Trial Outcomes & Findings for Deferasirox in Treating Patients With Very Low, Low, or Intermediate-Risk Red Blood Cell Transfusion Dependent Anemia or Myelodysplastic Syndrome (NCT NCT02943668)

Last Updated: 2020-07-01

Results Overview

As defined by the modified International Working Group (IWG) response criteria: Erythroid response (pretreatment, \<11 g/dL): 1. Hgb increase by ≥ 1.5 g/dL 2. Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk. Only RBC transfusions given for a Hgb of ≤ 0.9 g/dL pretreatment will count in the RBC transfusion response evaluation. Platelet response (pretreatment, \< 100 x 10\^9/L) 1. Absolute increase of ≥ 30 x 10\^9/L for patients starting with \> 20 x 10\^9/L platelets 2. Increase from \< 20 x 10\^9/L to \> 20 x 10\^9/L and by at least 100% Neutrophil response (pretreatment, \< 1.0 x 10\^9/L) 1\) At least 100% increase and an absolute increase \> 0.5 x 10\^9/L

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

At 6 months

Results posted on

2020-07-01

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Deferasirox) Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies
Overall Study STARTED	2
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Deferasirox) Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies
Overall Study Disease Progression	1

Baseline Characteristics

Deferasirox in Treating Patients With Very Low, Low, or Intermediate-Risk Red Blood Cell Transfusion Dependent Anemia or Myelodysplastic Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Deferasirox) n=2 Participants Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies
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	2 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 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	0 Participants n=5 Participants
Race (NIH/OMB) White	2 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
Region of Enrollment United States	2 participants n=5 Participants

PRIMARY outcome

Timeframe: At 6 months

Outcome measures

Outcome measures
Measure	Treatment (Deferasirox) n=2 Participants Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies Of the two participants on the study, none achieved erythroid hematologic improvement as defined by the modified IWG response criteria at 6 months.	Patient 2 Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies
Proportion of Patients That Achieve Erythroid Hematologic Improvement.	0 Participants	—

SECONDARY outcome

Timeframe: Baseline up to 12 months

Population: Data for the 2 participants is presented as 2 different groups to show the individual RBC transfusion requirements per month, at each point in time

Assessed monthly for up to twelve months.

Outcome measures

Outcome measures
Measure	Treatment (Deferasirox) n=1 Participants Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies Of the two participants on the study, none achieved erythroid hematologic improvement as defined by the modified IWG response criteria at 6 months.	Patient 2 n=1 Participants Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies
Change in Red Blood Cell (RBC) Transfusion Requirements Transfusion requirement at Baseline	4 RBC transfusions/month	1 RBC transfusions/month
Change in Red Blood Cell (RBC) Transfusion Requirements Transfusion requirement at time off-treatment	2 RBC transfusions/month	2 RBC transfusions/month

SECONDARY outcome

Timeframe: Baseline up to 12 months

Population: Data for the 2 participants is presented as 2 different groups to show the individual serum ferritin levels at each point in time

Assessed monthly for up to twelve months.

Outcome measures

Outcome measures
Measure	Treatment (Deferasirox) n=1 Participants Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies Of the two participants on the study, none achieved erythroid hematologic improvement as defined by the modified IWG response criteria at 6 months.	Patient 2 n=1 Participants Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies
Change in Serum Ferritin Levels Baseline	6025 ng/mL	755 ng/mL
Change in Serum Ferritin Levels 1 month	6637 ng/mL	778 ng/mL
Change in Serum Ferritin Levels 2 months	6010 ng/mL	827 ng/mL
Change in Serum Ferritin Levels 3 months	6405 ng/mL	583 ng/mL
Change in Serum Ferritin Levels 4 months	22360 ng/mL	414 ng/mL
Change in Serum Ferritin Levels 5 months	7252 ng/mL	652 ng/mL
Change in Serum Ferritin Levels 6 months	5697 ng/mL	776 ng/mL
Change in Serum Ferritin Levels 7 months	4832 ng/mL	880 ng/mL
Change in Serum Ferritin Levels 8 months	4054 ng/mL	NA ng/mL Patient went off-study after 7 months.
Change in Serum Ferritin Levels 9 months	3933 ng/mL	NA ng/mL Patient went off-study after 7 months.
Change in Serum Ferritin Levels 10 months	3728 ng/mL	NA ng/mL Patient went off-study after 7 months.
Change in Serum Ferritin Levels 11 months	NA ng/mL Patient went off-study after 10 months.	NA ng/mL Patient went off-study after 7 months.
Change in Serum Ferritin Levels 12 months	NA ng/mL Patient went off-study after 10 months.	NA ng/mL Patient went off-study after 7 months.

SECONDARY outcome

Timeframe: At 6 months

Outcome measures

Outcome measures
Measure	Treatment (Deferasirox) n=2 Participants Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies Of the two participants on the study, none achieved erythroid hematologic improvement as defined by the modified IWG response criteria at 6 months.	Patient 2 Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies
Proportion of Patients Who Achieve Granulocyte or Platelet Hematologic Improvement	1 Participants	—

Adverse Events

Treatment (Deferasirox)

Serious events: 1 serious events

Other events: 1 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Deferasirox) n=2 participants at risk Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies
Gastrointestinal disorders Constipation	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment

Other adverse events

Other adverse events
Measure	Treatment (Deferasirox) n=2 participants at risk Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Deferasirox: Given PO Laboratory Biomarker Analysis: Correlative studies
Infections and infestations Clostrid Difficile	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Infections and infestations Sinus Infection	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Injury, poisoning and procedural complications Fracture- Lft. Lateral Tibia Plateau	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Injury, poisoning and procedural complications Fall	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Injury, poisoning and procedural complications Fracture- Lft. Second Metatarsal	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Injury, poisoning and procedural complications Fracture- Lft. Wrist	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Gastrointestinal disorders Hemorrhoids	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Gastrointestinal disorders Constipation	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Cardiac disorders A-fib w/ rapid ventricular rate	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Blood and lymphatic system disorders Anemia- due to acute blood loss	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Infections and infestations Urinary tract infection	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Investigations Weight loss	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Psychiatric disorders Insomnia	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Renal and urinary disorders Elevated Creatine/ urine protein	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Investigations Alanine Amniotransferase- increased	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Investigations Asparate Aminotransferase increased	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment
Respiratory, thoracic and mediastinal disorders Dyspnea	50.0% 1/2 • Enrollment through 30 days after the last administration of the study treatment

Additional Information

Dr. Bart Scott

Fred Hutch Cancer Research Center

Phone: 206.667.1990

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place