Oral Treosulfan In Ewing's Sarcoma
| ISRCTN | ISRCTN11631773 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN11631773 |
| Secondary identifying numbers | 08/0230 |
- Submission date
- 28/11/2008
- Registration date
- 17/12/2008
- Last edited
- 25/01/2022
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Cancer
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Plain English Summary
Contact information
Dr Maria Michelagnoli
Scientific
Scientific
University College London
6th Floor Central
250 Euston Road
London
NW1 2PG
United Kingdom
Study information
| Study design | Non-randomised phase II study |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Non randomised study |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details below to request a patient information sheet |
| Scientific title | A phase II study to determine the efficacy and safety of conventional dose oral treosulfan in patients with advanced pre-treated Ewing's sarcoma |
| Study acronym | OTIS |
| Study hypothesis | This is a non-randomised phase II study which aims to explore the efficacy and toxicity of oral treosulfan in patients with advanced, pre-treated Ewing's sarcoma. If conventional dose oral treosulfan is demonstrated to be active in patients with advanced Ewing's sarcoma, it is proposed that this study should be followed by a phase III prospective randomised controlled trial comparing oral etoposide (the traditional active palliation agent in solid tumours) with oral treosulfan. |
| Ethics approval(s) | Derby 1 (Trent) and Derby 2 Research Ethics Committees, 07/08/2009, ref: 09H0405-29 |
| Condition | Ewing's sarcoma |
| Intervention | Non-randomised phase II study of oral treosulfan in advanced pre-treated Ewing's sarcoma. Treosulfan 1 g/m^2 will be administered in three divided doses, daily over 7 days and repeated every 28 days, until disease progression, unacceptable toxicity or patient refusal. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Phase II |
| Drug / device / biological / vaccine name(s) | Treosulfan |
| Primary outcome measure | Current primary outcomes measure as of 29/11/2011: Objective response rate achieved: tumour response will be measured as defined by the RECIST guidelines. Previous primary outcome measure: Objective response rate achieved: tumour response will be measured as defined by the RECIST guidelines. Following analysis of data for initial 15 patients, if the measured success is less than or equal to 10% then the study will be stopped. Otherwise, the study will close once a total of 25 eligible patients have been enrolled. |
| Secondary outcome measures | Current secondary outcome measures as of 29/11/2011: 1. Event-free survival 2. Overall survival 3. Toxicity : if there is more than one toxic death, or more than two unexpected treatment related serious adverse events occurring in two separate individuals, an Independent Data Monitoring Committee meeting will take place and advice sought regarding continuing the trial 4. Duration of response 5. Time to progression/relapse Previous secondary outcome measures: 1. Toxicity: if there is more than one toxic death, or more than two unexpected serious adverse events occurring in two separate individuals, the chief/co-investigators will consider stopping the trial 2. Duration of response 3. Time to progression 4. Correlation of deoxyribonucleic acid (DNA) cross-linking and retention with response and toxicity |
| Overall study start date | 01/03/2009 |
| Overall study end date | 11/09/2012 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Other |
| Sex | Both |
| Target number of participants | 25 patients in total |
| Total final enrolment | 21 |
| Participant inclusion criteria | Current inclusion criteria as of 29/11/2011: 1. Age less than 50 years, either sex 2. Histologically proven Ewing's sarcoma/peripheral neuroectodermal tumour 3. Patients with advanced, refractory disease who have failed at least one course of conventional chemotherapy and for whom no curative option exists 4. Measurable disease, defined by Response Evaluation Criteria in Solid Tumors (RECIST) 5. Evidence of disease progression within the preceding 8 weeks 6. Neutrophils greater than or equal to 1.5 x 10^9/l and platelet count greater than or equal to 100 x 10^9/l 7. Creatinine less than or equal to 1.5 x upper limit of normal (ULN) 8. Serum bilirubin and aspartate aminotransferase (AST)/alanine aminotransferase (ALT) less than or equal to 1.5 x ULN 9. Performance status 0-2 (patients 16 years or older) or Lansky Performance Status greater than 30 (patients under 16 years). (n.b. patients with WHO performance status 3 due to spinal disease will be eligible provided they are otherwise medically well) 10. Patient able to comply with protocol treatment (swallow capsules) and follow up 11. Life expectancy of three months or greater 12. Written informed consent of patient or parent/legal guardian Previous inclusion criteria: 9. World Health Organization (WHO) performance status greater than 3 (patients greater than 16 years); or Lansky Performance Status greater than 30 |
| Participant exclusion criteria | 1. Newly diagnosed, or resectable Ewing's sarcoma 2. Pregnant/lactating women, or women of childbearing potential unless using effective contraception 3. Concurrent treatment with any other anti-cancer therapy, except palliative radiotherapy to non-target lesions 4. Concurrent treatment with other experimental drugs 5. Concurrent treatment with growth factors |
| Recruitment start date | 02/02/2010 |
| Recruitment end date | 11/09/2012 |
Locations
Countries of recruitment
- England
- United Kingdom
Study participating centre
University College London
London
NW1 2PG
United Kingdom
NW1 2PG
United Kingdom
Sponsor information
University College London (UK)
University/education
University/education
c/o Joanna Galea-Lauri
Head of Clinical Trials
Joint UCLH and UCL Biomedical Research Unit
Ground Floor, Rosenheim Wing
25 Grafton Way
London
WC1E 5DB
England
United Kingdom
| Website | http://www.ucl.ac.uk/ |
|---|---|
"ROR" |
https://ror.org/02jx3x895 |
Funders
Funder type
Charity
Adam Dealey Foundation (UK)
No information available
Medac International (UK)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan | Not provided at time of registration |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | results | 01/01/2015 | Yes | No | |
| Plain English results | 28/05/2015 | 25/01/2022 | No | Yes |
Editorial Notes
25/01/2022: The following changes have been made:
1. The Cancer Research UK lay results summary has been added.
2. The total final enrolment number has been added.
