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Repurposing Atovaquone for the Treatment of Platinum-Resistant Ovarian Cancer

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT05998135
Recruitment Status : Recruiting
First Posted : August 18, 2023
Last Update Posted : February 20, 2024
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Namita Khanna, Emory University

Brief Summary:
This phase II trial test tests how well repurposing atovaquone works in treating patients with platinum-resistant ovarian cancer. Atovaquone is used for the treatment or prevention of certain infections. Atovaquone is in a class of medications called antiprotozoal agents. It works by stopping the growth of certain types of protozoa that can cause pneumonia. Giving atovaquone may be effective in treating platinum-resistant ovarian cancer and result in improved outcomes compared to standard chemotherapy regimens.

Condition or disease Intervention/treatment Phase
Ovarian High Grade Serous Adenocarcinoma Platinum-Resistant Ovarian Carcinoma Drug: Atovaquone Procedure: Biopsy Procedure: Computed Tomography Procedure: Paracentesis Phase 2

Detailed Description:


I. To determine progression free survival of twenty-eight patients with platinum-resistant ovarian cancer treated with atovaquone.


I. To determine clinical benefit rate (complete response, partial response or stable disease) at six months.

II. To determine overall survival. III. To quantitate the on-target STAT3 inhibitory effect of atovaquone on STAT3-dependent gene transcription.

IV. To quantitate changes of the tumor immune infiltrate by inhibition of STAT3 with atovaquone.


Patients receive atovaquone orally (PO) on study. Patients also undergo computed tomography (CT) and biopsy or paracentesis throughout the study.

After completion of study treatment, patients are followed up for 30 days and then every 6 month thereafter.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 28 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Clinical Trial Repurposing Atovaquone for the Treatment of Platinum-Resistant Ovarian Cancer
Actual Study Start Date : November 9, 2023
Estimated Primary Completion Date : June 30, 2024
Estimated Study Completion Date : June 30, 2025

Arm Intervention/treatment
Experimental: Treatment (atovaquone)
Patients receive atovaquone PO on study. Patients also undergo CT and biopsy or paracentesis throughout the study.
Drug: Atovaquone
Given PO
Other Names:
  • 566C80
  • BW-566C
  • BW-A566C
  • Mepron

Procedure: Biopsy
Undergo biopsy
Other Names:
  • Bx

Procedure: Computed Tomography
Undergo CT
Other Names:
  • CAT
  • CAT Scan
  • Computed Axial Tomography
  • Computerized Axial Tomography
  • Computerized axial tomography (procedure)
  • Computerized Tomography
  • CT
  • CT Scan
  • tomography

Procedure: Paracentesis
Undergo paracentesis

Primary Outcome Measures :
  1. Progression free survival (PFS) [ Time Frame: From initiation of atovaquone to progression or death, assessed up to 1 year ]
    Will be estimated using the Kaplan-Meier method, and a 95% confidence interval for median PFS will be estimated using the Brookmeyer-Crowley approach.

Secondary Outcome Measures :
  1. Clinical benefit rate [ Time Frame: At 6 months ]
    Clinical benefit rate is defined as complete response, partial response, and/or stable disease at six months. Clinical response will be assessed every 6 weeks for the first 24 months and every 12 weeks thereafter using Response Evaluation Criteria in Solid Tumors 1.1 criteria. Will be estimated as a proportion, with an exact 95% confidence interval estimated using the Clopper-Pearson method.

  2. Overall survival (OS) [ Time Frame: From diagnosis to death from any cause, where patients who are alive will be censored at last follow-up date, assessed up to 1 year ]
    OS will be estimated using the Kaplan-Meier method, and median survival will be calculated. A 95% confidence interval will be estimated using the Brookmeyer-Crowley approach.

  3. Incidence of adverse events [ Time Frame: Up to 30 days ]
    Safety will be determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5. Frequencies and percentages will be used to summarize safety events.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients with platinum-resistant, high-grade serous ovarian cancer, defined as disease progression within six months of completion of their last platinum-based chemotherapy
  • Patients must maintain Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
  • There will be no limitations on number of prior lines of therapy
  • Trial is open to non-English speaking patients
  • Trial is open to patients referred from community practice

Exclusion Criteria:

  • Patients who are < 18 years old
  • Patients who are pregnant or breastfeeding (due to cancer of their reproductive organs, patients enrolled in the trial are unable to conceive)
  • Patients who are incarcerated
  • Patients who are unable to provide consent / lack decision-making capacity

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT05998135

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Contact: Namita Khanna, MD, MSPH 404-778-3401

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United States, Georgia
Emory University Hospital/Winship Cancer Institute Recruiting
Atlanta, Georgia, United States, 30322
Contact: Nina Kimball    404-778-8145   
Contact: Maisey S Ratcliffe    404-778-3449   
Principal Investigator: Namita Khanna, MD, MSPH         
Sponsors and Collaborators
Emory University
National Cancer Institute (NCI)
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Principal Investigator: Namita Khanna, MD, MSPH Emory University Hospital/Winship Cancer Institute
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Responsible Party: Namita Khanna, Principal Investigator, Emory University Identifier: NCT05998135    
Other Study ID Numbers: STUDY00005363
NCI-2023-03479 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
STUDY00005363 ( Other Identifier: Emory University )
WINSHIP5782-22 ( Other Identifier: Emory University )
P30CA138292 ( U.S. NIH Grant/Contract )
First Posted: August 18, 2023    Key Record Dates
Last Update Posted: February 20, 2024
Last Verified: February 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Cystadenocarcinoma, Serous
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms, Cystic, Mucinous, and Serous
Anti-Infective Agents
Antiprotozoal Agents
Antiparasitic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action