BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
Purpose
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
Condition
- Sickle Cell Disease
Eligibility
- Eligible Ages
- Between 18 Years and 35 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
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
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental BEAM-101 |
BEAM-101 manufactured with autologous CD34+ hematopoietic stem cells collected by plerixafor mobilization and edited ex vivo. No maximum dose has been set for BEAM-101; all of the gene edited cells that pass release specifications will be administered to the patient. BEAM 101 will be administered as a single dose by IV infusion. |
|
Recruiting Locations
Birmingham, Alabama 35233
More Details
- Status
- Recruiting
- Sponsor
- Beam Therapeutics Inc.