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

ArmDescriptionAssigned 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.
  • Biological: BEAM-101
    Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan

Recruiting Locations

University of Alabama at Birmingham
Birmingham, Alabama 35233

More Details

Status
Recruiting
Sponsor
Beam Therapeutics Inc.

Study Contact

Medical Information
857-327-8641
clinicalinfo@beamtx.com