Trial Outcomes & Findings for Oral SM-88 in Patients With Metastatic HR+/HER2- Breast Cancer (NCT NCT04720664)
NCT ID: NCT04720664
Last Updated: 2023-08-28
Results Overview
Anti-tumor efficacy will be defined as partial response (PR) or complete response (CR) per Response Evaluation Criteria In Solid Tumors Criteria (RECIST) v1.1 for target lesions assessed by CT or MRI. Complete Response (CR): 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): At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
11 participants
Primary outcome timeframe
Every 8 weeks until progression (an average of 8 weeks)
Results posted on
2023-08-28
Participant Flow
Participant milestones
| Measure |
Oral SM-88
SM-88 taken with three conditioning agents: methoxsalen, phenytoin, and sirolimus
SM-88: • SM-88, Two 230 mg capsules taken orally twice daily (920 mg daily).
* Methoxsalen, One 10 mg capsule taken orally once daily.
* Phenytoin, One 50 mg tablet taken orally once daily.
* Sirolimus, One 0.5 mg tablet taken orally once daily.
|
|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
11
|
Reasons for withdrawal
| Measure |
Oral SM-88
SM-88 taken with three conditioning agents: methoxsalen, phenytoin, and sirolimus
SM-88: • SM-88, Two 230 mg capsules taken orally twice daily (920 mg daily).
* Methoxsalen, One 10 mg capsule taken orally once daily.
* Phenytoin, One 50 mg tablet taken orally once daily.
* Sirolimus, One 0.5 mg tablet taken orally once daily.
|
|---|---|
|
Overall Study
Adverse Event
|
2
|
|
Overall Study
Lack of Efficacy
|
9
|
Baseline Characteristics
Oral SM-88 in Patients With Metastatic HR+/HER2- Breast Cancer
Baseline characteristics by cohort
| Measure |
Oral SM-88
n=11 Participants
SM-88 taken with three conditioning agents: methoxsalen, phenytoin, and sirolimus
SM-88: • SM-88, Two 230 mg capsules taken orally twice daily (920 mg daily).
* Methoxsalen, One 10 mg capsule taken orally once daily.
* Phenytoin, One 50 mg tablet taken orally once daily.
* Sirolimus, One 0.5 mg tablet taken orally once daily.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Every 8 weeks until progression (an average of 8 weeks)Population: No patients responded to treatment on the first assessment.
Anti-tumor efficacy will be defined as partial response (PR) or complete response (CR) per Response Evaluation Criteria In Solid Tumors Criteria (RECIST) v1.1 for target lesions assessed by CT or MRI. Complete Response (CR): 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): At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.
Outcome measures
| Measure |
Oral SM-88
n=11 Participants
SM-88 taken with three conditioning agents: methoxsalen, phenytoin, and sirolimus
SM-88: • SM-88, Two 230 mg capsules taken orally twice daily (920 mg daily).
* Methoxsalen, One 10 mg capsule taken orally once daily.
* Phenytoin, One 50 mg tablet taken orally once daily.
* Sirolimus, One 0.5 mg tablet taken orally once daily.
|
|---|---|
|
Objective Response Rate (ORR)
|
0 percentage of patients
|
SECONDARY outcome
Timeframe: Every 8 weeks until Progression (an average of 8 weeks)Population: Outcome unable to be analyzed since there were no participants with a response.
Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST) v1.1 for target lesions assessed by CT or MRI. Complete Response (CR): 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): At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. Progressive Disease (PD): At least a 20% increase in the sum of 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. (Note: the appearance of one or more new lesions is also considered progression). Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Every 8 weeks until progression (an average of 8 weeks)Population: Median measure is in days.
Defined as the time in days from study entry to the first documented disease progression per RECIST v1.1 as assessed by local site or death. Patients who are alive and free from progression on the date of closing follow-up will be censored on that date.
Outcome measures
| Measure |
Oral SM-88
n=11 Participants
SM-88 taken with three conditioning agents: methoxsalen, phenytoin, and sirolimus
SM-88: • SM-88, Two 230 mg capsules taken orally twice daily (920 mg daily).
* Methoxsalen, One 10 mg capsule taken orally once daily.
* Phenytoin, One 50 mg tablet taken orally once daily.
* Sirolimus, One 0.5 mg tablet taken orally once daily.
|
|---|---|
|
Progression Free Survival (PFS)
|
51 days
Interval 28.0 to 79.0
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: No participants were on trial at 24 weeks so outcome can not be analyzed.
The percentage of patients who have complete response (CR), partial response (PR), or stable disease (SD) at ≥ 24 weeks will be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)The number of participants with treatment-related adverse events as assessed by CTCAE v5.0
Outcome measures
| Measure |
Oral SM-88
n=11 Participants
SM-88 taken with three conditioning agents: methoxsalen, phenytoin, and sirolimus
SM-88: • SM-88, Two 230 mg capsules taken orally twice daily (920 mg daily).
* Methoxsalen, One 10 mg capsule taken orally once daily.
* Phenytoin, One 50 mg tablet taken orally once daily.
* Sirolimus, One 0.5 mg tablet taken orally once daily.
|
|---|---|
|
Incidence of Treatment-Related Adverse Events [Safety and Tolerability]
|
6 Participants
|
Adverse Events
Oral SM-88
Serious events: 2 serious events
Other events: 10 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Oral SM-88
n=11 participants at risk
SM-88 taken with three conditioning agents: methoxsalen, phenytoin, and sirolimus
SM-88: • SM-88, Two 230 mg capsules taken orally twice daily (920 mg daily).
* Methoxsalen, One 10 mg capsule taken orally once daily.
* Phenytoin, One 50 mg tablet taken orally once daily.
* Sirolimus, One 0.5 mg tablet taken orally once daily.
|
|---|---|
|
Investigations
Infections and infestations - Other, specify
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Nervous system disorders
Dizziness
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
Other adverse events
| Measure |
Oral SM-88
n=11 participants at risk
SM-88 taken with three conditioning agents: methoxsalen, phenytoin, and sirolimus
SM-88: • SM-88, Two 230 mg capsules taken orally twice daily (920 mg daily).
* Methoxsalen, One 10 mg capsule taken orally once daily.
* Phenytoin, One 50 mg tablet taken orally once daily.
* Sirolimus, One 0.5 mg tablet taken orally once daily.
|
|---|---|
|
Ear and labyrinth disorders
Hearing impaired
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Gastrointestinal disorders
Abdominal distension
|
18.2%
2/11 • Number of events 2 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Gastrointestinal disorders
Abdominal pain
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Gastrointestinal disorders
Bloating
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Gastrointestinal disorders
Constipation
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Gastrointestinal disorders
Diarrhea
|
9.1%
1/11 • Number of events 2 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Gastrointestinal disorders
Dry mouth
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Gastrointestinal disorders
Gastrointestinal pain
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Gastrointestinal disorders
Nausea
|
36.4%
4/11 • Number of events 4 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Gastrointestinal disorders
Vomiting
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
General disorders
Fatigue
|
54.5%
6/11 • Number of events 7 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
General disorders
Fever
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
General disorders
General disorders and administration site conditions - Other, specify
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Infections and infestations
Infections and infestations - Other, specify
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Infections and infestations
Upper respiratory infection
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Investigations
Alanine aminotransferase increased
|
27.3%
3/11 • Number of events 3 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Investigations
Alkaline phosphatase increased
|
27.3%
3/11 • Number of events 4 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Investigations
Aspartate aminotransferase increased
|
63.6%
7/11 • Number of events 11 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Investigations
Blood bilirubin increased
|
18.2%
2/11 • Number of events 2 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Investigations
Cholesterol high
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Investigations
Serum amylase increased
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Investigations
Weight loss
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Metabolism and nutrition disorders
Anorexia
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Metabolism and nutrition disorders
Dehydration
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Metabolism and nutrition disorders
Hyponatremia
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Nervous system disorders
Dizziness
|
18.2%
2/11 • Number of events 4 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Nervous system disorders
Headache
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Psychiatric disorders
Depression
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Skin and subcutaneous tissue disorders
Scalp pain
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
9.1%
1/11 • Number of events 1 • From the time of the administration of study drug until discontinuation of therapy (every 4 weeks on Day 1 of each cycle in a 28 day cycle; approximately 3 months)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place