Trial Outcomes & Findings for PF-04449913 For Patients With Acute Myeloid Leukemia at High Risk of Relapse After Donor Stem Cell Transplant (NCT NCT01841333)

NCT ID: NCT01841333

Last Updated: 2022-01-25

Results Overview

Days after transplant until disease relapse or death as measured by Kaplan-Meier statistical method.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 31 participants
• Primary outcome timeframe: 1 year
• Results posted on: 2022-01-25

Participant Flow

Location: two tertiary care referral centers Dates of recruitment were April 2013 to May 2019

Participant milestones

Measure	PF-04449913 Beginning 80 days after allogeneic stem cell transplant, patients receive PF-04449913 (100mg) orally once daily on days 1-28. Treatment repeats every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity. PF-04449913: 100mg given orally
Overall Study STARTED	31
Overall Study COMPLETED	31
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

PF-04449913 For Patients With Acute Myeloid Leukemia at High Risk of Relapse After Donor Stem Cell Transplant

Baseline characteristics by cohort

Measure	High Risk for Relapse Postallogeneic Stem Cell Transplant n=31 Participants AML and MDS patients at high risk for postallogeneic stem cell transplant relapse
Age, Continuous	58 years n=93 Participants
Sex: Female, Male Female	17 Participants n=93 Participants
Sex: Female, Male Male	14 Participants n=93 Participants
Race/Ethnicity, Customized Race : White	29 Participants n=93 Participants
Race/Ethnicity, Customized Race : Black or African American	1 Participants n=93 Participants
Race/Ethnicity, Customized Race : American Indian or Alaska Native	1 Participants n=93 Participants

PRIMARY outcome

Timeframe: 1 year

Days after transplant until disease relapse or death as measured by Kaplan-Meier statistical method.

Outcome measures

Measure	PF-04449913 n=31 Participants Beginning 80 days after allogeneic stem cell transplant, patients receive PF-04449913 (100mg) orally once daily on days 1-28. Treatment repeats every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity. PF-04449913: 100mg given orally
Relapse-free Survival in Days	142 days Interval 28.0 to 336.0

SECONDARY outcome

Timeframe: Up to 5 years

Length of time before remission measured in days

Outcome measures

Measure	PF-04449913 n=31 Participants Beginning 80 days after allogeneic stem cell transplant, patients receive PF-04449913 (100mg) orally once daily on days 1-28. Treatment repeats every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity. PF-04449913: 100mg given orally
Remission Duration	333 days Interval 87.0 to 787.0

SECONDARY outcome

Timeframe: 30 days

Subjects will be evaluated for AEs at each visit with the NCI-CTCAE version 4.03 used as a guide for the grading of severity.

Outcome measures

Measure	PF-04449913 n=31 Participants Beginning 80 days after allogeneic stem cell transplant, patients receive PF-04449913 (100mg) orally once daily on days 1-28. Treatment repeats every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity. PF-04449913: 100mg given orally
Number of Patients With Adverse Events (AE) Related to Glasdegib	28 Participants

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Measure	PF-04449913 n=31 Participants Beginning 80 days after allogeneic stem cell transplant, patients receive PF-04449913 (100mg) orally once daily on days 1-28. Treatment repeats every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity. PF-04449913: 100mg given orally
Overall Survival of All Patients	64.5 percentage of participants

Adverse Events

PF-04449913

Serious events: 2 serious events

Other events: 26 other events

Deaths: 19 deaths

Serious adverse events

Measure	PF-04449913 n=31 participants at risk Beginning 80 days after allogeneic stem cell transplant, patients receive PF-04449913 (100mg) orally once daily on days 1-28. Treatment repeats every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity. PF-04449913: 100mg given orally
Gastrointestinal disorders Worsening nausea, appetite changes	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Gastrointestinal disorders nausea	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib

Other adverse events

Measure	PF-04449913 n=31 participants at risk Beginning 80 days after allogeneic stem cell transplant, patients receive PF-04449913 (100mg) orally once daily on days 1-28. Treatment repeats every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity. PF-04449913: 100mg given orally
Musculoskeletal and connective tissue disorders Cramping/Myalgias	25.8% 8/31 • AE's were collected over the 1 year subjects were on glasdegib
Gastrointestinal disorders Dysgeusia	12.9% 4/31 • AE's were collected over the 1 year subjects were on glasdegib
Metabolism and nutrition disorders Anorexia/Weight Loss	9.7% 3/31 • AE's were collected over the 1 year subjects were on glasdegib
Gastrointestinal disorders Nausea	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Gastrointestinal disorders Diarrhea	9.7% 3/31 • AE's were collected over the 1 year subjects were on glasdegib
Blood and lymphatic system disorders Thrombocytopenia	6.5% 2/31 • AE's were collected over the 1 year subjects were on glasdegib
Skin and subcutaneous tissue disorders Alopecia	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Musculoskeletal and connective tissue disorders arthralgia	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Blood and lymphatic system disorders lymphopenia	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Blood and lymphatic system disorders neutropenia	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Metabolism and nutrition disorders hypophosphatemia	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Metabolism and nutrition disorders hypocalcemia	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Metabolism and nutrition disorders hypoalbuminemia	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Hepatobiliary disorders hyperbilirubinemia	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Metabolism and nutrition disorders hypertriglyceridemia	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Hepatobiliary disorders LFT Increase	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib
Metabolism and nutrition disorders hyponatremia	3.2% 1/31 • AE's were collected over the 1 year subjects were on glasdegib

Additional Information

Dr. Daniel Pollyea

University of Colorado

Phone: 720-848-8084

Email: DANIEL.POLLYEA@cuanschutz.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place