Trial Outcomes & Findings for Phase II Study to Evaluate Overall Response in Patients With Higher Risk Myelodysplastic Syndromes (MDS) Treated With Azacitidine With or Without Deferasirox. (NCT NCT02159040)

NCT ID: NCT02159040

Last Updated: 2017-04-04

Results Overview

ORR (inclusive of CR, PR and HI) per IWG 2006 criteria including erythroid response, platelet response and neutrophil response over the course of one year. Hematologic improvement must be maintained for at least 8 weeks in order to count as HI.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 1 participants
• Primary outcome timeframe: 1 year
• Results posted on: 2017-04-04

Participant Flow

Recruitment ended with patient death.Only one patient in trial. No analysis will ever be done.

Participant milestones

Measure	Azacitidine 75mg/m2 7days/28 day cycle
Overall Study STARTED	1
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Azacitidine 75mg/m2 7days/28 day cycle
Overall Study Death	1

Baseline Characteristics

Phase II Study to Evaluate Overall Response in Patients With Higher Risk Myelodysplastic Syndromes (MDS) Treated With Azacitidine With or Without Deferasirox.

Baseline characteristics by cohort

Measure	Azacitidine n=1 Participants 75mg/m2 7days/28 day cycle
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Population: Only one patient in trial. No analysis will ever be done.

ORR (inclusive of CR, PR and HI) per IWG 2006 criteria including erythroid response, platelet response and neutrophil response over the course of one year. Hematologic improvement must be maintained for at least 8 weeks in order to count as HI.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 24 months

Population: Only one patient in trial. No analysis will ever be done.

Time to response is defined as time from the date of the first dose of study treatment to the date of the first documented hematologic improvement.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 24 months

Population: Only one patient in trial. No analysis will ever be done.

Duration of response is defined as time from the date of the first observed hematologic improvement to the date of the first subsequent documented disease progression or relapse per IWG 2006 criteria.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 24 months

Population: Only one patient in trial. No analysis will ever be done.

Progression free survival is defined as time from the date of the first dose of study treatment to the date of the first documented disease progression or relapse per IWG 2006 criteria.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 24 months

Population: Only one patient in trial. No analysis will ever be done.

Overall survival is defined as time from the date of the first dose of study treatment to the date of death from any cause.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 24 months

Population: Only one patient in trial. No analysis will ever be done.

Time to AML transformation is defined as time from the date of the first dose of study treatment to the date of the first documented bone marrow blast count ≥ 20% per WHO classification 1999.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 24 months

Population: Only one patient in trial. No analysis will ever be done.

Change in Serum Ferritin

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 24 months

Population: Only one patient in trial. No analysis will ever be done.

Incidence of adverse events (AEs) overall and by severity, and serious adverse events (SAEs).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 24 months

Population: Only one patient in trial. No analysis will ever be done.

Median number of infections (positive bacterial, viral or fungal culture, or infection requiring IV antimicrobial, or infection resulting in hospitalization or death) in patients treated with azacitidine alone vs. azacitidine + deferasirox

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline

Population: Only one patient in trial. No analysis will ever be done.

Prevalence of the following mutations in the study population (TP53, EZH2, ETV6, RUNX1, ASXL1, other mutation that is present in ≥ 5% of patients)

Outcome measures

Outcome data not reported

Adverse Events

Azacitidine

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Measure	Azacitidine n=1 participants at risk 75mg/m2 7days/28 day cycle
Cardiac disorders Death	100.0% 1/1 • Number of events 1

Other adverse events

Measure	Azacitidine n=1 participants at risk 75mg/m2 7days/28 day cycle
Musculoskeletal and connective tissue disorders Muscle spasms	100.0% 1/1 • Number of events 1
Musculoskeletal and connective tissue disorders Musculoskeletal pain	100.0% 1/1 • Number of events 1
Gastrointestinal disorders Infusion site	100.0% 1/1 • Number of events 1

Additional Information

Clinical Disclosure Office

Novartis Pharmaceuticals

Phone: 862-778-8300

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 from all sites) in the clinical trial.
• Publication restrictions are in place

Restriction type: OTHER