StudyFinder
MT2022-41: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease and Severe Vaso-Occlusive Crises (BEACON Trial)
Status: Recruiting
BEAM-101 is an experimental new therapy being developed for treating people with SCD and vaso-occlusive crises. The goal of this study is to see if BEAM-101 is safe and effective for people in the study. The study sponsor and study doctors would also like to see if individuals who are treated with BEAM-101 require fewer blood transfusions and experience fewer vasoocclusive crises requiring hospitalization, compared to before they received BEAM-101. This study will also measure the levels of fetal hemoglobin along with measures that assess quality of life and ability to function following treatment with BEAM-101.
Sex: Male or Female
Age Group: 18 years and over
Inclusion Criteria:
• 18 to 35 years old
• documented diagnosis of sickle cell disease with specific genotypes (study staff will review)
• disease is severe
Exclusion Criteria:
• HbF levels >20%, obtained at the time of screening on or off hydroxyurea therapy
• previous transplant
• history of an overt stroke
Interventions:
Biological: BEAM-101
Conditions:
Rare Diseases, Blood Disorders
Keywords:
Clinics and Surgery Center (CSC), SCD, Sickle Cell Disease
Study Contact: Ashish Gupta - gupta461@umn.edu
Principal Investigator: Ashish Gupta
Phase: PHASE1
IRB Number: STUDY00017341
See this study on ClinicalTrials.gov