NCT01842646

Phase II Hedgehog Inhibitor for Myelodysplastic Syndrome (MDS)

A Phase II Study Evaluating the Oral Smoothened Inhibitor PF-04449913 in Patients With Myelodysplastic Syndrome (MDS)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 35 (actual)
• Sponsor: H. Lee Moffitt Cancer Center and Research Institute · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study is being done to see how safe an investigational drug is and test how well it will work to help people with refractory/relapsed myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML).

Detailed description

The main purpose of this study is to see whether the participant's disease responds favorably to the investigational drug, PF-04449913. Post treatment Phase: After coming off of active treatment study drug (PF-04449913), participants will be followed monthly for survival only. No other data will be captured during this time.

Conditions

• Myelodysplastic Syndrome (MDS)
• Chronic Myelomonocytic Leukemia (CMML)

Interventions

Type	Name	Description
DRUG	PF-04449913	Patients will be enrolled according to a two-step study design. Twenty patients will be enrolled in the first stage. All patients will be given a daily oral dose of PF-0444913 100 mg for up to 4 cycles, with an optional continuation phase. Dose escalation to 200 mg will be provided for patients who do not have at least hematologic improvement following 2 cycles, and dose reduction to 50 mg will be permitted for patients with significant toxicity. If at least 2 patients respond in the initial stage, and additional 15 patients will be enrolled in the second stage.

Timeline

• Start date: 2013-08-29
• Primary completion: 2016-01-31
• Completion: 2021-06-10
• First posted: 2013-04-29
• Last updated: 2021-06-14
• Results posted: 2017-03-14

Locations

1 site across 1 country: United States