Mithramycin for Children and Adults With Solid Tumors or Ewing Sarcoma
- Mithramycin is a drug that was first tested as a cancer therapy in the 1960s. It acted against some forms of cancer, but was never accepted as a treatment. Research suggests that it may be useful against some solid tumors, particularly Ewing sarcoma. Researchers want to see if mithramycin can be used to treat solid tumors in children and adults. It will be tested in different groups of people, including those with a type of Ewing sarcoma that contains a chemical called EWS-FLI1.
- To see if mithramycin is safe and effective against solid tumors and Ewing sarcoma in children and adults.
- Children and young adults between 1 and 17 years of age with solid tumors that have not responded to standard treatment.
- Adults at least 18 years of age with EWS-FLI1 Ewing sarcoma that has not responded to standard treatment.
- Children and young adults between 1 and 17 years of age with EWS-FLI1 Ewing sarcoma that has not responded to standard treatment.
- Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Imaging studies and tumor tissue samples will be used to monitor the cancer before treatment. Individuals with solid brain tumors will not be eligible.
- Participants will receive mithramycin every day for 7 days, followed by 14 days without treatment. Each 28-day round of treatment is called a cycle.
- Treatment will be monitored with frequent blood tests and imaging studies.
- Participants will continue to take the drug for as long as the side effects are not severe and the tumor responds to treatment.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase I/II Trial of Mithramycin in Children and Adults With Refractory Extracranial Solid Tumors (Phase I) or Ewing Sarcoma and EWSFLI1 Fusion Transcript (Phase II)|
- Phase I: determine the tolerability, toxicity, and recommended dose of mithramycin in pediactric patients with extracranial tumors. [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
- Phase II: determine response in children and adults with ewings sarcoma [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
- Evaluate NR0B1 expression in archival tumor tissue
- Describe pharmacokinetic parameters for each dose level (phase1)
|Study Start Date:||May 2012|
|Estimated Study Completion Date:||December 2015|
|Estimated Primary Completion Date:||December 2015 (Final data collection date for primary outcome measure)|
Please refer to this study by its ClinicalTrials.gov identifier: NCT01610570
|Contact: Lauren M Long, R.N.||(301) firstname.lastname@example.org|
|Contact: Brigitte C Widemann, M.D.||(301) email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office 888-624-1937|
|Principal Investigator:||Brigitte C Widemann, M.D.||National Cancer Institute (NCI)|