NCT01858168CompletedPhase 1
Phase I Study of Olaparib and Temozolomide for Ewings Sarcoma or Rhabdoomyosarcoma
Phase I Study of Olaparib and Temozolomide in Adult Patients With Recurrent/Metastatic Ewing's Sarcoma or Rhabdomyosarcoma Following Failure of Prior Chemotherapy
Source: ClinicalTrials.gov · Last updated 19 Dec 2025
Brief summary (registry, in English)
This research study is a Three arm Phase I clinical trial, which tests the safety of an investigational drug or combination of drugs and also tries to define the appropriate dose of the combination of investigational drugs to use for further studies. "Investigational" means that the combination of drugs is being studied. It also means that the FDA has not yet approved the combination of drugs for your type of cancer. Olaparib works by blocking the activity of a protein called poly (ADP-ribose) polymerase (PARP) which is involved in DNA repair. Cancer cells rely on PARP to repair their DNA and enable them to continue dividing. Olaparib has been used in research studies with other cancers. Information from those other research studies suggests that this drug may help to treat patients with Ewing's sarcoma. The investigational drug olaparib is not approved for any use outside of research studies. Temozolomide (Temodar) is approved by the FDA for the treatment of a type of brain tumor, glioblastoma. It has been studied in Ewing sarcoma in previous research studies. While it is not approved by the FDA for Ewing sarcoma, it is considered part of standard treatment for relapsed disease. Irinotecan is approved by the FDA for the treatment of gastrointestinal cancers. It has been studied in Ewing sarcoma in previous research studies. While it is not approved by the FDA for Ewing sarcoma, it is considered part of standard treatment for relapsed disease. Laboratory studies suggest that the combination of olaparib and temozolomide and/or irinotecan may help kill Ewing sarcoma or rhabdomyosarcoma cells. In this research study, the investigators are looking for the highest dose of the combination of olaparib and irinotecan and/or temozolomide that can be given safely. The investigators will also begin to collect information about the effects of the combination on Ewing sarcoma and rhabdomyosarcoma.
- Status (registry)
- Completed
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 49 (actual)
- First submitted
- 8 May 2013
- Start date
- 1 Jul 2013
- Primary completion date
- 1 Nov 2024
- Lead sponsor
- Massachusetts General Hospital
- Diseases
- Childhood cancers, Sarcoma
- NCT number
- NCT01858168
What you commit to
- Study type: Interventional · Phase 1
- Participants enrolled: 49
- From 1 Jul 2013 to the primary completion date, 1 Nov 2024
- 5 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Previous enrollment in the present study
- Participation in another clinical study with an investigational product during the 21 days prior to first dose of olaparib and temozolomide
- Receiving any systemic chemotherapy, radiotherapy (except for palliative reasons), within 2 weeks from the last dose prior to study treatment
- Concurrent use of the following classes of inhibitors of CYP3A4: azole antifungals, macrolide antibiotics, protease inhibitors
- Major surgery within 14 days of starting study treatment
- Known to be serologically positive for HIV and receiving antiviral therapy
- Need to continue treatment with any prohibited medications or have not completed the appropriate washout period for a prohibited medication
Other illness
- Persistent clinically significant toxicities caused by previous cancer therapy
- Considered a poor medical risk due to a serious, uncontrolled medical disorder, non-malignant systemic disease or active, uncontrolled infection
- Unable to swallow orally administered medication and subjects with gastrointestinal disorders likely to interfere with absorption of study medication
- Subjects with known active hepatitis B or C
- Known hypersensitivity to olaparib or any of the excipients of the product
- Uncontrolled seizures
Lab values
- Previously documented diagnosis of myelodysplastic syndrome (or any dysplastic leukocyte morphology suggestive of MDS) or acute myeloid leukemia
Other
- Involvement in the planning and/or conduct of the study
- Symptomatic uncontrolled brain metastases
- Pregnant or breastfeeding
Who the study is looking for
- Histologically confirmed Ewing's sarcoma
- Ewing's sarcoma must have progressed following at least one standard prior chemotherapy regimen
- Life expectancy of at least 16 weeks
- Willing to comply with the protocol for the duration of the study including pre- and post-treatment biopsies, undergoing treatment, scheduled visits and examinations including follow up
- Presence of measurable disease
- Prior approval from insurance company to obtain oral temozolomide for the duration of the study
Age in the registry: 16 years to - · Sex: All · Does not accept healthy volunteers
Sites (5)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Dana-Farber Cancer Institute Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Boston Children's Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Massachusetts General Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Brigham and Women's Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| St. Jude's Children's Research Hospital Coordinator of this site? Confirm its status | Memphis | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.