Trial Outcomes & Findings for PhII 5-Azacytidine Plus Valproic Acid and Eventually Atra in Intermediate II and High Risk MDS (NCT NCT00439673)

NCT ID: NCT00439673

Last Updated: 2018-08-07

Results Overview

Overall survival

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 62 participants
• Primary outcome timeframe: At 60 months
• Results posted on: 2018-08-07

Participant Flow

Participant milestones

Measure	Study Group
Overall Study STARTED	62
Overall Study COMPLETED	26
Overall Study NOT COMPLETED	36

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

PhII 5-Azacytidine Plus Valproic Acid and Eventually Atra in Intermediate II and High Risk MDS

Baseline characteristics by cohort

Measure	Study Group n=62 Participants
Age, Customized Age	69.6 years n=5 Participants
Sex: Female, Male Female	19 Participants n=5 Participants
Sex: Female, Male Male	43 Participants n=5 Participants
Region of Enrollment Italy	62 participants n=5 Participants

PRIMARY outcome

Timeframe: At 60 months

Overall survival

Outcome measures

Measure	Study Group n=26 Participants
The Primary Objective of the Trial is to Assess the Efficacy of the Combined Use of Valproic Acid (VPA) in Combination With 5-Azacytidine (5-Aza C) in the Treatment of MDS.	8.3 months Interval 3.27 to 21.1

SECONDARY outcome

Timeframe: At 60 months

Outcome measures

Measure	Study Group n=23 Participants
Time to Transformation to AML	53.9 months Interval 39.2 to 66.0

Adverse Events

Study Group

Serious events: 24 serious events

Other events: 0 other events

Deaths: 15 deaths

Serious adverse events

Measure	Study Group n=62 participants at risk Other adverse events were not included in the final study report, thus, we have included in the present form, all serious adverse events and mortality.
Blood and lymphatic system disorders Perineal infiltration	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
General disorders Fever	4.8% 3/62 • Number of events 3 • For 38 months Non-serious adverse events are not reported.
Infections and infestations Bronchopneumonic outbreak	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
Blood and lymphatic system disorders Hemorragic shock	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
Infections and infestations Right pleuropneumonia	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
General disorders Local inflamation	3.2% 2/62 • Number of events 2 • For 38 months Non-serious adverse events are not reported.
General disorders Abscess	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
General disorders Sepsis	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
General disorders Sedation	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
General disorders Fever in neutropenia	6.5% 4/62 • Number of events 4 • For 38 months Non-serious adverse events are not reported.
Respiratory, thoracic and mediastinal disorders Pneumonia	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
Respiratory, thoracic and mediastinal disorders Broncopneumonia	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
General disorders Inflammatory bowel	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
General disorders Bowel abscess	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
Respiratory, thoracic and mediastinal disorders Right lung pneumonia	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
General disorders Hyperpyrexia and hypoxia	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
General disorders Confusion and disorientation	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
General disorders Somnolence and obtundation	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.
Injury, poisoning and procedural complications Head and facial contusion	1.6% 1/62 • Number of events 1 • For 38 months Non-serious adverse events are not reported.

Other adverse events

Adverse event data not reported

Additional Information

Alfonso Piciocchi

GIMEMA

Phone: +39 06.70390521

Email: a.piciocchi@gimema.it

Results disclosure agreements

• Principal investigator is a sponsor employee PIs of participating centers may disclose their centre's results only after the main study publication has been released, so that a single centre experience can be compared to the study overall results and give a more appropriate view of the study.
• Publication restrictions are in place

Restriction type: OTHER