A Study Of PF-04449913 In Japanese Patients With Select Hematologic Malignancies

A PHASE 1 STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF PF-04449913 (GLASDEGIB), AN ORAL HEDGEHOG INHIBITOR, ADMINISTERED AS A SINGLE AGENT IN JAPANESE PATIENTS WITH SELECT HEMATOLOGIC MALIGNANCIES AND IN COMBINATION WITH INTENSIVE CHEMOTHERAPY, LOW-DOSE ARA-C, OR AZACITIDINE IN PATIENTS WITH ACUTE MYELOID LEUKEMIA OR HIGH-RISK MYELODYSPLASTIC SYNDROME

Status

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080222383

Enrollment

Registered

2014-01-31

Start date

2014-03-25

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Untreated AML patients

Interventions

investigational material(s) Generic name etc :PF-04449913 INN of investigational material : - Therapeutic category code : 429 Other antitumor agents Dosage and Administration for Investigational mate

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Inclusion Criteria: *Patients with select advanced hematologic malignancies who are refractory, resistant or intolerant to prior therapies for monotherapy cohort. *Patients with AML or High-Risk MDS who are newly diagnosed and previously untreated for combination cohort. *Patients with AML who are newly diagnosed and previously untreated for azacitidine combination cohort. *ECOG [Eastern Cooperative Oncology Group] performance status 0 to 2 *Adequate organ function

Exclusion criteria

Exclusion criteria: Exclusion Criteria: *Patients with active CNS disease *Patient with active malignancy with the exception of basal cell carcinoma, non melanoma skin cancer, carcinoma in situ cervical *Patient has an active, life threatening or clinically significant uncontrolled systemic infection

Design outcomes

Primary

Measure	Time frame
safety efficacy Monotherapy cohort: Number of Participants With Dose-limiting Toxicities (DLTs) Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Treatment Related TEAEs, Grade 3 or 4 TEAEs Based on NCI CTCAE v4.0 Number of Participants With Clinically Significant Changes From Baseline in Vital Signs Abnormalities Number of Participants With Worst On-study Laboratory Abnormalities Combination cohort 1, 2, and 3: Number of Participants With DLTs Number of Participants With TEAEs, Serious TEAEs, Treatment Related TEAEs, Grade 3 or 4 TEAEs Based on NCI CTCAE v4.0 Number of Participants With Clinically Significant Changes From Baseline in Vital Signs Abnormalities Number of Participants With Worst On-study Laboratory Abnormalities Expansion cohort: Percentage of Participants Achieving Disease Modifying Response (DMR)	—

Measure

Time frame

safety efficacy Monotherapy cohort: Number of Participants With Dose-limiting Toxicities (DLTs) Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Treatment Related TEAEs, Grade 3 or 4 TEAEs Based on NCI CTCAE v4.0 Number of Participants With Clinically Significant Changes From Baseline in Vital Signs Abnormalities Number of Participants With Worst On-study Laboratory Abnormalities Combination cohort 1, 2, and 3: Number of Participants With DLTs Number of Participants With TEAEs, Serious TEAEs, Treatment Related TEAEs, Grade 3 or 4 TEAEs Based on NCI CTCAE v4.0 Number of Participants With Clinically Significant Changes From Baseline in Vital Signs Abnormalities Number of Participants With Worst On-study Laboratory Abnormalities Expansion cohort: Percentage of Participants Achieving Disease Modifying Response (DMR)

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Secondary

Measure	Time frame
efficacy pharmacokinetics pharmacodynamics Monotherapy cohort: Single Dose- Maximum Observed Plasma Concentration (Cmax) of PF-04449913 Single Dose- Time to Reach Maximum Observed Plasma Concentration (Tmax) of PF-04449913 Single Dose- Terminal Plasma Half-life (T1/2) of PF-04449913 Single Dose- Area Under the Plasma Concentration Curve: From Time Zero to End of Dosing Interval (AUCtau), From Time Zero to Last Quantifiable Concentration (AUClast) and From Time Zero to Infinity (AUCinf) of PF-04449913 Single Dose- Clearance (CL/F) of PF-04449913 Single Dose- Volume of Distribution (Vz/F) of PF-04449913 Multiple Dose Cmax, Minimum Observed Plasma Concentration (Cmin), Average Observed Plasma Concentration (Cavg), Trough Plasma Concentration (Ctrough) of PF-04449913 Multiple Dose- Tmax of PF-04449913 Multiple Dose- AUCtau of PF-04449913 Multiple Dose- Clearance (CL/F) of PF-04449913 Multiple Dose- Accumulation Ratio (Rac) of PF-04449913 Multiple Dose- Steady State Accumulation Ratio (Rss) of PF-04449913 Ratio of GLI1 Levels at Baseline to Day 21 Cycle 1 Number of Participants With Best Response Combination cohort 1: Multiple Dose- Cmax, Cmin, Cavg, and Ctrough of PF-04449913 Multiple Dose- Tmax of PF-04449913 Multiple Dose- AUCtau of PF-04449913 Multiple Dose- CL/F of PF-04449913 Multiple Dose- Cmax, Cmin, Cavg, and Ctrough of Cytarabine Multiple Dose- Tmax of Cytarabine Multiple Dose- T1/2 of Cytarabine Multiple Dose- AUCinf and AUCtau of Cytarabine Multiple Dose- Cmax, Cmin, and Ctrough of Ara-uridine Multiple Dose- Tmax of Ara-uridine Ratio of GLI1 Levels at Baseline to Day 21 Cycle 1 Number of Participants With Best Response Percentage of Participants With Complete Remission (CR) or CR With Incomplete Blood Count Recovery (CRi) and DMR Duration of Complete Remission (CR) or CR With Incomplete Blood Count Recovery (CRi) and DMR Response Time to Response Overall Survival Combination cohort 2: Multiple Dose- Cmax, Cmin, Cavg, and Ctrough of PF-04449913 Multiple Dose	—

Measure

Time frame

efficacy pharmacokinetics pharmacodynamics Monotherapy cohort: Single Dose- Maximum Observed Plasma Concentration (Cmax) of PF-04449913 Single Dose- Time to Reach Maximum Observed Plasma Concentration (Tmax) of PF-04449913 Single Dose- Terminal Plasma Half-life (T1/2) of PF-04449913 Single Dose- Area Under the Plasma Concentration Curve: From Time Zero to End of Dosing Interval (AUCtau), From Time Zero to Last Quantifiable Concentration (AUClast) and From Time Zero to Infinity (AUCinf) of PF-04449913 Single Dose- Clearance (CL/F) of PF-04449913 Single Dose- Volume of Distribution (Vz/F) of PF-04449913 Multiple Dose Cmax, Minimum Observed Plasma Concentration (Cmin), Average Observed Plasma Concentration (Cavg), Trough Plasma Concentration (Ctrough) of PF-04449913 Multiple Dose- Tmax of PF-04449913 Multiple Dose- AUCtau of PF-04449913 Multiple Dose- Clearance (CL/F) of PF-04449913 Multiple Dose- Accumulation Ratio (Rac) of PF-04449913 Multiple Dose- Steady State Accumulation Ratio (Rss) of PF-04449913 Ratio of GLI1 Levels at Baseline to Day 21 Cycle 1 Number of Participants With Best Response Combination cohort 1: Multiple Dose- Cmax, Cmin, Cavg, and Ctrough of PF-04449913 Multiple Dose- Tmax of PF-04449913 Multiple Dose- AUCtau of PF-04449913 Multiple Dose- CL/F of PF-04449913 Multiple Dose- Cmax, Cmin, Cavg, and Ctrough of Cytarabine Multiple Dose- Tmax of Cytarabine Multiple Dose- T1/2 of Cytarabine Multiple Dose- AUCinf and AUCtau of Cytarabine Multiple Dose- Cmax, Cmin, and Ctrough of Ara-uridine Multiple Dose- Tmax of Ara-uridine Ratio of GLI1 Levels at Baseline to Day 21 Cycle 1 Number of Participants With Best Response Percentage of Participants With Complete Remission (CR) or CR With Incomplete Blood Count Recovery (CRi) and DMR Duration of Complete Remission (CR) or CR With Incomplete Blood Count Recovery (CRi) and DMR Response Time to Response Overall Survival Combination cohort 2: Multiple Dose- Cmax, Cmin, Cavg, and Ctrough of PF-04449913 Multiple Dose

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Countries

Japan

Contacts

Public ContactClinical Trials Information Desk

Pfizer R&D Japan G.K.

clinical-trials@pfizer.com+81-3-5309-7000

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026