NCT03997903

Imatinib for Pain in Sickle Cell Anemia

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

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Indiana University · Academic / Other
• Sex: All
• Age: 18 Years – 25 Years
• Healthy volunteers: Accepted

Summary

In this protocol, the investigators propose to evaluate the biochemical effects of imatinib on sickle red blood cells (RBCs). Patients will be administered imatinib mesylate orally following the guidelines previously established for use of imatinib in other disorders. The biochemical effects of imatinib on sickle RBCs will be examined, including changes in their levels of band 3 tyrosine phosphorylation and the abundances of RBC-derived microparticles in their blood. In addition, the patients will be monitored for symptoms of sickle cell disease (SCD). The investigators expect band 3 tyrosine phosphorylation to decrease dramatically in patients treated with imatinib. The investigators also anticipate a reduction in the numbers of RBC-derived microparticles in circulation (quantitated by assaying the number of glycophorin A positive microparticles in peripheral blood samples by flow cytometry. Most importantly, the investigators expect to see a reduction in the frequency of vaso-occlusive crises, and possibly acute chest syndrome and utilization of opioids. The study duration is planned as 6 months in order to provide adequate time for potential change in the primary endpoints (e.g. percent irreversibly sickled cells).

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2020-02-26
• Primary completion: 2024-08-06
• Completion: 2024-08-06
• First posted: 2019-06-25
• Last updated: 2026-01-29
• Results posted: 2026-01-29

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study