Trial Outcomes & Findings for Deferasirox for Treating Patients Who Have Undergone Allogeneic Stem Cell Transplant and Have Iron Overload (NCT NCT01159067)

NCT ID: NCT01159067

Last Updated: 2019-06-18

Results Overview

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

At baseline

Results posted on

2019-06-18

Participant Flow

Participant milestones

Participant milestones
Measure	Arm I Patients receive oral deferasirox once daily for up to 6 months in the absence of unacceptable toxicity.
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Deferasirox for Treating Patients Who Have Undergone Allogeneic Stem Cell Transplant and Have Iron Overload

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm I n=1 Participants Patients receive oral deferasirox once daily for up to 6 months in the absence of unacceptable toxicity.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants

PRIMARY outcome

Timeframe: At baseline

Outcome measures

Outcome measures
Measure	Arm I n=1 Participants Patients receive oral deferasirox once daily for up to 6 months in the absence of unacceptable toxicity.
Number of Patients With Elevated Labile Plasma Iron (LPI) Above Threshold (0.5 Umol/L)	0 Participants

SECONDARY outcome

Timeframe: Assessed through 6 months from the start of treatment

Population: No patient had elevated LPI level above 0.5umol/L at baseline.

In patients with LPI values above threshold 0.5umol/L at baseline, number of patients had LPI suppressed below this value after treatment. Measurement of LPI is done on plasma specimens.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Assessed through 6 months from the start of treatment

Number of patients, whose Serum Ferritin levels are lower than 1500 ng/mL at two consecutive study visits. Serum Ferritin levels are measured at screening (baseline), week 4, 8, 12, 16, 20, 24 and end of study.

Outcome measures

Outcome measures
Measure	Arm I n=1 Participants Patients receive oral deferasirox once daily for up to 6 months in the absence of unacceptable toxicity.
Number of Patients With Serum Ferritin Level Lower Than 1500 ng/mL After Treatment	1 Participants

SECONDARY outcome

Timeframe: Assessed through 6 months from the start of treatment

Population: The enrolled patient's LPI level at baseline is below 0.5 umol/L and she had both LPI and Serum Ferritin levels measured at baseline only. Therefore, no sufficient data to estimate the correlation of LPI with Serum Ferritin.

Both LPI and Serum Ferritin levels are measured at screening (baseline), week 4, 12, 24 and end of study. The correlation between the levels of LPI and Serum Ferritin at screening, week 4, 12, 24 and end of study will be examined and plotted.

Outcome measures

Outcome data not reported

Adverse Events

Arm I

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Arm I n=1 participants at risk Patients receive oral deferasirox once daily for up to 6 months in the absence of unacceptable toxicity.
Blood and lymphatic system disorders Iron overload	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Blood and lymphatic system disorders Neutrophils/granulocytes (ANC/AGC)	100.0% 1/1 • Number of events 2 • From Cycle 1 to Cycle 6
Blood and lymphatic system disorders Platelets	100.0% 1/1 • Number of events 10 • From Cycle 1 to Cycle 6
Cardiac disorders Hypotension	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
General disorders Fatigue (asthenia, lethargy, malaise)	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
General disorders Fever	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
General disorders Rigors/chills	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Skin and subcutaneous tissue disorders Hypopigmentation	100.0% 1/1 • Number of events 6 • From Cycle 1 to Cycle 6
Skin and subcutaneous tissue disorders Ulceration	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Gastrointestinal disorders Anorexia	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Gastrointestinal disorders Nausea	100.0% 1/1 • Number of events 6 • From Cycle 1 to Cycle 6
Gastrointestinal disorders Vomiting	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Gastrointestinal disorders Hemorrhage, GU	100.0% 1/1 • Number of events 5 • From Cycle 1 to Cycle 6
Respiratory, thoracic and mediastinal disorders Hemorrhage, pulmonary/upper respiratory	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Infections and infestations Infection with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)	100.0% 1/1 • Number of events 2 • From Cycle 1 to Cycle 6
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Metabolism and nutrition disorders ALT, SGPT (serum glutamic pyruvic transaminase)	100.0% 1/1 • Number of events 6 • From Cycle 1 to Cycle 6
Metabolism and nutrition disorders AST, SGOT(serum glutamic oxaloacetic transaminase)	100.0% 1/1 • Number of events 7 • From Cycle 1 to Cycle 6
Metabolism and nutrition disorders Albumin, serum-low (hypoalbuminemia)	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Metabolism and nutrition disorders Alkaline phosphatase	100.0% 1/1 • Number of events 6 • From Cycle 1 to Cycle 6
Metabolism and nutrition disorders Creatinine	100.0% 1/1 • Number of events 4 • From Cycle 1 to Cycle 6
Metabolism and nutrition disorders Glucose, serum-high (hyperglycemia)	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Metabolism and nutrition disorders Magnesium, serum-high (hypermagnesemia)	100.0% 1/1 • Number of events 3 • From Cycle 1 to Cycle 6
Metabolism and nutrition disorders Proteinuria	100.0% 1/1 • Number of events 2 • From Cycle 1 to Cycle 6
Metabolism and nutrition disorders Sodium, serum-low (hyponatremia)	100.0% 1/1 • Number of events 2 • From Cycle 1 to Cycle 6
Metabolism and nutrition disorders Uric acid, serum-high (hyperuricemia)	100.0% 1/1 • Number of events 3 • From Cycle 1 to Cycle 6
Musculoskeletal and connective tissue disorders Muscle weakness, generalized or specific area (not due to neuropathy)	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Nervous system disorders Mood alteration	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Nervous system disorders Neuropathy: sensory	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
General disorders Pain	100.0% 1/1 • Number of events 5 • From Cycle 1 to Cycle 6
Respiratory, thoracic and mediastinal disorders Cough	100.0% 1/1 • Number of events 2 • From Cycle 1 to Cycle 6
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6
Respiratory, thoracic and mediastinal disorders Nasal cavity/paranasal sinus reactions	100.0% 1/1 • Number of events 1 • From Cycle 1 to Cycle 6

Additional Information

Dr. Vinod Pullarkat

City of Hope National Medical Center

Phone: 626-256-4673

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place