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NCT01858168

Phase I Study of Olaparib and Temozolomide for Ewings Sarcoma or Rhabdoomyosarcoma

Completed Phase 1 Last updated 19 December 2025
What this trial tests

Phase 1 trial testing Olaparib in Ewing Sarcoma in 49 participants. Completed in 12 December 2025.

Timeline
1 July 2013
Primary endpoint
1 November 2024
12 December 2025

Quick facts

Lead sponsorMassachusetts General Hospital
PhasePhase 1
StatusCompleted
Study typeINTERVENTIONAL
Allocationnon randomized
Designparallel
Maskingnone
Primary purposetreatment
Enrollment49
Start date1 July 2013
Primary completion1 November 2024
Estimated completion12 December 2025
Sites5 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Massachusetts General Hospital

Who can join

16 and older, any sex, with Ewing Sarcoma or Rhabdomyosarcoma. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Zebrafish disease models in drug discovery: from preclinical modelling to clinical trials.
    Patton EE, Zon LI, Langenau DM. · · 2021 · cited 379× · PMID 34117457 · DOI 10.1038/s41573-021-00210-8
  2. Targeting DNA damage response in cancer therapy.
    Hosoya N, Miyagawa K. · · 2014 · cited 232× · PMID 24484288 · DOI 10.1111/cas.12366
  3. Zebrafish patient avatars in cancer biology and precision cancer therapy.
    Fazio M, Ablain J, Chuan Y, Langenau DM, et al · · 2020 · cited 153× · PMID 32251397 · DOI 10.1038/s41568-020-0252-3
  4. Biology of Bone Sarcomas and New Therapeutic Developments.
    Brown HK, Schiavone K, Gouin F, Heymann MF, et al · · 2018 · cited 116× · PMID 29238848 · DOI 10.1007/s00223-017-0372-2
  5. Current status of poly(ADP-ribose) polymerase inhibitors and future directions.
    Ohmoto A, Yachida S. · · 2017 · cited 96× · PMID 29138572 · DOI 10.2147/ott.s139336
  6. Advances and perspectives of PARP inhibitors.
    Yi M, Dong B, Qin S, Chu Q, et al · · 2019 · cited 83× · PMID 31737426 · DOI 10.1186/s40164-019-0154-9
  7. Molecular mechanisms underpinning sarcomas and implications for current and future therapy.
    Damerell V, Pepper MS, Prince S. · · 2021 · cited 75× · PMID 34188019 · DOI 10.1038/s41392-021-00647-8
  8. Management of recurrent Ewing sarcoma: challenges and approaches.
    Van Mater D, Wagner L. · · 2019 · cited 71× · PMID 30988632 · DOI 10.2147/ott.s170585

Verify or expand the search:

Other trials of Olaparib

Trials testing the same drug.

Other recruiting trials for Ewing Sarcoma

Currently open trials in the same condition.

Other Massachusetts General Hospital trials

Trials by the same sponsor.

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Data sources for this page

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