Trial Outcomes & Findings for Massive Iron Deposit Assessment (NCT NCT01572922)

NCT ID: NCT01572922

Last Updated: 2019-06-11

Results Overview

Hepatic iron content in the liver using liver biopsy

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 142 participants
• Primary outcome timeframe: up to 30 days after MRI
• Results posted on: 2019-06-11

Participant Flow

Patients within the St. Jude Network (St. Jude Children's Research Hospital, St. Jude Domestic Affiliates, and the adult Hematology and Oncology program at the University of Tennessee Health Sciences Center) who have history of 12 or more lifetime erythrocyte transfusions, and need for liver iron content assessment.

One arm study

Participant milestones

Measure	Arm 1 All eligible patients with history of 12 or more lifetime erythrocyte transfusions, and need for liver iron content assessment.
Overall Study STARTED	142
Overall Study COMPLETED	139
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	Arm 1 All eligible patients with history of 12 or more lifetime erythrocyte transfusions, and need for liver iron content assessment.
Overall Study Adverse Event	1
Overall Study Protocol Violation	1
Overall Study Death	1

Baseline Characteristics

Massive Iron Deposit Assessment

Baseline characteristics by cohort

Measure	Arm 1 n=142 Participants All eligible patients with history of 12 or more lifetime erythrocyte transfusions, and need for liver iron content assessment
Age, Categorical <=18 years	85 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	57 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Age, Continuous	15.9 years n=93 Participants
Sex: Female, Male Female	79 Participants n=93 Participants
Sex: Female, Male Male	63 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	8 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	131 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=93 Participants
Race/Ethnicity, Customized Asian	10 Participants n=93 Participants
Race/Ethnicity, Customized Black or African American	85 Participants n=93 Participants
Race/Ethnicity, Customized White	40 Participants n=93 Participants
Race/Ethnicity, Customized More than one race	1 Participants n=93 Participants
Race/Ethnicity, Customized Unknown or Not Reported	6 Participants n=93 Participants
Region of Enrollment United States	142 Participants n=93 Participants

PRIMARY outcome

Timeframe: up to 30 days after MRI

Population: Eligible iron-overloaded patients with both liver biopsy measurement and 1.5T R2\*-UTE measurement.

Hepatic iron content in the liver using liver biopsy

Outcome measures

Measure	Iron-overloaded Patients n=41 Participants Eligible iron-overloaded patients with both liver biopsy measurement and 1.5T R2\*-UTE measurement
Hepatic Iron Content in the Liver Using Liver Biopsy	19.8 mcg Interval 0.1 to 45.0

PRIMARY outcome

Timeframe: Up to 30 days after MRI

Population: Eligible iron-overloaded patients with both liver biopsy measurement and 1.5T R2\*-UTE measurement.

Hepatic iron content of the liver using MRI-derived 1.5T R2*-UTE measurement, with results in Hz. R2* is a measure obtained with MRI, i.e., MRI R2*. It is measured in hertz (Hz). In lay terms, the MRI machine picks up a signal back from the tissue during the process of scanning the tissues. With every "picture taken", this signal is strong in the beginning and then wanes off. R2* reflects how fast the signal wanes off. If there is too much iron in the tissue, the signal disappears faster, making the T2* value low. T2* is the reciprocal of R2* (R2*= 1/T2*). So, if the signal drops fast, the T2* is low and the R2* is high. In this study, we are measuring the R2* value. The higher the R2*, the more iron in the liver tissue. We can compare the R2* value with that of a liver biopsy to then use the R2* value to tell us how much iron is in the liver without having to biopsy the liver.

Outcome measures

Measure	Iron-overloaded Patients n=41 Participants Eligible iron-overloaded patients with both liver biopsy measurement and 1.5T R2\*-UTE measurement
MRI-derived R2* Values Using 1.5T UTE Technique	864.4 Hz Interval 45.4 to 2245.5

SECONDARY outcome

Timeframe: Up to 30 days after MRI

Population: Iron-overloaded patients had 1.5T R2\*-GRE measurements.

MRI-derived R2* Using 1.5T GRE Technique in Hz. R2* is a measure obtained with MRI, i.e., MRI R2*. It is measured in hertz (Hz). In lay terms, the MRI machine picks up a signal back from the tissue during the process of scanning the tissues. With every "picture taken", this signal is strong in the beginning and then wanes off. R2* reflects how fast the signal wanes off. If there is too much iron in the tissue, the signal disappears faster, making the T2* value low. T2* is the reciprocal of R2* (R2*= 1/T2*). So, if the signal drops fast, the T2* is low and the R2* is high. In this study, we are measuring the R2* value. The higher the R2*, the more iron in the liver tissue. We can compare the R2* value with that of a liver biopsy to then use the R2* value to tell us how much iron is in the liver without having to biopsy the liver.

Outcome measures

Measure	Iron-overloaded Patients n=139 Participants Eligible iron-overloaded patients with both liver biopsy measurement and 1.5T R2\*-UTE measurement
MRI-derived R2* Using 1.5T GRE Technique	333.8 Hz Interval 33.6 to 2726.3

SECONDARY outcome

Timeframe: up to 30 days after MRI

Population: Iron-overloaded patients had 1.5T R2\*-UTE-measurement.

MRI-derived R2* value using 1.5T R2*-UTE in Hz. R2* is a measure obtained with MRI, i.e., MRI R2*. It is measured in hertz (Hz). In lay terms, the MRI machine picks up a signal back from the tissue during the process of scanning the tissues. With every "picture taken", this signal is strong in the beginning and then wanes off. R2* reflects how fast the signal wanes off. If there is too much iron in the tissue, the signal disappears faster, making the T2* value low. T2* is the reciprocal of R2* (R2*= 1/T2*). So, if the signal drops fast, the T2* is low and the R2* is high. In this study, we are measuring the R2* value. The higher the R2*, the more iron in the liver tissue. We can compare the R2* value with that of a liver biopsy to then use the R2* value to tell us how much iron is in the liver without having to biopsy the liver.

Outcome measures

Measure	Iron-overloaded Patients n=139 Participants Eligible iron-overloaded patients with both liver biopsy measurement and 1.5T R2\*-UTE measurement
MRI Derived R2* Using 1.5T UTE Technique	319.2 HZ Interval 45.2 to 2245.5

SECONDARY outcome

Timeframe: Up to 30 days after MRI

Population: 1.5T R2\*-UTE from eligible patients who had 1.5T R2\*-UTE, serum iron, and transferrin saturation measurements.

MRI-derived R2* value using 1.5T R2*-UTE in Hz for patients who have had serum iron and transferrin saturation measurements. R2* is a measure obtained with MRI, i.e., MRI R2*. It is measured in hertz (Hz). In lay terms, the MRI machine picks up a signal back from the tissue during the process of scanning the tissues. With every "picture taken", this signal is strong in the beginning and then wanes off. R2* reflects how fast the signal wanes off. If there is too much iron in the tissue, the signal disappears faster, making the T2* value low. T2* is the reciprocal of R2* (R2*= 1/T2*). So, if the signal drops fast, the T2* is low and the R2* is high. In this study, we are measuring the R2* value. The higher the R2*, the more iron in the liver tissue. We can compare the R2* value with that of a liver biopsy to then use the R2* value to tell us how much iron is in the liver without having to biopsy the liver.

Outcome measures

Measure	Iron-overloaded Patients n=104 Participants Eligible iron-overloaded patients with both liver biopsy measurement and 1.5T R2\*-UTE measurement
R2* Using 1.5T UTE Technique for Patients With Serum Iron and Transferrin Saturation Measurements	340.8 Hz Interval 45.2 to 2245.5

SECONDARY outcome

Timeframe: Up to 30 days after MRI

Population: Iron-overloaded patients had 1.5T R2\*-UTE and serum iron and transferrin saturation measurements.

Serum iron measurements from eligible patients had 1.5T R2*-UTE and serum iron and transferrin saturation measurements.

Outcome measures

Measure	Iron-overloaded Patients n=104 Participants Eligible iron-overloaded patients with both liver biopsy measurement and 1.5T R2\*-UTE measurement
Serum Iron Measurements Compared With 1.5T R2* UTE	156.5 ug/dL Interval 35.0 to 311.0

SECONDARY outcome

Timeframe: Up to 30 days after MRI

Population: Iron-overloaded patients had 1.5T R2\*-UTE and serum iron and transferrin saturation measurements.

Iron Transferrin Saturation in % measurements Transferrin Saturation measurements from eligible patients had 1.5T R2*-UTE and serum iron and transferrin saturation measurements.

Outcome measures

Measure	Iron-overloaded Patients n=104 Participants Eligible iron-overloaded patients with both liver biopsy measurement and 1.5T R2\*-UTE measurement
Transferrin Saturation Measurements	71 percentage Interval 12.0 to 157.0

Adverse Events

Iron-overloaded Patients

Serious events: 1 serious events

Other events: 2 other events

Deaths: 1 deaths

Serious adverse events

Measure	Iron-overloaded Patients n=142 participants at risk Eligible iron-overloaded patients with both liver biopsy measurement and 1.5T R2\*-UTE measurement
Gastrointestinal disorders Infections and infestations	0.70% 1/142 • Number of events 1 • Time frame for adverse event reporting is 10 days following the last study procedure for eligible patients.

Other adverse events

Measure	Iron-overloaded Patients n=142 participants at risk Eligible iron-overloaded patients with both liver biopsy measurement and 1.5T R2\*-UTE measurement
Psychiatric disorders Anxiety	0.70% 1/142 • Number of events 1 • Time frame for adverse event reporting is 10 days following the last study procedure for eligible patients.
Gastrointestinal disorders abdominal pain	0.70% 1/142 • Number of events 1 • Time frame for adverse event reporting is 10 days following the last study procedure for eligible patients.

Additional Information

Jane Hankins, MD

St. Jude Children's Research Hospital

Phone: 866-278-5833

Email: Jane.Hankins@stjude.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place