Clinical trial · Interventional
A Study Of PF-04449913 In Select Hematologic Malignancies
A PHASE 1 STUDY TO EVALUATE THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF PF-04449913, AN ORAL HEDGEHOG INHIBITOR, ADMINISTERED AS SINGLE AGENT IN SELECT HEMATOLOGIC MALIGNANCIES
NCT00953758CI-TRIAL-00076489completedPhase 1Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This study examines the effect of a small molecule inhibitor to the Sonic Hedgehog pathway on select hematologic malignancies.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Hematologic Malignancies | Hematopoietic and Lymphoid Cell Neoplasm | ALIAS | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| PF-04449913 | Drug | Glasdegib | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- 1
- interventionNames
- Drug: PF-04449913
Primary outcomes (1)
- measure
- Number of Participants With First Cycle Dose-limiting Toxicities (DLTs)
- timeFrame
- Cycle 1 Day 1 to end of Cycle 1 (28 days)
- description
- Any DLT event in Cycle 1: 1) Grade \>=3 non-hematologic toxicity that had been maximally treated, 2) prolonged myelosupression that lasted greater than (\>) 42 days from the point of detection in a normal bone marrow (less than \[\<\] 500 per microliter \[/uL\] or platelet count \<10,000/uL, or hemoglobin \<8 gram per deciliter \[g/dL\] with \<5% blasts and no evidence of disease or dysplasia), 3) inability to deliver \>= 80% of the planned doses due to PF-04449913 related non-hematologic and hematologic toxicities
Secondary outcomes (30)
- measure
- Percentage of Participants With Treatment-emergent Adverse Events (AEs) by National Cancer Institute (NCI) Common Terminology Criteria (CTC) for AEs (CTCAE) (Version 3.0) Grade
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Patients with select advanced hematologic malignancies who are refractory, resistant or intolerant to prior therapies. They may be newly diagnosed and previously untreated, but not eligible for standard treatment options, or for whom standard therapies are not anticipated to result in a durable response. * ECOG performance status 0 to 2 * Adequate organ function 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 or skin cancer * Active GVHD other than Grade 1 skin involvement * Known malabsorption syndrome * Patient has an active, life threatening or clinically significant uncontrolled systemic infection
References
Publications (2)
- DERIVEDFostvedt LK, Shaik N, Martinelli G, Wagner AJ, Ruiz-Garcia A. Exposure-response modeling of the effect of glasdegib on cardiac repolarization in patients with cancer. Expert Rev Clin Pharmacol. 2021 Jul;14(7):927-935. doi: 10.1080/17512433.2021.1925538. Epub 2021 May 18. PMID 33993815
- DERIVEDMartinelli G, Oehler VG, Papayannidis C, Courtney R, Shaik MN, Zhang X, O'Connell A, McLachlan KR, Zheng X, Radich J, Baccarani M, Kantarjian HM, Levin WJ, Cortes JE, Jamieson C. Treatment with PF-04449913, an oral smoothened antagonist, in patients with myeloid malignancies: a phase 1 safety and pharmacokinetics study. Lancet Haematol. 2015 Aug;2(8):e339-46. doi: 10.1016/S2352-3026(15)00096-4. Epub 2015 Jul 26. PMID 26688487