Trial Outcomes & Findings for A Phase II Pilot Study to Assess the Presence of Molecular Factors Predictive for Hematologic Response in Myelodysplastic Syndrome Patients Receiving Deferasirox Therapy. (NCT NCT02663752)
NCT ID: NCT02663752
Last Updated: 2018-08-23
Results Overview
Using next-generation sequencing, gene expression profiling in responder and non-responder patients were to be performed on existing bone marrow aspirate samples. Gene transcription were then to be compared between the two groups and the fold increase/decrease in differentially expressed genes were to be calculated.
TERMINATED
PHASE2
1 participants
18 months
2018-08-23
Participant Flow
Study has been discontinued because of low patient recruitment.
Participant milestones
| Measure |
Deferasirox
All patients are already on commercial deferasirox before entering the study.
|
|---|---|
|
Overall Study
STARTED
|
1
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Phase II Pilot Study to Assess the Presence of Molecular Factors Predictive for Hematologic Response in Myelodysplastic Syndrome Patients Receiving Deferasirox Therapy.
Baseline characteristics by cohort
Baseline data not reported
PRIMARY outcome
Timeframe: 18 monthsPopulation: There was no treatment administration specific to this study. The trial was terminated due to low enrollment. Because of the limited number of data collected an efficacy analysis was not possible.
Using next-generation sequencing, gene expression profiling in responder and non-responder patients were to be performed on existing bone marrow aspirate samples. Gene transcription were then to be compared between the two groups and the fold increase/decrease in differentially expressed genes were to be calculated.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 18 monthsPopulation: There was no treatment administration specific to this study. The trial was terminated due to low enrollment. Because of the limited number of data collected an efficacy analysis was not possible.
The time to response is defined as the time (in months) between the date of deferasirox initiation and the date of the first documented hematological response only in the responder group.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, 18 monthsPopulation: There was no treatment administration specific to this study. The trial was terminated due to low enrollment. Because of the limited number of data collected an efficacy analysis was not possible.
From baseline to time of response (responder group) or time to last follow up (non-responders)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 18 monthsPopulation: There was no treatment administration specific to this study. The trial was terminated due to low enrollment. Because of the limited number of data collected an efficacy analysis was not possible.
Deferasirox dose is defined as the average daily dose (mg/kg/d) given to the patient from treatment initiation to the emergence of hematological response in the responder group or the time of enrollment in the study in the non-responder group.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, 18 monthsPopulation: There was no treatment administration specific to this study. The trial was terminated due to low enrollment. Because of the limited number of data collected an efficacy analysis was not possible.
From baseline to time of response (responder group) or time to last follow up (non-responders)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, 18 monthsPopulation: There was no treatment administration specific to this study. The trial was terminated due to low enrollment. Because of the limited number of data collected an efficacy analysis was not possible.
From baseline to time of response (responder group) or time to last follow up (non-responders)
Outcome measures
Outcome data not reported
Adverse Events
Deferasirox
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data form all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER