A Phase II Trial of the DNA Methyl Transferase Inhibitor, Guadecitabine (SGI-110), in Children and Adults With Wild Type GIST,Pheochromocytoma and Paraganglioma Associated With Succinate Dehydrogenase Deficiency and HLRCC-associated Kidney 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.|
|ClinicalTrials.gov Identifier: NCT03165721|
Recruitment Status : Recruiting
First Posted : May 24, 2017
Last Update Posted : March 6, 2019
Wild-type gastrointestinal stromal tumor (GIST) is a cancer in the esophagus, stomach, or intestines. It does not respond well to standard chemotherapy or radiation therapy. Most people with GIST are treated with imatinib. But it may not work in many children with GIST. Researchers think the drug SGI-110 may help treat people with GIST, pheochromocytoma and paraganglioma (PHEO/PGL), or kidney cancer related to hereditary leiomyomatosis and renal cell carcinoma (HLRCC).
To learn if SGI-110 causes GIST tumors to shrink or slows their growth. Also to test how it acts in the body.
People ages 12 and older who have GIST, PHEO/PGL, or HLRCC that has not responded to other treatments
Participants will be screened with:
- Physical exam
- Urine tests
- CT or MRI, or FDG-PET scan: A machine takes pictures of the body.
- Blood tests
Participants will be injected with SGI-110 under the skin each day for 5 days. This cycle will repeat every 28 days. The cycles repeat until their side effects get too bad or their cancer gets worse.
Participants will have tests throughout study:
- Physical exam and blood and urine tests before each cycle
- Blood tests on days 1, 7, 14, and 28 of the first cycle.
- Scans before cycle 1 and then every other cycle.
- Questionnaires about their pain and quality of life
- Tumor biopsy for those 18 and older: A needle removes a small piece of tumor.
After they stop treatment, participants will have a final visit. This includes an evaluation of their health, pain, and quality of life.
|Condition or disease||Intervention/treatment||Phase|
|Paraganglioma Gastrointestinal Stromal Tumors Carcinoma, Renal Cell Renal Neoplasms Pheochromocytoma||Drug: SGI-110 (guadecitabine)||Phase 2|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||70 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Trial of the DNA Methyl Transferase Inhibitor, SGI-110 (Guadecitabine), In Children And Adults With Wild Type GIST, Pheochromocytoma And Paraganglioma Associated With Succinate Dehydrogenase Deficiency And HLRCC-Associated Kidney Cancer|
|Actual Study Start Date :||August 16, 2017|
|Estimated Primary Completion Date :||December 1, 2020|
|Estimated Study Completion Date :||December 1, 2021|
Experimental: Arm 1
SGI-110 administered subcutaneously at 45mg/m2/day x 5 days on a 28-day cycle
Drug: SGI-110 (guadecitabine)
SGI-110 will be administered subcutaneously at 45mg/m2/day x 5 days on a 28-day cycle. Cycles may be repeated until there is evidence of tumor progression clinically or by RECIST v1.1 or there is intolerable toxicity that is not alleviated by dose reduction.
- Assess clinical activity of SGI-11 using RECIST [ Time Frame: After the first 4 weeks, then every 8 weeks ]Efficacy
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 ClinicalTrials.gov identifier (NCT number): NCT03165721
|Contact: Melissa M Spencer (Amaya), R.N.||(240) firstname.lastname@example.org|
|United States, Maryland|
|National Institutes of Health Clinical Center||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:||John W Glod, M.D.||National Cancer Institute (NCI)|