MedTrace Logo

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

NCT03165721 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2026-02-18

Study results available
· View outcomes & findings →

Summary

Background:

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).

Objective:

To learn if SGI-110 causes GIST tumors to shrink or slows their growth. Also to test how it acts in the body.

Eligibility:

People ages 12 and older who have GIST, PHEO/PGL, or HLRCC that has not responded to other treatments

Design:

Participants will be screened with:

* Physical exam
* Urine tests
* Computed tomography (CT) or magnetic resonance imaging (MRI), or fluorodeoxyglucose (FDG)-positron emission tomography (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.

...

Conditions

Interventions

DRUG

SGI-110 (guadecitabine)

SGI-110 will be administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle. Cycles may be repeated until there is evidence of tumor progression clinically or by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 or there is intolerable toxicity that is not alleviated by dose reduction.

Sponsors & Collaborators

  • National Cancer Institute (NCI)

    lead NIH

Principal Investigators

  • John W Glod, M.D. · National Cancer Institute (NCI)

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
12 Years
Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-08-16
Primary Completion
2020-02-24
Completion
2020-02-24
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03165721 on ClinicalTrials.gov