Evaluation of a New Anti-cancer Immunotherapy in Adult Acute Myeloid Leukemia Patients With a Suboptimal Clinical Response to Induction Chemotherapy
CompletedPhase 1Results postedLast updated 29 November 2018
What this trial tests
Phase 1 trial testing GSK Biologicals' recombinant WT1 Antigen-Specific Cancer Immunotherapeutic (ASCI) GSK2130579A in Leukaemia, Myelocytic, Acute in 17 participants. Completed in 26 April 2016.
18 and older, any sex, with Leukaemia, Myelocytic, Acute. 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 Patients With Severe ToxicitiesPrimary· During the entire study (from Month 0 to Month 49)
Severe toxicities (as classified according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0) during the study treatment period defined as a study product-related or possibly study product-related:
* Grade 4 toxicity (exception: study product-related or possibly study product-related Grade 4 fatigue - including lethargy, asthenia, and malaise - had to have a duration of at least 48 hours to be taken into account).
* Grade 3 toxicity lasting for at least 48 hours (exceptions: myalgia, arthralgia, headache, and fever, regardless of duration).
Group
Value
95% CI
Complete Remission Group
1
Partial Remission Group
0
Number of Patients With Best Overall Response, Defined by Either Complete Response (CR), Partial Response (PR), Stable Disease (SD) or Progressive Disease (PD)Primary· During the entire study (from Month 0 to Month 49)
CR = having \< 5% blasts in aspirate sample with marrow spicules and with a count of ≥ 200 nucleated cells, no blasts with Auer rods or persistence of extramedullary disease; different phenotype (by flow cytometry) to the pre-treatment specimen; absolute neutrophil count \> 1000/mm3; platelet count ≥ 100 000/mm3 and being independent of red blood cell (RBC) transfusions.
PR = a decrease of ≤ 50% in the % of blasts in bone marrow aspirate compared to Visit 4; being independent of RBC transfusions and the absolute neutrophil ≥ 1000/mm3 and platelet counts and ≥ 100 000/mm3.
SD = no sufficient
CR
Group
Value
95% CI
Complete Remission Group
5
Partial Remission Group
0
PR
Group
Value
95% CI
Complete Remission Group
0
Partial Remission Group
3
SD
Group
Value
95% CI
Complete Remission Group
6
Partial Remission Group
1
PD
Group
Value
95% CI
Complete Remission Group
1
Partial Remission Group
1
Anti-WT1 Seropositivity RateSecondary· At baseline (PRE), weeks (W) 5, 9, 13, 15, 21, 32, 40, 54, at months 15, 18, 21, 24, 30, 36, 42 and 49 (concluding visit), as well as follow-up visits 1 to 4
Seropositivity rate was defined as the number of patients with anti-WT1 antibody concentrations greater than or equal to (≥) 9 Enzyme-Linked Immunosorbent Assay (ELISA) units per milliliter (EU/mL).
Anti-WT1 PRE
Group
Value
95% CI
Total Treated Group
0
Anti-WT1 Post-dose 2, Week 5
Group
Value
95% CI
Total Treated Group
1
Anti-WT1 Post-dose 4, Week 9
Group
Value
95% CI
Total Treated Group
10
Anti-WT1 Post-dose 6, Week 13
Group
Value
95% CI
Total Treated Group
12
Anti-WT1 Post-dose 6, Week 15
Group
Value
95% CI
Total Treated Group
12
Anti-WT1 Post-dose 8, Week 21
Group
Value
95% CI
Total Treated Group
9
Anti-WT1 Post-dose 12, Week 32
Group
Value
95% CI
Total Treated Group
6
Anti-WT1 Post-dose 13, Week 40
Group
Value
95% CI
Total Treated Group
5
Anti-WT1 Antibody ConcentrationsSecondary· At baseline (PRE), weeks (W) 5, 9, 13, 15, 21, 32, 40, 54, at months 15, 18, 21, 24, 30, 36, 42 and 49 (concluding visit), as well as follow-up visits 1 to 4
Antibody concentrations were expressed as geometric mean concentrations, measured in ELISA units per milliliter (EU/mL).
Anti-WT1 PRE
Group
Value
95% CI
Total Treated Group
4.5
4.5 – 4.5
Anti-WT1 Post-dose 2, Week 5
Group
Value
95% CI
Total Treated Group
5.4
3.7 – 7.9
Anti-WT1 Post-dose 4, Week 9
Group
Value
95% CI
Total Treated Group
52.0
18.6 – 145.4
Anti-WT1 Post-dose 6, Week 13
Group
Value
95% CI
Total Treated Group
123.8
50.0 – 306.5
Anti-WT1 Post-dose 6, Week 15
Group
Value
95% CI
Total Treated Group
138.3
65.3 – 292.8
Anti-WT1 Post-dose 8, Week 21
Group
Value
95% CI
Total Treated Group
105.0
35.6 – 309.7
Anti-WT1 Post-dose 12, Week 32
Group
Value
95% CI
Total Treated Group
136.4
29.4 – 632.2
Anti-WT1 Post-dose 13, Week 40
Group
Value
95% CI
Total Treated Group
113.0
21.1 – 603.4
Anti-WT1 Antibody ResponseSecondary· At baseline (PRE), weeks (W) 5, 9, 13, 15, 21, 32, 40, 54, at months 15, 18, 21, 24, 30, 36, 42 and 49 (concluding visit), as well as follow-up visits 1 to 4
The anti-WT1 antibody response was defined as:
For initially seronegative patients, post-vaccination antibody concentration ≥ 9 EU/mL; For initially seropositive patients, post-vaccination antibody concentration ≥ 2 fold the pre-vaccination antibody concentration.
Anti-WT1 Post-dose 2, Week 5
Group
Value
95% CI
Total Treated Group
1
Anti-WT1 Post-dose 4, Week 9
Group
Value
95% CI
Total Treated Group
10
Anti-WT1 Post-dose 6, Week 13
Group
Value
95% CI
Total Treated Group
12
Anti-WT1 Post-dose 6, Week 15
Group
Value
95% CI
Total Treated Group
12
Anti-WT1 Post-dose 8, Week 21
Group
Value
95% CI
Total Treated Group
9
Anti-WT1 Post-dose 12, Week 32
Group
Value
95% CI
Total Treated Group
6
Anti-WT1 Post-dose 13, Week 40
Group
Value
95% CI
Total Treated Group
5
Anti-WT1 Post-dose 16, Week 54
Group
Value
95% CI
Total Treated Group
5
Number of Subjects With Any and Related Unsolicited Adverse Events (AEs)Secondary· Starting with the first administration of study treatment and ending 30 days after the last study treatment administration
An unsolicited AE covers any untoward medical occurrence in a clinical investigation subject temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product and reported in addition to those solicited during the clinical study and any solicited symptom with onset outside the specified period of follow-up for solicited symptoms. Any was defined as the occurrence of any unsolicited AE regardless of intensity grade or relation to vaccination. Related = AE assessed by the investigator as causally related to the study treatment.
Any AE(s)
Group
Value
95% CI
Total Treated Group
15
Related AE(s)
Group
Value
95% CI
Total Treated Group
10
Number of Subjects With Study Treatment FailureSecondary· During the entire study (from Month 0 to Month 49)
Study treatment failure was defined as withdrawal from investigational product because of disease progression or death. Among the characteristics evaluated were: Progression, Death in absence of Relapse, Relapse or progression or Death (Progression Free Survival event), Death \[An event that was part of the natural course of the disease under study (i.e., disease progression, recurrence) was captured in the study as an efficacy measure; therefore it did not need to be reported as an SAE\], Autopsy performed and Cause of death.
Progression - yes
Group
Value
95% CI
Complete Remission Group
6
Partial Remission Group
5
Progression - no
Group
Value
95% CI
Complete Remission Group
6
Partial Remission Group
0
Death in absence of Relapse - yes
Group
Value
95% CI
Complete Remission Group
0
Partial Remission Group
0
Death in absence of Relapse - no
Group
Value
95% CI
Complete Remission Group
12
Partial Remission Group
5
Progression Free Survival event - yes
Group
Value
95% CI
Complete Remission Group
6
Partial Remission Group
5
Progression Free Survival event - no
Group
Value
95% CI
Complete Remission Group
6
Partial Remission Group
0
Death - yes
Group
Value
95% CI
Complete Remission Group
4
Partial Remission Group
2
Death - no
Group
Value
95% CI
Complete Remission Group
8
Partial Remission Group
3
Number of Subjects With Any or Related Serious Adverse Events (SAEs)Secondary· During the entire study (from Month 0 to Month 49)
SAEs assessed include medical occurrences that result in death, are life-threatening, require hospitalization or prolongation of hospitalization or result in disability/incapacity. Related = SAE assessed by the investigator as causally related to the study treatment. An event that was part of the natural course of the disease under study (i.e., disease progression, recurrence) was captured in the study as an efficacy measure, therefore, it did not need to be reported as an SAE. Death due to progressive disease was recorded on a specific form in the CRF, but not as an SAE.
Any SAE(s)
Group
Value
95% CI
Total Treated Group
7
Related SAE(s)
Group
Value
95% CI
Total Treated Group
1
Number of Patients With Abnormal Hematological and Biochemical ParametersSecondary· During the entire study (Month 0 to Month 49)
Haematological and biochemical parameters assessed were Alanine aminotransferase, Aspartate aminotransferase, Alkaline Phosphatase, Bilirubin, Creatinine, Gamma-glutamyl transpeptidase, Hemoglobin, Hypercalcemia, Hyperkalemia, Hypernatremia, Hypoalbuminemia, Hypocalcemia, Hypokalemia, Hyponatremia, Leukocytes, Lymphopenia, Neutrophils, Platelets and Proteinuria. Parameters were assessed as per the Common Terminology Criteria for adverse events (CTCAE), where Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe but not life-threatening and Grade 4 = life-threatening.
Alanine aminotransferase, Grade 0
Group
Value
95% CI
Total Treated Group
8
Alanine aminotransferase, Grade 1
Group
Value
95% CI
Total Treated Group
7
Alanine aminotransferase, Grade 2
Group
Value
95% CI
Total Treated Group
2
Alanine aminotransferase, Grade 3
Group
Value
95% CI
Total Treated Group
0
Alanine aminotransferase, Grade 4
Group
Value
95% CI
Total Treated Group
0
Alanine aminotransferase, Unknown
Group
Value
95% CI
Total Treated Group
0
Aspartate aminotransferase, Grade 0
Group
Value
95% CI
Total Treated Group
10
Aspartate aminotransferase, Grade 1
Group
Value
95% CI
Total Treated Group
6
Adverse events — posted to ClinicalTrials.gov
Time frame: Unsolicited AEs: starting with the first administration of study treatment (Week 1) and ending 30 days after the last study treatment administration (Month 49); SAEs: During the entire study (From Month 0 to Month 49)..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The purpose of this study is to evaluate the clinical activity and safety of a WT1 Antigen-Specific Cancer Immunotherapeutic (WT1 ASCI) as post-induction therapy in adult patients with WT1-positive AML presenting a suboptimal clinical response to induction chemotherapy. The study will also assess whether this treatment induces a specific immune response to the malignancy.
Publications & conference data
4 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07569081 — A Study Evaluating the Efficacy and Safety of Momelotinib in Participants With Vacuoles, E1-enzyme, X-linked, Autoinflam
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· not yet recruiting
NCT07406347 — A Trial to Evaluate the Safety and Reactogenicity of an Investigational Pneumococcal Vaccine in Infants Receiving 3-dose
· Phase 1
· not yet recruiting
NCT07286266 — A Study to Investigate GSK5733584 Compared With Chemotherapy in Participants With Platinum-resistant Ovarian Cancer (BEH
· Phase 3
· not yet recruiting
NCT07286331 — A Study to Investigate GSK5733584 Compared With Chemotherapy in Participants With Recurrent Endometrial Cancer (BEHOLD-E
· Phase 3
· not yet recruiting
NCT07406334 — A Trial to Evaluate the Safety and Reactogenicity of an Investigational Pneumococcal Vaccine in Toddlers 12 to 15 Months
· Phase 1
· not yet recruiting
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: 29 November 2018
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/NCT01051063.