95% CI
All NEMI	0.0	± 1.83

Potassium, Day 14, n= 5

Group	Value	95% CI
All NEMI	0.24	± 0.397

Potassium, Day 28, n= 4

Group	Value	95% CI
All NEMI	0.30	± 0.346

Potassium, Day 56, n= 4

Group	Value	95% CI
All NEMI	0.35	± 0.370

Potassium, Day 83, n= 4

Group	Value	95% CI
All NEMI	0.38	± 0.275

Change From Baseline Values in Clinical Chemistry Parameters: Direct Bilirubin, Total Bilirubin and Creatinine Primary · Baseline (Day -1) and at Days 14, 28, 56 and 83

Blood samples were collected for the analysis of clinical chemistry parameters: direct bilirubin, total bilirubin and creatinine. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.

Direct bilirubin, Day 14, n= 5

Group	Value	95% CI
All NEMI	-1.0	± 0.71

Direct bilirubin, Day 28, n= 4

Group	Value	95% CI
All NEMI	-1.8	± 0.96

Direct bilirubin, Day 56, n= 4

Group	Value	95% CI
All NEMI	-0.5	± 1.00

Direct bilirubin, Day 83, n= 4

Group	Value	95% CI
All NEMI	-0.5	± 1.00

Total bilirubin, Day 14, n= 5

Group	Value	95% CI
All NEMI	-1.8	± 1.64

Total bilirubin, Day 28, n= 4

Group	Value	95% CI
All NEMI	-3.0	± 2.45

Total bilirubin, Day 56, n= 4

Group	Value	95% CI
All NEMI	-0.8	± 1.89

Total bilirubin, Day 83, n= 4

Group	Value	95% CI
All NEMI	-1.5	± 2.52

Change From Baseline Values in Clinical Chemistry Parameter: C-Reactive Protein Primary · Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83

Blood samples were collected for the analysis of clinical chemistry parameter:C-Reactive Protein. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value.

Day 14, n= 5

Group	Value	95% CI
All NEMI	0.56	± 1.592

Day 28, n= 4

Group	Value	95% CI
All NEMI	0.75	± 1.085

Day 56, n= 4

Group	Value	95% CI
All NEMI	2.75	± 6.222

Day 83, n= 4

Group	Value	95% CI
All NEMI	3.90	± 7.141

Adverse events — posted to ClinicalTrials.gov

Time frame: Non-serious and serious adverse events were collected up to 7.5 months. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

All NEMI

Serious: 0/5 (0%)

Deaths: 0/5

Other adverse events (38 terms — click to expand)

Reaction	System	All NEMI
Cough	Respiratory, thoracic and mediastinal disorders	—
Nasopharyngitis	Infections and infestations	—
Headache	Nervous system disorders	—
Vomiting	Gastrointestinal disorders	—
Seasonal allergy	Immune system disorders	—
Bronchospasm	Respiratory, thoracic and mediastinal disorders	—
Epistaxis	Respiratory, thoracic and mediastinal disorders	—
Nasal congestion	Respiratory, thoracic and mediastinal disorders	—
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	—
Pleuritic pain	Respiratory, thoracic and mediastinal disorders	—
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	—
Wheezing	Respiratory, thoracic and mediastinal disorders	—
Chest discomfort	General disorders	—
Fatigue	General disorders	—
Feeling hot	General disorders	—
Feeling of body temperature change	General disorders	—
Pain	General disorders	—
Swelling face	General disorders	—
Abdominal discomfort	Gastrointestinal disorders	—
Abdominal pain	Gastrointestinal disorders	—
Abdominal tenderness	Gastrointestinal disorders	—
Constipation	Gastrointestinal disorders	—
Diarrhoea	Gastrointestinal disorders	—
Mouth ulceration	Gastrointestinal disorders	—
Oral candidiasis	Infections and infestations	—
Sinusitis	Infections and infestations	—
Fungal skin infection	Infections and infestations	—
Skin bacterial infection	Infections and infestations	—
Migraine	Nervous system disorders	—
Contusion	Injury, poisoning and procedural complications	—
Skin abrasion	Injury, poisoning and procedural complications	—
Back pain	Musculoskeletal and connective tissue disorders	—
Myalgia	Musculoskeletal and connective tissue disorders	—
Iron deficiency	Metabolism and nutrition disorders	—
Vitamin B complex deficiency	Metabolism and nutrition disorders	—
Vitamin D deficiency	Metabolism and nutrition disorders	—
Product taste abnormal	Product Issues	—
Rash	Skin and subcutaneous tissue disorders	—

Data from ClinicalTrials.gov NCT02593539 adverse events section.

Sponsor's own description

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)

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

PI3Kδ and primary immunodeficiencies.
Lucas CL, Chandra A, Nejentsev S, Condliffe AM, et al · · 2016 · cited 266× · PMID 27616589 · DOI 10.1038/nri.2016.93
Conformational disruption of PI3Kδ regulation by immunodeficiency mutations in <i>PIK3CD</i> and <i>PIK3R1</i>.
Dornan GL, Siempelkamp BD, Jenkins ML, Vadas O, et al · · 2017 · cited 95× · PMID 28167755 · DOI 10.1073/pnas.1617244114
A randomized, placebo-controlled phase 3 trial of the PI3Kδ inhibitor leniolisib for activated PI3Kδ syndrome.
Rao VK, Webster S, Šedivá A, Plebani A, et al · · 2023 · cited 81× · PMID 36399712 · DOI 10.1182/blood.2022018546
Activated PI3 Kinase Delta Syndrome: From Genetics to Therapy.
Michalovich D, Nejentsev S. · · 2018 · cited 78× · PMID 29535736 · DOI 10.3389/fimmu.2018.00369
Inborn Errors of Immunity With Immune Dysregulation: From Bench to Bedside.
Delmonte OM, Castagnoli R, Calzoni E, Notarangelo LD. · · 2019 · cited 76× · PMID 31508401 · DOI 10.3389/fped.2019.00353
The Treatment of Activated PI3Kδ Syndrome.
Coulter TI, Cant AJ. · · 2018 · cited 71× · PMID 30245694 · DOI 10.3389/fimmu.2018.02043
PI3Kδ hyper-activation promotes development of B cells that exacerbate Streptococcus pneumoniae infection in an antibody-independent manner.
Stark AK, Chandra A, Chakraborty K, Alam R, et al · · 2018 · cited 66× · PMID 30093657 · DOI 10.1038/s41467-018-05674-8
Novel PIK3CD mutations affecting N-terminal residues of p110δ cause activated PI3Kδ syndrome (APDS) in humans.
Takeda AJ, Zhang Y, Dornan GL, Siempelkamp BD, et al · · 2017 · cited 63× · PMID 28414062 · DOI 10.1016/j.jaci.2017.03.026

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Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT02593539 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by GlaxoSmithKline
Last refreshed: 18 June 2021

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02593539.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing

NCT02593539

Quick facts

Drugs / interventions tested

Conditions studied

Sponsor

Who can join

Results — posted to ClinicalTrials.gov

Adverse events — posted to ClinicalTrials.gov

Sponsor's own description

Publications & conference data

Related trials

Other trials of Nemiralisib

Other GlaxoSmithKline trials

Verify against primary sources