Trial Outcomes & Findings for 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 NCT03165721)

Last Updated: 2022-04-05

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

After the first 4 weeks, then every 8 weeks up to 65 weeks

Results posted on

2022-04-05

Participant Flow

Participant milestones

Participant milestones
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Overall Study STARTED	7	1	1
Overall Study COMPLETED	3	1	1
Overall Study NOT COMPLETED	4	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Overall Study Refused further treatment	3	0	0
Overall Study Physician Decision	1	0	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=7 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.	Total n=9 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	9 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Continuous	34.86 years STANDARD_DEVIATION 16.03 • n=5 Participants	41 years STANDARD_DEVIATION 0 • n=7 Participants	23 years STANDARD_DEVIATION 0 • n=5 Participants	34.22 years STANDARD_DEVIATION 14.64 • n=4 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	6 Participants n=4 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	3 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	3 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	6 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	6 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Region of Enrollment United States	7 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants	9 participants n=4 Participants

PRIMARY outcome

Timeframe: After the first 4 weeks, then every 8 weeks up to 65 weeks

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=7 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Number of Participants With an Overall Response (Complete Response or Partial Response) of SGI-11 Using the Response Evaluation Criteria in Solid Tumors (RECIST) Complete Response	0 Participants	0 Participants	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) Partial Response	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, end of cycle 4, and post therapy follow up, approximately 30 days after the final dose of study drug

Population: 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.

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).

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=7 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Change in Distress From Baseline Baseline	6.4 Scores on a scale Standard Deviation 1.4	7 Scores on a scale	2 Scores on a scale
Change in Distress From Baseline End of cycle 4	4.5 Scores on a scale Standard Deviation 1.5	2 Scores on a scale	—
Change in Distress From Baseline Post therapy follow up, approximately 30 days after the final dose of study drug	4.1 Scores on a scale Standard Deviation 2.4	3 Scores on a scale	—

POST_HOC outcome

Timeframe: 6 months

Population: Patient's progressed at 3.9 months and 1.1 month respectively, for the second and third groups.

Time interval from start of treatment to documented evidence of disease progression. Progression were evaluated in this study using the new international criteria proposed by the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1). Disease progression is defined as at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered progressions.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=7 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Progression Free Survival Probability at 6 Months	85.7 Percent probability Interval 33.4 to 97.9	0 Percent probability	0 Percent probability

POST_HOC outcome

Timeframe: 12 months

Population: Patient's progressed at 3.9 months and 1.1 month respectively, for the second and third groups.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=7 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Progression Free Survival (PFS) Probability at 12 Months	53.6 Percent probability Interval 13.2 to 82.5	0 Percent probability	0 Percent probability

POST_HOC outcome

Timeframe: Time from treatment start date until date of death or date last known alive, up to approximately 3 years, 2 months.

Time from treatment start date until date of death or date last known alive.

Outcome measures

Outcome data not reported

POST_HOC outcome

Timeframe: Baseline, at the end of cycle 4, and post therapy follow up, approximately 30 days after the final dose of study drug

Population: Participant off therapy due to progressive disease (PD) at the end of cycle 1 in the third Group.

Quality of Life (QOL) was assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) Global Physical Health. Overall scores were derived from five quality of life domains: physical function, pain interference, fatigue, emotional health and social health). T-Score distributions are standardized so that a score of 50 represents the mean for the US general population with a standard deviation around that mean of 10 points.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=7 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Quality of Life (QOL) Relative to Any Degree of Response (Patient-Reported Outcomes Measurement Information System (PROMIS) Global Physical Health) Post therapy follow up, approximately 30 days after the final dose of study drug	44.2 t-score Interval 37.5 to 50.9	54.1 t-score	NA t-score Participant off therapy due to progressive disease (PD) at the end of cycle 1.
Quality of Life (QOL) Relative to Any Degree of Response (Patient-Reported Outcomes Measurement Information System (PROMIS) Global Physical Health) Baseline	45.9 t-score Interval 40.5 to 51.3	54.1 t-score	57.7 t-score
Quality of Life (QOL) Relative to Any Degree of Response (Patient-Reported Outcomes Measurement Information System (PROMIS) Global Physical Health) End of cycle 4	46.5 t-score Interval 40.6 to 52.4	61.9 t-score	NA t-score Participant off therapy due to progressive disease (PD) at the end of cycle 1.

POST_HOC outcome

Timeframe: Baseline, at the end of cycle 4, and post therapy follow up, approximately 30 days after the final dose of study drug

Population: 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.

Quality of Life (QOL) was assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) Global Mental Health. Overall scores were derived from five quality of life domains: physical function, pain interference, fatigue, emotional health and social health). T-Score distributions are standardized so that a score of 50 represents the mean for the US general population with a standard deviation around that mean of 10 points.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=7 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Quality of Life (QOL) Relative to Any Degree of Response (Patient-Reported Outcomes Measurement Information System (PROMIS) Global Mental Health) Baseline	43.2 t-score Interval 39.7 to 46.7	41.1 t-score	62.5 t-score
Quality of Life (QOL) Relative to Any Degree of Response (Patient-Reported Outcomes Measurement Information System (PROMIS) Global Mental Health) End of cycle 4	46.0 t-score Interval 41.9 to 50.1	53.3 t-score	—
Quality of Life (QOL) Relative to Any Degree of Response (Patient-Reported Outcomes Measurement Information System (PROMIS) Global Mental Health) Post therapy follow up, approximately 30 days after the final dose of study drug	41.2 t-score Interval 36.8 to 45.6	45.8 t-score	—

POST_HOC outcome

Timeframe: 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.

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=7 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca n=1 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)	7 Participants	1 Participants	1 Participants

POST_HOC outcome

Timeframe: 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.

Population: 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).

The maximum observed analyte concentration in serum was reported.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=9 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Maximum Observed Plasma Concentration (Cmax) of Guadecitabine (SGI-110)	45 ng/mL Geometric Coefficient of Variation 42.7	—	—

POST_HOC outcome

Timeframe: Day 14 of cycle 1

Population: 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 from baseline in long interspersed nuclear element -1 (LINE-1) demethylation in peripheral blood mononuclear cells (PBMC) using pyrosequencing and urine and plasma glycolytic metabolites.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=2 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Percent Decrease in Long Interspersed Nuclear Element -1 (LINE-1) Demethylation	45 Percent decrease Interval 42.2 to 47.8	—	—

POST_HOC outcome

Plasma decay half-life is the time measured for the plasma concentration of the drug to decrease by one half.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=2 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Half Life (t1/2) of Guadecitabine (SGI-110) Patient 2	1.73 hrs	—	—
Half Life (t1/2) of Guadecitabine (SGI-110) Patient 3	1.35 hrs	—	—

POST_HOC outcome

Time it takes Guadecitabine (SGI-110) the reach the maximum concentration in plasma.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=9 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Time to Maximum Observed Plasma Concentration (Tmax) of Guadecitabine (SGI-110)	4.0 hr Interval 1.0 to 5.5	—	—

POST_HOC outcome

The AUC is a measure of the serum concentration of the drug over time. It is used to characterize drug absorption.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=9 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Area Under the Concentration Time Curve (AUC 0-5hr)	114.1 hr*ng/mL Geometric Coefficient of Variation 48.1	—	—

POST_HOC outcome

Apparent volume of distribution is the theoretical volume that would be necessary to contain the total amount of an administered drug at the same concentration that it is observed in the blood plasma.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=2 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
V(2)/F (Apparent Volume of Distribution) Patient 2	1127 L	—	—
V(2)/F (Apparent Volume of Distribution) Patient 3	1147 L	—	—

POST_HOC outcome

Apparent total body clearance is the time it takes for all Guadecitabine (SGI-110) to be removed from the body.

Outcome measures

Outcome measures
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=2 Participants SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
(CL/F) Apparent Total Body Clearance Patient 2	452 L/hr	—	—
(CL/F) Apparent Total Body Clearance Patient 3	588 L/hr	—	—

Adverse Events

Patients ≥ 12 Years of Age w/Wild-Type GIST

Serious events: 4 serious events

Other events: 7 other events

Deaths: 0 deaths

Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Patients ≥ 12 Years of Age w/Wild-Type GIST n=7 participants at risk SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Gastrointestinal stromal tumor (GIST) 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.	Patients ≥ 12 Years of Age w/PHEO/PGL With SDH-deficient PHE n=1 participants at risk SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Pheochromocytoma and Paraganglioma (PHEO/PGL) with succinate dehydrogenase (SDH)-deficient PHE 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.	Patients ≥ 12 Years of Age w/HLRCC-associated Renal Cell Ca n=1 participants at risk SGI-110 administered subcutaneously at 45mg/m\^2/day x 5 days on a 28-day cycle Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated Renal Cell Ca 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.
Investigations Neutrophil count decreased	28.6% 2/7 • Number of events 2 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	100.0% 1/1 • Number of events 1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.
Investigations White blood cell decreased	14.3% 1/7 • Number of events 1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	100.0% 1/1 • Number of events 1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.
Gastrointestinal disorders Abdominal pain	14.3% 1/7 • Number of events 1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.
Vascular disorders Hypertension	14.3% 1/7 • Number of events 2 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.
Infections and infestations Lung infection	14.3% 1/7 • Number of events 1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.
Nervous system disorders Reversible posterior leukoencephalopathy syndrome	14.3% 1/7 • Number of events 1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.
Nervous system disorders Seizure	14.3% 1/7 • Number of events 1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.
Infections and infestations Sepsis	14.3% 1/7 • Number of events 1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.
Gastrointestinal disorders Small intestinal obstruction	14.3% 1/7 • Number of events 1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.
Surgical and medical procedures Surgical and medical procedures - Other, Tumor debulking	14.3% 1/7 • Number of events 1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.
Gastrointestinal disorders Upper gastrointestinal hemorrhage	14.3% 1/7 • Number of events 1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.	0.00% 0/1 • Date treatment consent signed to date off study, approximately 1 month and 5 days for the Gastrointestinal stromal tumor (GIST) group, 3 months and 25 days for the Pheochromocytoma and Paraganglioma (Pheo/PGL) group, and 30 months and 2 days for the Renal Cell Cancer group.

Other adverse events

Additional Information

Dr. John Glod

National Cancer Institute

Phone: 240-760-6194

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place