Trial Outcomes & Findings for Imatinib for Pain in Sickle Cell Anemia (NCT NCT03997903)
NCT ID: NCT03997903
Last Updated: 2026-01-29
Results Overview
Percent change in Band 3 Phosphorylation tested in red blood cells. Band 3 is a protein found on the membrane of red blood cells.
TERMINATED
PHASE1/PHASE2
7 participants
Change from baseline Band 3 Phosphorylation at 7 months
2026-01-29
Participant Flow
7 met inclusion criteria. 3 of the participants were on treatment arm and 4 were healthy participants.
Participant milestones
| Measure |
Imatinib Intervention
Imatinib Mesylate: The starting dose for subjects will be 340 mg/m2/day with a maximum dose of 600 mg daily.
Patients will receive Imatinib orally once daily for 6 months.
|
Control Group for Blood Draws
Participants who were consented for the purposes of providing blood samples to serve as normal controls for laboratory testing. They did not participate in any other study aspects, nor had data collected related to demographics or laboratory parameters.
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|---|---|---|
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Overall Study
STARTED
|
3
|
4
|
|
Overall Study
COMPLETED
|
1
|
4
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Imatinib for Pain in Sickle Cell Anemia
Baseline characteristics by cohort
| Measure |
Imatinib Intervention
n=3 Participants
Imatinib Mesylate: The starting dose for subjects will be 340 mg/m2/day with a maximum dose of 600 mg daily.
Patients will receive Imatinib orally once daily for 6 months.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=41 Participants
|
|
Age, Categorical
Between 18 and 65 years
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3 Participants
n=41 Participants
|
|
Age, Categorical
>=65 years
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0 Participants
n=41 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=41 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=41 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=41 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=41 Participants
|
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Sickle Cell Genotype: HbSS
|
3 participants
n=41 Participants
|
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Disease Status
|
3 Participants
n=41 Participants
|
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Performance Level
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3 Participants
n=41 Participants
|
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Organ Function
|
3 Participants
n=41 Participants
|
PRIMARY outcome
Timeframe: Change from baseline Band 3 Phosphorylation at 7 monthsPopulation: Baseline samples for 2 of the 3 participants were processed but were not measurable (possible damage during transportation). 2 of the 3 participants did not have month 7 samples collected. 1 was collected by were not measurable (possible damage during transportation).
Percent change in Band 3 Phosphorylation tested in red blood cells. Band 3 is a protein found on the membrane of red blood cells.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: change from baseline microparticle release at 7 monthsPopulation: Change in microparticle release from baseline to 7 months was calculated for the one participant that completed the study
Change in Microparticles released from red blood cells. Microparticles are small vesicles released from red blood cells during aging, stress, or other types of damage that contain various proteins from inside the red blood cell, as well as from its membrane.
Outcome measures
| Measure |
Imatinib Intervention
n=1 Participants
Imatinib Mesylate: The starting dose for subjects will be 340 mg/m2/day with a maximum dose of 600 mg daily.
Patients will receive Imatinib orally once daily for 6 months.
|
|---|---|
|
Change in Amount of Microparticles Released From Red Blood Cells
|
-48 Microparticles per microliter of plasma
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PRIMARY outcome
Timeframe: Change from baseline of percent irreversibly sickled cells at 7 monthsPopulation: Only one participant completed 7 months of the study to be analyzed
Functional RBCs is analyzed by two components, one of which is percent irreversibly sickled cells. The percent of irreversibly sickled cells is measure by ektacytometry. Red blood cells that sickle and then cannot revert back to its normal shape are considered irreversibly sickled, increasing the likelihood of adhesion to vessel walls, as well as breakdown or hemolysis. Understanding the point of susceptibility of sickling helps us at what partial pressure of oxygen the red blood cell will sickle. The higher the partial pressure, the more resistant the red blood cell is to sickling/breakdown.
Outcome measures
| Measure |
Imatinib Intervention
n=1 Participants
Imatinib Mesylate: The starting dose for subjects will be 340 mg/m2/day with a maximum dose of 600 mg daily.
Patients will receive Imatinib orally once daily for 6 months.
|
|---|---|
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Change in Percent Irreversibly Sickled Cells
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0.32 percent
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PRIMARY outcome
Timeframe: Change from baseline of point of susceptibility of sickling at 7 monthsPopulation: Only 1 participant completed 7 months of the study to be analyzed.
Functional RBCs is analyzed by two components, one of which is Change in Point of Susceptibility to Sickling. This is measured by OxygenScan. The point of susceptibility of sickling shows at what partial pressure of oxygen (mmHg) the red blood cell will sickle. The higher the partial pressure, the more resistant the red blood cell is to sickling/breakdown.
Outcome measures
| Measure |
Imatinib Intervention
n=1 Participants
Imatinib Mesylate: The starting dose for subjects will be 340 mg/m2/day with a maximum dose of 600 mg daily.
Patients will receive Imatinib orally once daily for 6 months.
|
|---|---|
|
Change in Point of Susceptibility to Sickling by OxygenScan
|
-6.39 mmHg
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SECONDARY outcome
Timeframe: Assessed monthly from treatment start up to 7 monthsDefined as an acute episode of pain lasting greater than 24 hours, with no medically determined cause other than a vaso-occlusive event that resulted in treatment with oral or parenteral opiate agents and/or parenteral nonsteroidal anti-inflammatory agents. Degree of pain was measured by the standard numerical pain scale (0 being little to no pain, 10 being the worst pain).
Outcome measures
| Measure |
Imatinib Intervention
n=3 Participants
Imatinib Mesylate: The starting dose for subjects will be 340 mg/m2/day with a maximum dose of 600 mg daily.
Patients will receive Imatinib orally once daily for 6 months.
|
|---|---|
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Number of Instances of Vaso-occlusive Crisis (VOC)
|
4 events
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SECONDARY outcome
Timeframe: Assessed monthly from treatment start up to 7 monthsDefined as respiratory distress (hypoxia, shortness of breath, chest pain, tachypnea) with evidence of an infiltrate on chest x-ray Measured with clinical evaluation.
Outcome measures
| Measure |
Imatinib Intervention
n=3 Participants
Imatinib Mesylate: The starting dose for subjects will be 340 mg/m2/day with a maximum dose of 600 mg daily.
Patients will receive Imatinib orally once daily for 6 months.
|
|---|---|
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Number of Instances of Acute Chest Syndrome (ACS)
|
1 event
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SECONDARY outcome
Timeframe: Assessed monthly from treatment start up to 7 monthsPopulation: Data was not analyzed as participants did not utilize the pain diary or were lost-to-follow-up so pain diary data was not obtained.
Defined as oral opioid use. Oral use will be documented in pain diary by patient/guardian and reviewed at each visit.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Assessed monthly from treatment start up to 7 monthsDefined as an emergency room or clinic visit resulting in an inpatient admission or observation for a sickle cell-related event (e.g. vaso-occlusive pain, acute chest syndrome, etc).
Outcome measures
| Measure |
Imatinib Intervention
n=3 Participants
Imatinib Mesylate: The starting dose for subjects will be 340 mg/m2/day with a maximum dose of 600 mg daily.
Patients will receive Imatinib orally once daily for 6 months.
|
|---|---|
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Number of Hospitalizations
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5 hospitalizations
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SECONDARY outcome
Timeframe: Assessed monthly from treatment start up to 7 monthsPopulation: Total number of serious adverse events or adverse events during study participation.
Assessment of toxicities based on clinical and laboratory evaluation
Outcome measures
| Measure |
Imatinib Intervention
n=3 Participants
Imatinib Mesylate: The starting dose for subjects will be 340 mg/m2/day with a maximum dose of 600 mg daily.
Patients will receive Imatinib orally once daily for 6 months.
|
|---|---|
|
Assessment of Toxicities of Imatinib in Patients With Sickle Cell Anemia
|
21 events
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Adverse Events
Imatinib Intervention
Serious adverse events
| Measure |
Imatinib Intervention
n=3 participants at risk
Imatinib Mesylate: The starting dose for subjects will be 340 mg/m2/day with a maximum dose of 600 mg daily.
Patients will receive Imatinib orally once daily for 6 months.
|
|---|---|
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Musculoskeletal and connective tissue disorders
Grade 3 Non Cardiac Chest Pain
|
33.3%
1/3 • Number of events 1 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
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Respiratory, thoracic and mediastinal disorders
Grade 3 Lung Infection
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33.3%
1/3 • Number of events 1 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
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Musculoskeletal and connective tissue disorders
Grade 3 Pain in Extremity
|
33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
Other adverse events
| Measure |
Imatinib Intervention
n=3 participants at risk
Imatinib Mesylate: The starting dose for subjects will be 340 mg/m2/day with a maximum dose of 600 mg daily.
Patients will receive Imatinib orally once daily for 6 months.
|
|---|---|
|
Renal and urinary disorders
Hyperuricemia
|
33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Hepatobiliary disorders
Alkaline Phosphatase Increased
|
33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Endocrine disorders
Hyperglycemia
|
33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Gastrointestinal disorders
Nausea
|
66.7%
2/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
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Blood and lymphatic system disorders
Anemia
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66.7%
2/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
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Metabolism and nutrition disorders
Hypoglycemia
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33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Ear and labyrinth disorders
Nasal Congestion
|
33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Hepatobiliary disorders
Alanine Aminotransferase Increased
|
66.7%
2/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Hepatobiliary disorders
Aspartate Aminotransferase Increased
|
66.7%
2/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Gastrointestinal disorders
Vomiting
|
66.7%
2/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
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Nervous system disorders
Headache
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66.7%
2/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Gastrointestinal disorders
Abdominal Pain
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33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
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Gastrointestinal disorders
Constipation
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33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Hepatobiliary disorders
Hyperbilirubinemia
|
66.7%
2/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Skin and subcutaneous tissue disorders
Rash
|
33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Blood and lymphatic system disorders
Neutropenia
|
33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
|
|
Blood and lymphatic system disorders
Lymphocytosis
|
33.3%
1/3 • Number of events 3 • 3 years
Systematic Assessment through regular investigator assessment and regular laboratory testing. Adverse events were not collected for the control group as they only provided blood samples to serve as controls during shipment/processing.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place