Imatinib for Pain in Sickle Cell Anemia (IMPACT)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03997903|
Recruitment Status : Not yet recruiting
First Posted : June 25, 2019
Last Update Posted : February 12, 2020
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Imatinib Mesylate||Early Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Primary Purpose:||Basic Science|
|Official Title:||IMatinib for PAin in Chronic Treatment of Sickle Cell Anemia (IMPACT SCA): A Pilot Study of Imatinib in Patients With Sickle Cell Anemia and Recurrent Vaso-occlusive Pain|
|Estimated Study Start Date :||February 26, 2020|
|Estimated Primary Completion Date :||September 2021|
|Estimated Study Completion Date :||September 2022|
|Experimental: Imatinib Intervention||
Drug: 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.
- Changes in Biochemical Effects - Band 3 Phosphorylation [ Time Frame: change from baseline Band 3 Phosphorylation at 7 months ]Percent change in Band 3 Phosphorylation tested in red blood cells
- Changes in Biochemical Effects - Microparticle Release [ Time Frame: change from baseline micro particle release at 7 months ]Percent change in Microparticle Release tested in red blood cells
- Change in Functional RBC analysis [ Time Frame: change from baseline functional RBC at 7 months ]Percent Irreversibly Sickled Cells by ektacytometry and Percent altered susceptibility to sickling by OxygenScan
- Vaso-occlusive Crisis (VOC) [ Time Frame: monthly over 7 months ]Defined 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. Measured by pain scale (1-10) or Wong-Baker Faces scale based on age:
- Acute Chest Syndrome (ACS) [ Time Frame: monthly over 7 months ]Defined as respiratory distress (hypoxia, shortness of breath, chest pain, tachypnea) with evidence of an infiltrate on chest x-ray Measured with clinical evaluation.
- Opioid Use [ Time Frame: monthly over 7 months ]Defined as both oral and parenteral opioid use Oral use will be documented in pain diary by patient/guardian and reviewed at each visit.
- Hospitalizations [ Time Frame: monthly over 7 months ]Defined 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).
- Assessment toxicities of imatinib in patients with sickle cell anemia (SCA) [ Time Frame: monthly over 7 months ]Clinical evaluation
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03997903
|Contact: Seethal Jacob, MDemail@example.com|
|United States, Indiana|
|Riley Hospital for Children at IU Health|
|Indianapolis, Indiana, United States, 46202|
|Contact: Anne Bubnick 317-948-0101 firstname.lastname@example.org|
|Principal Investigator: Seethal Jacob, MD|