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GSK2330811

Phase 2

Scleroderma, Systemic | Small molecule | Immunology |GSK plc|Last Updated: May 24, 2021

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials2
Total Enrollment76

FDA Designations

No designations recorded

Clinical trial landscape

GSK2330811 · 3 trials · 2 indications

Phase 2 1Phase 1 2
NCT03041025Proof of Mechanism Study of GSK2330811 in Diffuse Cutaneous Systemic SclerosisScleroderma, Systemic
COMPLETED35 Analytics
PHASE2COMPLETED
Proof of Mechanism Study of GSK2330811 in Diffuse Cutaneous Systemic Sclerosis
Scleroderma, SystemicUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Non-serious Adverse Events (Non-SAEs) and Serious Adverse Events (SAEs)
Up to Day 197, but protocol allowed for additional events to be collected; up to Day 603 post first dose

An adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study treatment, whether or not considered related to the study treatment. An SAE is defined as any untoward medical occurrence that: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect, other situations judged by physician. SAEs were collected up to Day 197, but the protocol also allowed investigators to report SAEs occurring after participants had completed the study. This outcome measure includes two SAEs reported after participants had completed the study, occurring on Day 306 and Day 603 following first dose. Safety Population consisted of all randomized participants who have taken at least 1 dose of study treatment.

Change From Baseline in Hematology Parameters: Basophils, Eosinophils, Lymphocytes, Monocytes, Total Neutrophils, Platelet Count, White Blood Cell (WBC) Count
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze the hematology parameters: Basophils, Eosinophils, Lymphocytes, Monocytes, Total Neutrophils, Platelet Count and WBC count. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Change From Baseline in Hematology Parameters: Hemoglobin, Mean Corpuscle Hemoglobin Concentration (MCHC)
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze the hematology parameters: hemoglobin and MCHC. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Change From Baseline in Hematology Parameter: Hematocrit
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze the hematology parameter: hematocrit. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Change From Baseline in Hematology Parameter: Mean Corpuscle Hemoglobin (MCH)
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze the hematology parameter: MCH. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Change From Baseline in Hematology Parameters: Mean Corpuscle Volume (MCV), Mean Platelet Volume (MPV)
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze the hematology parameters: MCV and MPV. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Change From Baseline in Hematology Parameters: Red Blood Cell (RBC) Count, Reticulocyte Count
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze the hematology parameters: RBC count and reticulocyte count. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Change From Baseline in Hematology Parameter: Red Cell Distribution Width (RDW)
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze the hematology parameter: RDW. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Change From Baseline in Reticulocyte Production Index
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze the hematology parameter: Reticulocyte Production Index. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value. Reticulocyte Production Index (RPI) was calculated as 'Reticulocyte Production Index = Reticulocyte Count (percent \[%\]) multiply by (x) (hematocrit \[%\] divided by \[/\] 45) x 1/ reticulocyte maturation time'.

Change From Baseline Hematology Parameter: Reticulocytes
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze the hematology parameter: reticulocytes. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Number of Participants With Worst-Case Chemistry Results Relative to Potential Clinical Importance (PCI) Criteria Post-Baseline Relative to Baseline
Up to Day 197

Blood samples were collected for analysis of clinical chemistry parameters. PCI ranges were low: \<30 grams per liter (g/L) (albumin), high: \>44.2 micromoles per liter (µmol/L) increase from Baseline (creatinine), low: \<3 or high: \>9 mmol/L (glucose), low: \<3 or high: \>5.5 mmol/L (potassium), and low: \<130 or high: \>150 mmol/L (sodium). Participants were counted in the worst case category that their value changes to (low, within range or no change, or high), unless there was no change in their category. Participants whose laboratory value category was unchanged (e.g. High to High), or whose value became within range, were recorded in the "To within Range or No Change" category. Participants were counted twice if the participant had values that changed 'To Low' and 'To High', so the percentages may not add up to 100%.

Change From Baseline in Chemistry Parameter: Total Protein
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze chemistry parameter: total protein. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Change From Baseline in Chemistry Parameters: Alkaline Phosphatase (ALP), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Lactate Dehydrogenase (LDH)
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze chemistry parameters: ALP, ALT, AST and LDH. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Change From Baseline in Chemistry Parameters: Total Bilirubin, Direct Bilirubin
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze chemistry parameters: total bilirubin and direct bilirubin. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Change From Baseline in Chemistry Parameters: Cholesterol, Direct High-density Lipoprotein (HDL) Cholesterol, Low-density Lipoprotein (LDL) Cholesterol, Triglycerides
Baseline (Day 1: Pre-dose) and Day 85

Blood samples were collected to analyze chemistry parameters: cholesterol, direct HDL cholesterol, LDL cholesterol and triglycerides. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from post-dose visit value.

Change From Baseline in Chemistry Parameter: Corrected Calcium, Urea
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze chemistry parameters: corrected calcium and urea. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Change From Baseline in Chemistry Parameter: Estimated Glomerular Filtration Rate
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Blood samples were collected to analyze chemistry parameter: estimated glomerular filtration rate. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Number of Participants With Emergent Worst Case Urinalysis Results by Dipstick
Up to Day 197

Urine samples were collected for the assessment of potential of hydrogen, specific gravity, glucose, ketones, occult blood and protein by dipstick method. The dipstick test gave results in a semi-quantitative manner, and results for urinalysis parameters: potential of hydrogen, specific gravity, glucose, ketones, occult blood and protein were categorized as 'any increase from Baseline', which imply any increase in their concentrations in the urine sample. Only participants with emergent worst case any increase from Baseline values are presented.

Number of Participants With Vital Signs Relative to Change From Baseline by Potential Clinical Importance (PCI) Criteria
Baseline (Day 1: Pre-dose) and up to Day 197

Systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR) were measured in a seated or semi-supine position after 5 minutes of rest using a completely automated device. PCI ranges were: SBP (increase or decrease from Baseline of \>=40 millimeter of mercury \[mmHg\]), DBP (increase or decrease from Baseline of \>=20 mmHg), and HR (increase or decrease from Baseline of \>=30 beats per minute). Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment.

Change From Baseline in Body Temperature
Baseline (Day 1: Pre-dose), Days 15, 29, 43, 57, 71, 85, 113, 155 and 197

Body temperature was measured in a seated or semi-supine position after 5 minutes of rest. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Change from Baseline was calculated by subtracting the Baseline value from the post-dose visit value.

Number of Participants With Worst-case Post-Baseline Abnormal Electrocardiogram (ECG) Findings
Up to Day 57

Twelve lead ECGs were obtained using an ECG machine that automatically calculated the heart rate and measured PR, QRS, QT, and corrected QT intervals. Abnormal findings were categorized as clinically significant and not clinically significant. Clinically significant abnormal findings were those which were not associated with the underlying disease, unless judged by the investigator to be more severe than expected for the participant's condition. Data for number of participants with worst case post-Baseline abnormal ECG findings have been presented.

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Up to Day 126

An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. A SAE is defined as any untoward medical occurrence that, at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, congenital anomaly/birth defect, any other situation according to medical or scientific judgment. Safety Population consisted of all randomized participants who received at least one dose of study treatment.

Number of Participants With Worst Case Vital Sign Results Relative to Potential Clinical Importance (PCI) Criteria Post-Baseline Relative to Baseline
Baseline (Pre-dose, Day 1) and up to Day 126

Vital signs were measured in semi-supine position after 5 minutes of rest and included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR). Participants were counted in the worst case category that their value changed to low, within range or high, unless there was no change in their category. Participants whose value category was unchanged (e.g., High to High), or whose value became within range, were recorded in the "Within Range or No Change" category. Participants were counted twice if values changed 'To Low' and 'To High', so the percentages were not added up to 100 percent (%). Participants with missing Baseline values were assumed as within range value. PCI ranges were: SBP (lower: \<85, upper: \>160 millimeter of mercury \[mmHg\]); DBP (lower: \<45, upper: \>100 mmHg); HR (lower: \<40, upper: \>110 beats per minute). Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment.

Number of Participants With Worst Case Abnormal Electrocardiogram (ECG) Findings
Up to Day 126

12-lead ECGs were recorded in semi-supine position using an ECG machine. Number of participants with worst-case clinically significant and not clinically significant abnormal ECG findings have been presented. Clinically significant abnormal laboratory findings were those which were not associated with the underlying disease, unless judged by the investigator to be more severe than expected for the participant's condition.

Number of Participants With Maximum Common Terminology Criteria for Adverse Events (CTCAE) Grade 1 or Higher Clinical Chemistry Parameters
Up to Day 126

Blood samples were collected for the analysis of clinical chemistry parameters. Clinical chemistry parameters were summarized according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), version 5.0: Grade 1: mild; Grade 2: moderate; Grade 3: severe; Grade 4: life-threatening or disabling. Higher grade indicates more severity. Clinical chemistry parameters included: total bilirubin, calcium, creatinine and triglycerides.

Number of Participants With Grade Increase Post-Baseline Relative to Baseline in Hematology Parameters
Baseline (Pre-dose, Day 1) and up to Day 126

Blood samples were collected for the analysis of the following hematology parameters: hemoglobin (Hb), lymphocytes (Lympho), platelet count (PC), neutrophil count (Neutro) and White Blood Cell count (WBC). The laboratory parameters were graded according to NCI-CTCAE version 5.0. Grade 1: mild; Grade 2: moderate; Grade 3: severe or medically significant; Grade 4: life-threatening consequences. Higher grade indicates more severity. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. An increase was defined as an increase in CTCAE Grade relative to Baseline Grade.

Number of Participants With Worst Case Any Increase in Urinalysis Results Post-Baseline Relative to Baseline
Baseline (Pre-dose, Day 1) and up to Day 126

Urine samples were collected for analysis of blood, glucose, ketones and protein by a dipstick method. The dipstick test gives results in a semi-quantitative manner, and results for urinalysis parameters of urine glucose, protein, blood and ketones can be read as negative (-), trace, 1+, 2+, 3+ indicating proportional concentrations in the urine sample. Baseline was defined as the pre-dose Day 1 assessment, unless unavailable, in which case it was the latest pre-dose assessment. Any increase means any increase to trace, 1+, 2+ or 3+ post-Baseline relative to Baseline. Number of participants with worst case any increase in urinalysis results post-Baseline relative to Baseline has been presented.

Safety and tolerability as assessed by number of subjects with adverse event (AEs) and serious adverse events (SAEs)
From Day 1 up to Day 105 (For Cohort 5 up to Day 133)
Safety and tolerability as assessed by composite of clinical laboratory tests: clinical chemistry, hematology and urinalysis
From Day 1 up to Day 105(For Cohort 5 up to Day 133)

Clinical laboratory tests will include clinical chemistry, hematology and urinalysis.

Safety and tolerability as assessed by composite of vital signs assessment: blood pressure, heart rate and temperature
From Day 1 up to Day 105 (For Cohort 5 up to Day 133)

Vital signs assessment including systolic and diastolic blood pressure, heart rate and body temperature.

Safety as assessed by electrocardiogram (ECG) measurements
From Day 1 up to Day 105

ECGs will be measured in the semi-supine position. Triplicate 12-lead ECGs will be recorded at screening, Day 1 pre-dose and Day 5 and single 12-lead ECGs at all other time points.

Secondary Endpoints

Plasma Concentrations of GSK2330811
Days 1, 15, 29, 57, 85, 113, 155 and 197
Concentration at the End of the Dosing Interval (Ctrough) of GSK2330811
Days 1, 15, 29, 57, 85, 113, 155 and 197
Apparent Clearance (CL/F) of GSK2330811
Days 1, 15, 29, 57, 85, 113, 155 and 197
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1: GSK2330811 100 mgEXPERIMENTALDuring Cohort 1, participants will receive a single dose of GSK2330811 100 mg by SC injection via needle and syringe in to the abdomen, thigh or upper arm by the investigator or designee at every other week from D1 to D71 (total 6 doses) for 10 weeks. Participants will be followed-up up to W28 D197.
Cohort 2: GSK2330811 300 mgEXPERIMENTALDuring Cohort 2, participants will receive a single dose of GSK2330811 300 mg by SC injection (3 vials of 1 mL each of GSK2330811 100 mg) via needle and syringe in to the abdomen, thigh or upper arm by the investigator or designee at every other week from D1 to D71 (total 6 doses) for 10 weeks. Participants will be followed-up up to W28 D197.
Cohort 1: PlaceboPLACEBO_COMPARATORDuring Cohort 1, participants will receive a single dose of placebo by SC injection via needle and syringe in to the abdomen, thigh or upper arm by the investigator or designee at every other week from D1 to D71 (total 6 doses) for 10 weeks. Participants will be followed-up up to W28 D197.
Cohort 2: PlaceboPLACEBO_COMPARATORDuring Cohort 2, participants will receive a single dose of placebo by SC injection (3 vials of 1 mL each) via needle and syringe in to the abdomen, thigh or upper arm by the investigator or designee at every other week from D1 to D71 (total 6 doses) for 10 weeks. Participants will be followed-up up to W28 D197.
GSK2330811 450 mgEXPERIMENTALParticipants will receive a single 450 mg SC dose of GSK2330811, administered as three separate SC injections of 150 milligrams per milliliter \[mg/mL\]).
PlaceboPLACEBO_COMPARATORParticipants will receive GSK2330811 matching placebo administered as three separate SC injections.
Cohort 1- GSK2330811(0.1 mg/kg)EXPERIMENTALSubjects will be randomised to receive either 0.1 mg/kg GSK2330811 or placebo s.c single dose in a 3:1 ratio.
Cohort 2- GSK2330811(0.3 mg/kg)EXPERIMENTALSubjects will be randomised to receive either 0.3 mg/kg GSK2330811 or placebo s.c single dose in a 3:1 ratio.
Cohort 3- GSK2330811(1 mg/kg)EXPERIMENTALSubjects will be randomised to receive either 1 mg/kg GSK2330811 or placebo s.c single dose in a 3:1 ratio.
Cohort 4- GSK2330811(3 mg/kg)EXPERIMENTALSubjects will be randomised to receive either 3 mg/kg GSK2330811 or placebo s.c single dose in a 3:1 ratio.
Cohort 5- GSK2330811(6 mg/kg)EXPERIMENTALSubjects will be randomised to receive either 6 mg/kg GSK2330811 or placebo s.c single dose in a 3:1 ratio.

Interventions

NameTypeDescription
GSK2330811DRUGGSK2330811 will be provided in vials and packed in 1 vial per carton. Each vial will contain 1.2 mL fill with 1 mL extractable volume at 100 mg/mL.
PlaceboDRUGNormal saline (0.9 percent weight per volume sodium chloride).
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites14

Inclusion Criteria: * Participants of 18 years or over, at the time of signing the informed consent. * Documented diagnosis of systemic sclerosis with diffuse cutaneous involvement. * Modified rodnan skin score (mRSS) \>=10 and \<=35 at screening. * In all cases, a disease duration of \<=60 months ...

Countries:United StatesCanadaNetherlandsUnited Kingdom
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Frequently asked questions about GSK2330811

What is GSK2330811 used for?

GSK2330811 is an investigational small molecule being studied for scleroderma, specifically diffuse cutaneous systemic sclerosis, and in healthy volunteers. It is being developed by GSK plc (GSK). The drug is in clinical development and is not approved by regulatory authorities.

Who makes GSK2330811?

GSK2330811 is being developed by GSK plc, which trades under the ticker GSK. The company is conducting clinical trials to evaluate the drug's safety, tolerability, and mechanism of action in scleroderma and healthy volunteer populations.

What phase is GSK2330811 in?

GSK2330811 has completed Phase 1 and Phase 2 clinical trials. The Phase 2 study was a proof of mechanism trial in diffuse cutaneous systemic sclerosis. All trials are completed, and the drug remains investigational, with no regulatory approval granted.

What clinical trials is GSK2330811 in?

GSK2330811 has been studied in three completed trials: NCT02386436, a Phase 1 study in healthy subjects in the United Kingdom; NCT03041025, a Phase 2 proof of mechanism study in diffuse cutaneous systemic sclerosis across the US, Canada, Netherlands, and UK; and NCT04138043, a Phase 1 study in healthy Japanese male participants in the UK.

Is GSK2330811 the same as any other drug?

No alternative names for GSK2330811 have been reported. The drug is identified solely by its investigational code GSK2330811 in clinical trial registries and related documentation.