Number of Participants With Disease-related Gene Mutations at Phase 1B
A Study To Evaluate PF-04449913 With Chemotherapy In Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome · Source last updated 2020-03-03
Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.
Measure
NUMBER
Unit
Participants
Interval / dispersion
Not reported
Time frame
Baseline (Cycle 1/Day 1 pre-dose for Glasdegib + LDAC and Glasdegib + Decitabine Arms; Induction Cycle 1/Day -3 pre-dose for Glasdegib +Cytarabine/Daunorubicin Arm)
What was measured
Peripheral blood and bone marrow aspirate were collected for baseline mutational analyses. Genetic abnormalities frequently associated with AML were analyzed. These genetic abnormalities included known mutations in the genes NPM1, CEBPA, FLT3, RUNX1, IDH1, IDH2, KIT, K Ras, N Ras and WT1. Additional genes with mutations known to be associated with AML and MDS such as TET2 and DNMT3A were also evaluated.
Analysis population: Pharmacodynamic (PD) analysis set: all enrolled participants in the Phase 1B portion who received at least 1 dose of glasdegib; responders and non-responders in each arm with at least 1 PD parameter from the corresponding assay sample with a baseline and an adequate post treatment assessment.
Groups in this outcome
Phase 1B: Glasdegib + LDAC (Biomarker, Responder)
AML participants who achieved CR, CRi, MLFS, PR, or PRi (AML investigator reported best overall response), and MDS participants who achieved CR, mCR, PR, or SD (MDS investigator reported best overall response).
AML subjects who did not achieve CR, CRi, MLFS, PR, or PRi; and MDS subjects who did not achieve CR, mCR, PR or SD were defined as non-responders for best overall response.
AML participants who achieved CR, CRi, MLFS, PR, or PRi (AML investigator reported best overall response), and MDS participants who achieved CR, mCR, PR, or SD (MDS investigator reported best overall response).
AML participants who did not achieve CR, CRi, MLFS, PR, or PRi; and MDS participants who did not achieve CR, mCR, PR or SD were defined as non-responders for best overall response.
AML subjects who achieved CR, CRi, MLFS, PR, or PRi (AML investigator reported best overall response), and MDS subjects who achieved CR, mCR, PR, or SD (MDS investigator reported best overall response). Dauno is short for daunorubicin.
AML participants who did not achieve CR, CRi, MLFS, PR, or PRi; and MDS participants who did not achieve CR, mCR, PR or SD were defined as non-responders for best overall response. Dauno is short for daunorubicin.
Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.
KRAS(Kirsten rat sarcoma 2 viral oncogene homolog)
categories
measurements
group Id
OG001
value
0
group Id
OG003
value
0
group Id
OG004
value
4
group Id
OG005
value
0
title
NPM1 (Nucleophosmin)
categories
measurements
group Id
OG001
value
5
group Id
OG003
value
0
group Id
OG004
value
1
group Id
OG005
value
0
title
NRAS(Neuroblastoma RAS viral oncogene homolog)
categories
measurements
group Id
OG001
value
1
group Id
OG003
value
0
group Id
OG004
value
1
group Id
OG005
value
0
title
RUNX1 (Runt related transcription factor 1)
categories
measurements
group Id
OG001
value
3
group Id
OG003
value
0
group Id
OG004
value
1
group Id
OG005
value
0
title
TET2 (Tet methylcytosine dioxygenase 2)
denoms
counts
group Id
OG000
value
0
group Id
OG001
value
9
group Id
OG002
value
0
group Id
OG003
value
1
group Id
OG004
value
11
group Id
OG005
value
1
units
Participants
description
Peripheral blood and bone marrow aspirate were collected for baseline mutational analyses. Genetic abnormalities frequently associated with AML were analyzed. These genetic abnormalities included known mutations in the genes NPM1, CEBPA, FLT3, RUNX1, IDH1, IDH2, KIT, K Ras, N Ras and WT1. Additional genes with mutations known to be associated with AML and MDS such as TET2 and DNMT3A were also evaluated.
groups
description
AML participants who achieved CR, CRi, MLFS, PR, or PRi (AML investigator reported best overall response), and MDS participants who achieved CR, mCR, PR, or SD (MDS investigator reported best overall response).
id
OG000
title
Phase 1B: Glasdegib + LDAC (Biomarker, Responder)
description
AML subjects who did not achieve CR, CRi, MLFS, PR, or PRi; and MDS subjects who did not achieve CR, mCR, PR or SD were defined as non-responders for best overall response.
AML participants who achieved CR, CRi, MLFS, PR, or PRi (AML investigator reported best overall response), and MDS participants who achieved CR, mCR, PR, or SD (MDS investigator reported best overall response).
AML participants who did not achieve CR, CRi, MLFS, PR, or PRi; and MDS participants who did not achieve CR, mCR, PR or SD were defined as non-responders for best overall response.
AML subjects who achieved CR, CRi, MLFS, PR, or PRi (AML investigator reported best overall response), and MDS subjects who achieved CR, mCR, PR, or SD (MDS investigator reported best overall response). Dauno is short for daunorubicin.
AML participants who did not achieve CR, CRi, MLFS, PR, or PRi; and MDS participants who did not achieve CR, mCR, PR or SD were defined as non-responders for best overall response. Dauno is short for daunorubicin.
Pharmacodynamic (PD) analysis set: all enrolled participants in the Phase 1B portion who received at least 1 dose of glasdegib; responders and non-responders in each arm with at least 1 PD parameter from the corresponding assay sample with a baseline and an adequate post treatment assessment.
reporting Status
POSTED
time Frame
Baseline (Cycle 1/Day 1 pre-dose for Glasdegib + LDAC and Glasdegib + Decitabine Arms; Induction Cycle 1/Day -3 pre-dose for Glasdegib +Cytarabine/Daunorubicin Arm)
title
Number of Participants With Disease-related Gene Mutations at Phase 1B
Triangle