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:
1. 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.
2. Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes.
3. 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:
1. HbF levels \>20%, obtained at the time of screening on or off hydroxyurea therapy
2. Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation
3. Available and willing matched sibling donor
4. Definitive diagnosis of moyamoya syndrome based on screening brain MRA
5. History of overt stroke
Intervention
Intervention Type
Intervention Name
BIOLOGICAL
BEAM-101
Phase
PHASE1, PHASE2
Gender
ALL
Min Age
18 Years
Max Age
35 Years
Download Date
2025-01-10
Principal Investigator
Primary Investigator: Matthew Heeney, MD
Co-Investigator: David Williams, MD
This field has been modified from ClinicalTrials.gov to show a contact specific to Boston Children's.
Primary Contact Information
Matthew.Heeney@childrens.harvard.edu
This field has been modified from ClinicalTrials.gov to show a contact specific to Boston Children's.
For more information on this trial, visit clinicaltrials.gov.
Contact
For more information and to contact the study team: