Phase II Hedgehog Inhibitor for Myelodysplastic Syndrome (MDS)
NCT01842646
ABOUT THIS STUDY
FOR MORE INFORMATION
Contact a representative by phone, email, or visiting the study website. Please see the references below:
Pfizer Clinical Trials Contact Center
1-800-718-1021
- Must have a pathologically confirmed diagnosis by World Health Organization (WHO) Criteria of MDS, CMML, or acute myeloid leukemia (AML) (except acute promyelocytic leukemia) with < 30% bone marrow blasts (RAEB-t by French American British criteria)
- Hypomethylating agent (azacitidine and/or decitabine) failure, defined as lack of response, disease progression, loss of response, or intolerance as deemed by the study investigator
- Adequate renal function, as evidenced by a serum creatinine ≤ 2 times the institutional upper limit of normal
- Adequate hepatic function, as evidenced by a serum bilirubin < 2 times the institutional upper limit of normal and an aspartic transaminase (AST) and alanine transaminase (ALT) < 2 times the institutional upper limit of normal. Indirect hyperbilirubinemia due to Gilbert's disease or hemolysis is permitted.
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
- Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
- Ability to understand and the willingness to sign a written informed consent document
- Patients with a history of prior therapy with another investigational agent within 4
weeks of the first planned dose of PF-0444913
- Patients may not be receiving any other investigational agents.
- History of allergic reactions attributed to compounds of similar chemical or biologic
composition to PF-04449913
- Prior therapy with another hedgehog inhibitor
- Concurrent use of any other agent for MDS, CMML, or AML. Growth factor use with
epoetin, darbepoetin, or granulocyte colony-stimulating factor must be terminated at
least 2 weeks before initiation of study treatment.
- Any uncontrolled concurrent illness that would, in the opinion of the investigator,
limit compliance with study requirements
- Second malignancy requiring active therapy
- A prolonged corrected QT interval (QTc) of ≥480 ms interval on electrocardiogram
- History of metastatic cancer diagnosed less than 2 years prior to the first planned
dose of PF-0444913
- Uncontrolled concurrent illness including, but not limited to, ongoing or active
infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
arrhythmia, or psychiatric illness/social situations that would limit compliance with
study requirements
- Pregnant women are excluded from this study because PF-04449913 is smoothened
inhibitor with the potential for teratogenic or abortifacient effects. Because there
is an unknown but potential risk for adverse events in nursing infants secondary to
treatment of the mother with PF-04449913. Breastfeeding should be discontinued if the
mother is treated with PF-04449913.
- Patients with immune deficiency are at increased risk of lethal infections when
treated with marrow-suppressive therapy. Therefore, HIV-positive patients receiving
combination antiretroviral therapy are excluded from the study because of possible
pharmacokinetic interactions with PF-04449913.
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Descriptive Information | ||||
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Brief Title ICMJE | Phase II Hedgehog Inhibitor for Myelodysplastic Syndrome (MDS) | |||
Official Title ICMJE | A Phase II Study Evaluating the Oral Smoothened Inhibitor PF-04449913 in Patients With Myelodysplastic Syndrome (MDS) | |||
Brief 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. | |||
Study Type ICMJE | Interventional | |||
Study Phase ICMJE | Phase 2 | |||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment | |||
Condition ICMJE |
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Intervention ICMJE | 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. Other Names:
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Study Arms ICMJE | Experimental: PF-04449913 Treatment
Treatment will be administered on an outpatient basis. All patients will be treated with an oral dose PF-04449913 at 100 mg daily in 4-week cycles for a total of 4 cycles. Patients who demonstrate no evidence of progressive disease (i.e. stable disease or better) may continue on treatment until disease progression or loss of response, limiting toxicity, or death. Intervention: Drug: PF-04449913 | |||
Publications * | Not Provided | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. | ||||
Recruitment Information | ||||
Recruitment Status ICMJE | Active, not recruiting | |||
Actual Enrollment ICMJE | 35 | |||
Original Estimated Enrollment ICMJE | Same as current | |||
Estimated Study Completion Date ICMJE | December 2021 | |||
Actual Primary Completion Date | January 31, 2016 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years and older (Adult, Older Adult) | |||
Accepts Healthy Volunteers ICMJE | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT01842646 | |||
Other Study ID Numbers ICMJE | MCC-17302 | |||
Has Data Monitoring Committee | No | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement ICMJE | Not Provided | |||
Responsible Party | H. Lee Moffitt Cancer Center and Research Institute | |||
Study Sponsor ICMJE | H. Lee Moffitt Cancer Center and Research Institute | |||
Collaborators ICMJE | Pfizer | |||
Investigators ICMJE |
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PRS Account | H. Lee Moffitt Cancer Center and Research Institute | |||
Verification Date | November 2020 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |