Trial Outcomes & Findings for A Pilot Study of 5-AZA and ATRA for Prostate Cancer With PSA-only Recurrence After Local Treatment (NCT NCT03572387)
NCT ID: NCT03572387
Last Updated: 2024-04-30
Results Overview
Number of participants with PSA response, as defined by PSA decreased \> 30% as compared from baseline.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
14 participants
Primary outcome timeframe
baseline and 24 weeks
Results posted on
2024-04-30
Participant Flow
Participant milestones
| Measure |
'Early' 5-AZA+ATRA
Patients will receive Lupron 7.5 mg x 1. Four weeks later, they will receive 5-AZA+ATRA treatment on a 28-day cycle, in the absence of toxicities, for 3 cycles, followed by 3 cycles of observation
Treatment:
5-Azacitidine: subcutaneously on days 1-5 at a dose of 40 mg/m\^2 all trans retinoic acid: 45 mg/m\^2, will be taken orally on days 3-7 of each cycle, divided into two doses
|
'Delayed' 5-AZA+ATRA
Patients will receive Lupron 7.5 mg x 1. Four weeks later, they will undergo "observation" every 28 days for 3 cycles. Thereafter, patients will receive 5-AZA+ATRA treatment on a 28-day cycle, in the absence of toxicities, for 3 cycles.
Treatment:
5-Azacitidine: subcutaneously on days 1-5 at a dose of 40 mg/m\^2 all trans retinoic acid: 45 mg/m\^2, will be taken orally on days 3-7 of each cycle, divided into two doses
-Taken for 3 cycles
|
|---|---|---|
|
Treatment Phase 1 - 3 Cycles
STARTED
|
6
|
8
|
|
Treatment Phase 1 - 3 Cycles
COMPLETED
|
6
|
8
|
|
Treatment Phase 1 - 3 Cycles
NOT COMPLETED
|
0
|
0
|
|
Treatment Phase 2 - 3 Cycles
STARTED
|
6
|
8
|
|
Treatment Phase 2 - 3 Cycles
COMPLETED
|
6
|
8
|
|
Treatment Phase 2 - 3 Cycles
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Pilot Study of 5-AZA and ATRA for Prostate Cancer With PSA-only Recurrence After Local Treatment
Baseline characteristics by cohort
| Measure |
'Early' 5-AZA+ATRA
n=6 Participants
Patients will receive Lupron 7.5 mg x 1. Four weeks later, they will receive treatment on a 28-day cycle, in the absence of toxicities, for 3 cycles.
Treatment:
5-Azacitidine: subcutaneously on days 1-5 at a dose of 40 mg/m\^2 all trans retinoic acid: 45 mg/m\^2, will be taken orally on days 3-7 of each cycle, divided into two doses
-Taken for 3 cycles
|
'Delayed' 5-AZA+ATRA
n=8 Participants
Patients will receive Lupron 7.5 mg x 1. Four weeks later, they will undergo "observation" every 28 days for 3 cycles. Thereafter, patients will receive 5-AZA+ATRA treatment on a 28-day cycle, in the absence of toxicities, for 3 cycles.
Treatment:
5-Azacitidine: subcutaneously on days 1-5 at a dose of 40 mg/m\^2 all trans retinoic acid: 45 mg/m\^2, will be taken orally on days 3-7 of each cycle, divided into two doses
-Taken for 3 cycles
|
Total
n=14 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
62.3 years
STANDARD_DEVIATION 6.8 • n=5 Participants
|
71.8 years
STANDARD_DEVIATION 5.04 • n=7 Participants
|
66.4 years
STANDARD_DEVIATION 7.64 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Gleason score
<=7 (low to intermediate grade cancer)
|
0 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Gleason score
>=8 (high grade cancer)
|
5 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Gleason score
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Prostate-Specific Antigen (PSA) doubling time
|
3.51 months
STANDARD_DEVIATION 1.48 • n=5 Participants
|
2.43 months
STANDARD_DEVIATION 1.06 • n=7 Participants
|
2.89 months
STANDARD_DEVIATION 1.33 • n=5 Participants
|
|
PSA
|
7.11 ng/ml
STANDARD_DEVIATION 11.7 • n=5 Participants
|
2.99 ng/ml
STANDARD_DEVIATION 2.56 • n=7 Participants
|
4.75 ng/ml
STANDARD_DEVIATION 7.81 • n=5 Participants
|
PRIMARY outcome
Timeframe: baseline and 24 weeksNumber of participants with PSA response, as defined by PSA decreased \> 30% as compared from baseline.
Outcome measures
| Measure |
'Early' 5-AZA+ATRA
n=6 Participants
Patients will receive Lupron 7.5 mg x 1. Four weeks later, they will receive 5-AZA+ATRA treatment on a 28-day cycle, in the absence of toxicities, for 3 cycles, followed by 3 cycles of observation
Treatment:
5-Azacitidine: subcutaneously on days 1-5 at a dose of 40 mg/m\^2 all trans retinoic acid: 45 mg/m\^2, will be taken orally on days 3-7 of each cycle, divided into two doses
|
'Delayed' 5-AZA+ATRA
n=8 Participants
Patients will receive Lupron 7.5 mg x 1. Four weeks later, they will undergo "observation" every 28 days for 3 cycles. Thereafter, patients will receive 5-AZA+ATRA treatment on a 28-day cycle, in the absence of toxicities, for 3 cycles.
Treatment:
5-Azacitidine: subcutaneously on days 1-5 at a dose of 40 mg/m\^2 all trans retinoic acid: 45 mg/m\^2, will be taken orally on days 3-7 of each cycle, divided into two doses
-Taken for 3 cycles
|
|---|---|---|
|
Number of Participants With PSA Response
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: baseline and 24 weeksPercentage of patients with prolongation of PSA doubling time (PSADT) post-treatment compared to baseline after treatment with 3 cycles of Aza and ATRA.
Outcome measures
| Measure |
'Early' 5-AZA+ATRA
n=6 Participants
Patients will receive Lupron 7.5 mg x 1. Four weeks later, they will receive 5-AZA+ATRA treatment on a 28-day cycle, in the absence of toxicities, for 3 cycles, followed by 3 cycles of observation
Treatment:
5-Azacitidine: subcutaneously on days 1-5 at a dose of 40 mg/m\^2 all trans retinoic acid: 45 mg/m\^2, will be taken orally on days 3-7 of each cycle, divided into two doses
|
'Delayed' 5-AZA+ATRA
n=8 Participants
Patients will receive Lupron 7.5 mg x 1. Four weeks later, they will undergo "observation" every 28 days for 3 cycles. Thereafter, patients will receive 5-AZA+ATRA treatment on a 28-day cycle, in the absence of toxicities, for 3 cycles.
Treatment:
5-Azacitidine: subcutaneously on days 1-5 at a dose of 40 mg/m\^2 all trans retinoic acid: 45 mg/m\^2, will be taken orally on days 3-7 of each cycle, divided into two doses
-Taken for 3 cycles
|
|---|---|---|
|
Percentage of Patients With Prolongation of PSA Doubling Time (PSADT) Post-treatment
|
33.3 percentage of participants
|
25 percentage of participants
|
Adverse Events
'Early' 5-AZA+ATRA
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
'Delayed' 5-AZA+ATRA
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
'Early' 5-AZA+ATRA
n=6 participants at risk
Patients will receive Lupron 7.5 mg x 1. Four weeks later, they will receive 5-AZA+ATRA treatment on a 28-day cycle, in the absence of toxicities, for 3 cycles, followed by 3 cycles of observation
Treatment:
5-Azacitidine: subcutaneously on days 1-5 at a dose of 40 mg/m\^2 all trans retinoic acid: 45 mg/m\^2, will be taken orally on days 3-7 of each cycle, divided into two doses
|
'Delayed' 5-AZA+ATRA
n=8 participants at risk
Patients will receive Lupron 7.5 mg x 1. Four weeks later, they will undergo "observation" every 28 days for 3 cycles. Thereafter, patients will receive 5-AZA+ATRA treatment on a 28-day cycle, in the absence of toxicities, for 3 cycles.
Treatment:
5-Azacitidine: subcutaneously on days 1-5 at a dose of 40 mg/m\^2 all trans retinoic acid: 45 mg/m\^2, will be taken orally on days 3-7 of each cycle, divided into two doses
-Taken for 3 cycles
|
|---|---|---|
|
Investigations
Alanine Aminotransferrase increased
|
33.3%
2/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Investigations
Anemia
|
66.7%
4/6 • 24 months
|
37.5%
3/8 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Arthalgia
|
0.00%
0/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Investigations
Aspartate Aminotransferase increased
|
50.0%
3/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Cardiac disorders
Atrial Fibrillation
|
0.00%
0/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Investigations
Cholesterol high
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Gastrointestinal disorders
Constipation
|
16.7%
1/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Investigations
Creatinine increased
|
0.00%
0/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Psychiatric disorders
Depression
|
33.3%
2/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Nervous system disorders
Dizziness
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Vascular disorders
Edema Limbs
|
0.00%
0/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Reproductive system and breast disorders
Erectile dysfunction
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
General disorders
Fatigue
|
33.3%
2/6 • 24 months
|
25.0%
2/8 • 24 months
|
|
Gastrointestinal disorders
Gastroesophageal Reflux Disease
|
33.3%
2/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
General disorders
Generalized muscle weakness
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Eye disorders
Glaucoma
|
0.00%
0/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
General disorders
Headache
|
16.7%
1/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Renal and urinary disorders
Hematuria
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
General disorders
Hot flashes
|
33.3%
2/6 • 24 months
|
25.0%
2/8 • 24 months
|
|
Investigations
Hypercalcemia
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Investigations
Hyperglycemia
|
83.3%
5/6 • 24 months
|
37.5%
3/8 • 24 months
|
|
Investigations
Hyperkalemia
|
0.00%
0/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Metabolism and nutrition disorders
Hyperlipidemia
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Cardiac disorders
Hypertension
|
33.3%
2/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Investigations
Hypoalbuminemia
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Investigations
Hyponatremia
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Immune system disorders
Immune system disorders other
|
0.00%
0/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
General disorders
Insomnia
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Investigations
Lymphocyte count decreased
|
33.3%
2/6 • 24 months
|
37.5%
3/8 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Gastrointestinal disorders
Nausea
|
16.7%
1/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Investigations
Neutrophil count decreased
|
16.7%
1/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
General disorders
Pain
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/6 • 24 months
|
25.0%
2/8 • 24 months
|
|
Skin and subcutaneous tissue disorders
Papulopustular rash
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Reproductive system and breast disorders
Penile pain
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
0.00%
0/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Investigations
Plaelet Count Decreased
|
16.7%
1/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
16.7%
1/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Gastrointestinal disorders
Rectal pain
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Sinusitis
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Renal and urinary disorders
Urinary frequency
|
16.7%
1/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Renal and urinary disorders
Urinary incontinence
|
33.3%
2/6 • 24 months
|
12.5%
1/8 • 24 months
|
|
Infections and infestations
Urinary tract infection
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
16.7%
1/6 • 24 months
|
0.00%
0/8 • 24 months
|
|
Investigations
White blood cell decreased
|
83.3%
5/6 • 24 months
|
37.5%
3/8 • 24 months
|
Additional Information
Vaibhav Patel, MD
Icahn School of Medicine at Mount Sinai
Phone: 212-659-5511
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place