The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.

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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02177812




Registration number
NCT02177812
Ethics application status
Date submitted
26/06/2014
Date registered
30/06/2014

Titles & IDs
Public title
A Phase I Dose Escalation Study of GSK2879552 in Subjects With Acute Myeloid Leukemia (AML)
Scientific title
A Phase I Open-label, Dose Escalation Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of GSK2879552 Given Orally in Subjects With Relapsed/Refractory Acute Myeloid Leukemia
Secondary ID [1] 0 0
200200
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Leukaemia, Myelocytic, Acute 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GSK2879552
Treatment: Drugs - ATRA

Experimental: Dose Escalation Phase (Part 1) - The safety and PK/PD data will be reviewed prior to the dose decision, and the dose escalation will be guided by the Neuenschwander -continuous reassessment method (N-CRM).The dose escalation will complete when RP2D is determined. The RP2D will be the MTD or a lower dose that provides adequate PK exposure and biologic activity with superior tolerability.

Experimental: Expansion Phase (Part 2) - Once the MTD and/or RP2D has been determined in Part 1, an expansion cohort of up to 30 subjects will be enrolled in order to characterize the clinical activity and safety profile of the RP2D. Subjects may continue treatment in the study until disease progression, unacceptable toxicity, or withdrawal of consent.


Treatment: Drugs: GSK2879552
GSK2879552 capsules contain 0.25 mg, 0.5 mg, 2 mg or 5 mg of GSK2879552 as parent. The initial dosing regimen will be continuous oral daily dosing.

Treatment: Drugs: ATRA
ATRA (Tretinoin) will be supplied as a 10 mg capsule for oral administration. The initial dosing regimen will be continuous oral twice daily dosing

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part 1: Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
Timepoint [1] 0 0
Median of 4 weeks of drug exposure
Primary outcome [2] 0 0
Part 1: Number of Participants With Dose Limiting Toxicities (DLT)
Timepoint [2] 0 0
Median of 4 weeks of drug exposure
Primary outcome [3] 0 0
Part 1: Number of Participants With AE Leading to Dose Reductions or Delays
Timepoint [3] 0 0
Median of 4 weeks of drug exposure
Primary outcome [4] 0 0
Part 1: Number of Participants With Withdrawals Due to Toxicities
Timepoint [4] 0 0
Median of 4 weeks of drug exposure
Primary outcome [5] 0 0
Number of Participants With Clinical Chemistry Parameters Changes From Baseline With Respect to the Normal Range
Timepoint [5] 0 0
Median of 4 weeks of drug exposure
Primary outcome [6] 0 0
Number of Participants With Clinical Chemistry Toxicity Grade Changes From Baseline
Timepoint [6] 0 0
Median of 4 weeks of drug exposure
Primary outcome [7] 0 0
Number of Participants With Hematology Parameters Change From Baseline With Respect to the Normal Range
Timepoint [7] 0 0
Median of 4 weeks of drug exposure
Primary outcome [8] 0 0
Number of Participants With Hematology Toxicity Grade Changes From Baseline
Timepoint [8] 0 0
Median of 4 weeks of drug exposure
Primary outcome [9] 0 0
Part 1: Number of Participants With Change From Baseline in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)
Timepoint [9] 0 0
Median of 4 weeks of drug exposure
Primary outcome [10] 0 0
Part 1: Number of Participants With Change From Baseline in Heart Rate
Timepoint [10] 0 0
Median of 4 weeks of drug exposure
Primary outcome [11] 0 0
Part 1: Number of Participants With Change From Baseline in Temperature
Timepoint [11] 0 0
Median of 4 weeks of drug exposure
Primary outcome [12] 0 0
Part 1: Number of Participants With Change From Baseline in Respiratory Rate
Timepoint [12] 0 0
Median of 4 weeks of drug exposure
Primary outcome [13] 0 0
Part 1: Number of Participants With Abnormal Electrocardiograms (ECGs) Findings
Timepoint [13] 0 0
Median of 4 weeks of drug exposure
Primary outcome [14] 0 0
Part 1: Number of Participants With Abnormal Physical Examinations
Timepoint [14] 0 0
Median of 4 weeks of drug exposure
Primary outcome [15] 0 0
Part 2: Objective Response Rate of Participants
Timepoint [15] 0 0
Up to 14 months
Secondary outcome [1] 0 0
Part 1: Area Under the Plasma Concentration-time Curve From Time Zero (Pre-dose) to Last Time of Quantifiable Concentration [AUC(0-t)] and From Time Zero (Pre-dose) Extrapolated to Infinite Time [(AUC(0-inf)] After Single Dose Administration
Timepoint [1] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 1
Secondary outcome [2] 0 0
Part 1: AUC Over the Dosing Interval (0-tau) After Single Dose Administration
Timepoint [2] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 1
Secondary outcome [3] 0 0
Part 1: Area Under the Concentration-time Curve From Time Zero (Pre-dose) to Last Time of Quantifiable Concentration [AUC(0-t)] and AUC Over the Dosing Interval (0-tau) After Repeated Administration
Timepoint [3] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 15
Secondary outcome [4] 0 0
Part 1: Maximum Observed Plasma Concentration (Cmax) of GSK2879552
Timepoint [4] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 1 and 15
Secondary outcome [5] 0 0
Part 1: Apparent Terminal Phase Half-life (t½)
Timepoint [5] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 1 and 15
Secondary outcome [6] 0 0
Part 1: Time of Occurrence of Cmax (Tmax) of GSK2879552
Timepoint [6] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 1 and 15
Secondary outcome [7] 0 0
Part 1: Accumulation Ratio for GSK2879552
Timepoint [7] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 1 and 15
Secondary outcome [8] 0 0
Part 1:Time Invariance
Timepoint [8] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 1 and 15
Secondary outcome [9] 0 0
Part 1:Percentage of Participants With Objective Response
Timepoint [9] 0 0
Median of 4 weeks drug response
Secondary outcome [10] 0 0
Part 1: AUC(0-t), AUC (0-tau) and AUC(0-inf) of ATRA
Timepoint [10] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 1and Day 15
Secondary outcome [11] 0 0
Part 1: Cmax of ATRA
Timepoint [11] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 1 and 15
Secondary outcome [12] 0 0
Part 1: Apparent Terminal Phase Half-life (t½) of ATRA
Timepoint [12] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 1 and 15
Secondary outcome [13] 0 0
Part 1: Time of Occurrence of Cmax (Tmax) of ATRA
Timepoint [13] 0 0
Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 24 hours post dose on Day 1 and 15
Secondary outcome [14] 0 0
Part 2: Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
Timepoint [14] 0 0
Up to 14 months
Secondary outcome [15] 0 0
Part 2: Number of Participants With AE Leading to Dose Reductions or Delays
Timepoint [15] 0 0
Up to 14 months
Secondary outcome [16] 0 0
Part 2: Number of Participants With Withdrawals Due to Toxicities
Timepoint [16] 0 0
Up to 14 months
Secondary outcome [17] 0 0
Part 2: Number of Participants With Abnormal Clinical Chemistry Parameters
Timepoint [17] 0 0
Up to 14 months
Secondary outcome [18] 0 0
Part 2: Number of Participants With Abnormal Hematology Parameters
Timepoint [18] 0 0
Up to 14 months
Secondary outcome [19] 0 0
Part 2: Number of Participants With Abnormal Vital Signs
Timepoint [19] 0 0
Up to 14 months
Secondary outcome [20] 0 0
Part 2: Number of Participants With Abnormal Electrocardiogram (ECG) Findings
Timepoint [20] 0 0
Up to 14 months
Secondary outcome [21] 0 0
Part 2: Number of Participants With Abnormal Physical Examinations
Timepoint [21] 0 0
Up to 14 months
Secondary outcome [22] 0 0
Part 2: Clearance (CL) of GSK2879552 for Part 2
Timepoint [22] 0 0
Day 1 (pre-dose, 0.5 and 3 hours post dose), Days 4, 8 (pre-dose), Day 15 (pre-dose, 0.5, 1, 4, 6 hours), Weeks 4, 5, 6, 7, 8 and every 4 weeks up to Week 48
Secondary outcome [23] 0 0
Part 2: Volume of Distribution of GSK2879552 for Part 2
Timepoint [23] 0 0
Day 1 (pre-dose, 0.5 and 3 hours post dose), Days 4, 8 (pre-dose), Day 15 (pre-dose, 0.5, 1, 4, 6 hours), Weeks 4, 5, 6, 7, 8 and every 4 weeks up to Week 48
Secondary outcome [24] 0 0
Number of Participants With Abnormal Covariates: Part 2
Timepoint [24] 0 0
Up to 14 months
Secondary outcome [25] 0 0
Duration of Response: Part 2
Timepoint [25] 0 0
Up to 14 months
Secondary outcome [26] 0 0
Time to Time to Response: Part 2
Timepoint [26] 0 0
Up to 14 months
Secondary outcome [27] 0 0
Progression-free Survival: Part 2
Timepoint [27] 0 0
Up to 14 months

Eligibility
Key inclusion criteria
* Subjects >=18 years of age and provided signed written informed consent.
* Subjects must have relapsed/refractory AML by world health organization (WHO) classification for which no standard therapies are available or anticipated to result in a durable remission. French- American- British system (FAB) subtype M3 will be excluded.
* Subjects >= 60 years of age with AML who are not candidates for or have refused standard chemotherapy.
* Subjects who have previously received an autologous stem cell transplant are allowed if a minimum of 3 months has elapsed from the time of transplant and the subject has recovered from transplant-associated toxicities prior to the first dose of GSK2879552.
* Subjects with a history of allogeneic stem cell transplant are eligible for study participation provided the following eligibility criteria are met: transplant was >60 days prior to study enrolment; subject has not taken immunosuppressive medications for at least 1 month; no signs or symptoms of graft versus host disease other than Grade 1 skin involvement; no active infection.
* Eastern Cooperative Oncology Group (ECOG) performance status of 0-1.
* Subjects must be stable and, in the opinion of the investigator, be expected to complete 4 week treatment period.
* Able to swallow and retain orally administered medication and does not have any clinically significant gastrointestinal abnormalities that may alter absorption such as malabsorption syndrome or major resection of the stomach or bowels.
* All prior treatment-related toxicities must be National Cancer Institute- Common Toxicity Criteria for Adverse Events (NCI-CTCAE), version 4.0 <=Grade 1 at the time of enrollment (except for alopecia).
* Adequate baseline organ function.
* Women of childbearing potential must have a negative serum pregnancy test within 7 days of first dose of study treatment and agree to use effective contraception, during the study and for 7 days (GSK2879552 mono therapy) or 30 days (combination with ATRA), following the last dose of study treatment.
* Men with a female partner of childbearing potential must have either had a prior vasectomy or agree to use effective contraception from the administration of the first dose of study treatment until 3 months after the last dose of study treatment to allow for clearance of any altered sperm.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Active human immunodeficiency virus (HIV), Hepatitis B Virus (HBV) or hepatitis C virus (HCV) infections at the time of screening. Subjects with laboratory evidence of HCV clearance may be enrolled.
* History of or concurrent malignancy of solid tumours, except: subjects who have been disease-free for 5 years, or subjects with a history of completely resected non-melanoma skin cancer or successfully treated in situ carcinoma are eligible. Subjects with second malignancies that are indolent or definitively treated may be enrolled even if less than 5 years have elapsed since treatment. Consult GlaxoSmithKline (GSK) Medical Monitor if unsure whether second malignancies meet requirements specified above.
* Currently receiving cancer therapy. Hydroxyurea will be allowed.
* Received major surgery, radiotherapy, or immunotherapy within 4 weeks of GSK2879552 administration.
* Prior treatment with temozolomide, dacarbazine or procarbazine
* Prior treatment with poly ADP ribose polymerase (PARP) inhibitors (eg., olaparib, ABT-888)
* Baseline Montreal Cognitive Assessment (MOCA) score of 22 or lower
* Evidence of severe or uncontrolled systemic diseases. Any serious and/or unstable pre-existing medical, psychiatric disorder, or other conditions that could interfere with subject's safety, obtaining informed consent or compliance to the study procedures, in the opinion of the Investigator
* Current active liver or biliary disease.
* Patients at risk of non-AML related major bleeding (e.g. recent gastrointestinal [GI] hemorrhage or neurosurgery).
* Symptomatic or untreated central nervous system (CNS) leukemia. Subjects are permitted to enroll if previously treated for CNS disease, free of symptoms at the time of screening, and have not required intrathecal chemotherapy at least 1 month prior to study Day 1.
* Cardiac abnormalities
* Administration of an investigational drug within 14 days or 5 half-lives, whichever is shorter with a minimum of 14 days preceding the first dose of study treatment(s) in this study.
* Have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to GSK2879552 or LSD1 inhibitors that contraindicates their participation.
* Lactating female.
* Consumption of Seville oranges, grapefruit, grapefruit hybrids, grapefruit juice, pommelos, or exotic citrus fruits, from 1 day prior to the first dose of study treatment(s) until the last dose of study drug.
* Current use of a prohibited medication including anticoagulants or platelet inhibitors or expected to require any of these medications during treatment with the investigational drug.
* Previous treatment with GSK2879552 For ATRA Combination arm ONLY
* Known hypersensitivity to ATRA, parabens (preservatives in the gelatin capsule) or other retinoids.
* ATRA capsule contains sorbitol. Subjects with rare hereditary problems of fructose intolerance are excluded.
* History of seizure within 12 months or brain tumor (primary)
* History of taking mega-dose vitamin A (>25,000 USP U/day) within 3 months from the dosing start.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
GSK Investigational Site - Melbourne
Recruitment postcode(s) [1] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia
Country [2] 0 0
United States of America
State/province [2] 0 0
New York
Country [3] 0 0
United States of America
State/province [3] 0 0
Texas
Country [4] 0 0
Canada
State/province [4] 0 0
Ontario

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
GlaxoSmithKline
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
GSK Clinical Trials
Address 0 0
GlaxoSmithKline
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
IPD for this study will be made available via the Clinical Study Data Request site.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
When will data be available (start and end dates)?
IPD will be made available within 6 months of publishing the results of the primary endpoints of the study.
Available to whom?
Access is provided after a research proposal is submitted and has received approval from the Independent Review Panel and after a Data Sharing Agreement is in place. Access is provided for an initial period of 12 months but an extension can be granted, when justified, for up to another 12 months.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: http://clinicalstudydatarequest.com


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.