Trial · NCT03165721
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, Phase 2, Terminated, National Cancer Institute (NCI); results first posted 2021-05-10
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ConductScience Library. 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. Trials, record NCT03165721. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03165721
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
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
- NCT number
- NCT03165721
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 9 participants, actual
- Lead sponsor
- National Cancer Institute (NCI)
- Conditions
- Paraganglioma, Gastrointestinal Stromal Tumors, Carcinoma, Renal Cell, Renal Neoplasms, Pheochromocytoma
- Interventions
- SGI-110 (guadecitabine)
- First posted
- 2017-05-24
- Last updated
- 2026-02-18
- Results first posted
- 2021-05-10
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With an Overall Response (Complete Response or Partial Response) of SGI-11 Using the Response Evaluation Criteria in Solid Tumors (RECIST) | After the first 4 weeks, then every 8 weeks up to 65 weeks | Clinical activity of SGI-11 was assessed using the RECISTv1.1. Complete Response (CR) is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial Response (PR) is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum of diameters. |
| Secondary | Change in Distress From Baseline | Baseline, end of cycle 4, and post therapy follow up, approximately 30 days after the final dose of study drug | Change in distress from baseline was assessed by The Distress Thermometer (DT) visual analog scale. An overall score is derived from specific physical, emotional and family issues identified by the participant to be stressful. No distress (0-4), moderate stress (5) and high distress (6-10). |
Source ClinicalTrials.gov
Outcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With an Overall Response (Complete Response or Partial Response) of SGI-11 Using the Response Evaluation Criteria in Solid Tumors (RECIST) | After the first 4 weeks, then every 8 weeks up to 65 weeks | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Change in Distress From Baseline | Baseline, end of cycle 4, and post therapy follow up, approximately 30 days after the final dose of study drug | n=6 for the first Group at the end of cycle 4; this time data at this time point was not collected for one participant. Participant off therapy due to progressive disease (PD) at the end of cycle 1 for the third Group. | Scores on a scale | MEAN |
| POST_HOC | Progression Free Survival Probability at 6 Months | 6 months | Patient's progressed at 3.9 months and 1.1 month respectively, for the second and third groups. | Percent probability | NUMBER |
| POST_HOC | Progression Free Survival (PFS) Probability at 12 Months | 12 months | Patient's progressed at 3.9 months and 1.1 month respectively, for the second and third groups. | Percent probability | NUMBER |
| POST_HOC | Overall Survival (OS) | Time from treatment start date until date of death or date last known alive, up to approximately 3 years, 2 months. | |||
| POST_HOC | Quality of Life (QOL) Relative to Any Degree of Response (Patient-Reported Outcomes Measurement Information System (PROMIS) Global Physical Health) | Baseline, at the end of cycle 4, and post therapy follow up, approximately 30 days after the final dose of study drug | Participant off therapy due to progressive disease (PD) at the end of cycle 1 in the third Group. | t-score | MEAN |
| POST_HOC | Quality of Life (QOL) Relative to Any Degree of Response (Patient-Reported Outcomes Measurement Information System (PROMIS) Global Mental Health) | Baseline, at the end of cycle 4, and post therapy follow up, approximately 30 days after the final dose of study drug | n=6 for the first Group at the end of cycle 4; this time data at this time point was not collected for one participant. Participant off treatment at cycle 1 due to progressive disease (PD) in the third Group. | t-score | MEAN |
| POST_HOC | Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0) | Date treatment consent signed to date off study, approx. 1 mos and 5 days for the Gastrointestinal stromal tumor (GIST) group; 3 mos and 25 days for the Pheochromocytoma/Paraganglioma (Pheo/PGL) group;and 30 mos and 2 days for the Renal Cell Cancer group. | Participants | COUNT_OF_PARTICIPANTS | |
| POST_HOC | Maximum Observed Plasma Concentration (Cmax) of Guadecitabine (SGI-110) | Prior to first dose in cycle 1 and at 0.5 hour (+/- 15 minutes), 1 hour (+/- 15 minutes), 2 hour (+/-30 minutes), 4 hour (+/- 30 minutes), 6 hour (+/- 1 hour) and 24 hours (+/- 2 hours) after the first dose of drug in Cycle 1. | Data was analyzed together for all participants because tumor type would not be expected to impact pharmacokinetic parameters or measures of pharmacodynamics that use peripheral blood mononuclear cells (LINE-1 demethylation). | ng/mL | GEOMETRIC_MEAN |
| POST_HOC | Percent Decrease in Long Interspersed Nuclear Element -1 (LINE-1) Demethylation | Day 14 of cycle 1 | Data was analyzed together for all patients because tumor type would not be expected to impact pharmacokinetic parameters or measures of pharmacodynamics that use peripheral blood mononuclear cells (LINE-1 demethylation). | Percent decrease | MEAN |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group48
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With an Overall Response (Complete Response or Partial Response) of SGI-11 Using the Response Evaluation Criteria in Solid Tumors (RECIST) | Patients ≥ 12 Years of Age w/Wild-Type GIST | Complete Response | 0 | Participants | |
| Number of Participants With an Overall Response (Complete Response or Partial Response) of SGI-11 Using the Response Evaluation Criteria in Solid Tumors (RECIST) | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | Complete Response | 0 | Participants | |
| Number of Participants With an Overall Response (Complete Response or Partial Response) of SGI-11 Using the Response Evaluation Criteria in Solid Tumors (RECIST) | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | Complete Response | 0 | Participants | |
| Number of Participants With an Overall Response (Complete Response or Partial Response) of SGI-11 Using the Response Evaluation Criteria in Solid Tumors (RECIST) | Patients ≥ 12 Years of Age w/Wild-Type GIST | Partial Response | 0 | Participants | |
| Number of Participants With an Overall Response (Complete Response or Partial Response) of SGI-11 Using the Response Evaluation Criteria in Solid Tumors (RECIST) | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | Partial Response | 0 | Participants | |
| Number of Participants With an Overall Response (Complete Response or Partial Response) of SGI-11 Using the Response Evaluation Criteria in Solid Tumors (RECIST) | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | Partial Response | 0 | Participants | |
| Change in Distress From Baseline | Patients ≥ 12 Years of Age w/Wild-Type GIST | Baseline | 6.4 | Standard Deviation 1.4 | Scores on a scale |
| Change in Distress From Baseline | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | Baseline | 7 | Scores on a scale | |
| Change in Distress From Baseline | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | Baseline | 2 | Scores on a scale | |
| Change in Distress From Baseline | Patients ≥ 12 Years of Age w/Wild-Type GIST | End of cycle 4 | 4.5 | Standard Deviation 1.5 | Scores on a scale |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Patients ≥ 12 Years of Age w/Wild-Type GIST | 7 | |
| Overall Study | STARTED | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | 1 | |
| Overall Study | STARTED | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | 1 | |
| Overall Study | COMPLETED | Patients ≥ 12 Years of Age w/Wild-Type GIST | 3 | |
| Overall Study | COMPLETED | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | 1 | |
| Overall Study | COMPLETED | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | 1 | |
| Overall Study | NOT COMPLETED | Patients ≥ 12 Years of Age w/Wild-Type GIST | 4 | |
| Overall Study | NOT COMPLETED | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | 0 | |
| Overall Study | NOT COMPLETED | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | 0 |
Source ClinicalTrials.gov
Adverse events294
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anemia | Blood and lymphatic system disorders | Patients ≥ 12 Years of Age w/Wild-Type GIST | 2 | 7 | 12 |
| other | Anemia | Blood and lymphatic system disorders | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | 0 | 1 | 0 |
| other | Anemia | Blood and lymphatic system disorders | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | 1 | 1 | 2 |
| other | Anorexia | Metabolism and nutrition disorders | Patients ≥ 12 Years of Age w/Wild-Type GIST | 2 | 7 | 2 |
| other | Anorexia | Metabolism and nutrition disorders | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | 0 | 1 | 0 |
| other | Anorexia | Metabolism and nutrition disorders | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | 1 | 1 | 1 |
| other | Dyspnea | Respiratory, thoracic and mediastinal disorders | Patients ≥ 12 Years of Age w/Wild-Type GIST | 2 | 7 | 2 |
| other | Dyspnea | Respiratory, thoracic and mediastinal disorders | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | 0 | 1 | 0 |
| other | Dyspnea | Respiratory, thoracic and mediastinal disorders | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | 1 | 1 | 1 |
| other | Fatigue | General disorders | Patients ≥ 12 Years of Age w/Wild-Type GIST | 3 | 7 | 3 |
1 to 10 of 294Source ClinicalTrials.gov
See all 294 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Patients ≥ 12 Years of Age w/Wild-Type GIST | 0 | 7 |
| Total, all-cause mortality | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | 0 | 1 |
| Total, all-cause mortality | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | 0 | 1 |
| Total, other adverse events | Patients ≥ 12 Years of Age w/Wild-Type GIST | 7 | 7 |
| Total, other adverse events | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | 1 | 1 |
| Total, other adverse events | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | 1 | 1 |
| Total, serious adverse events | Patients ≥ 12 Years of Age w/Wild-Type GIST | 4 | 7 |
| Total, serious adverse events | Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE | 1 | 1 |
| Total, serious adverse events | Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca | 0 | 1 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 36302175 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03165721
GET https://conductscience.org/api/trials/v1/NCT03165721/results?page=2