Summary
This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD
Conditions
Sickle Cell Disease
Recruitment Status
Recruiting
Eligibility Criteria
Key Inclusion Criteria Include:
Age ≥18 years to ≤35 years for the initial sentinel cohort; for subsequent enrollment patients from ≥12 years up to ≤35 years may be enrolled only upon approval by FDA.
Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes.
Severe SCD defined by the occurrence of at least 4 severe VOCs in the 24 months prior to screening despite receiving hydroxyurea or other supportive care measures
Key Exclusion Criteria Include:
HbF levels >20%, obtained at the time of screening on or off hydroxyurea therapy
Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation
Available and willing matched sibling donor
Definitive diagnosis of moyamoya syndrome based on screening brain MRA
History of overt stroke
Intervention
Intervention Type
Intervention Name
Biological
BEAM-101
Phase
Phase 1, Phase 2
Gender
All
Min Age
18 Years
Max Age
35 Years
Download Date
March 13, 2024
Principal Investigator
Primary Investigator: Matthew Heeney, MD
Co-Investigator: David Williams, MD
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Primary Contact Information
For more information on this trial, visit clinicaltrials.gov.
Contact
For more information and to contact the study team: