BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

Phase 2Active Not RecruitingNCT05456880

Beam Therapeutics Inc.15 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Sickle Cell Disease

Interventions

BEAM-101BIOLOGICAL

Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan

Eligibility

Sex: ALLMin age: 12 YearsMax age: 35 Years

Medical Language ↔ Plain English

Key Inclusion Criteria Include: 1. Age ≥12 years to ≤35 years 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

Locations (19)

University of Alabama at Birmingham

Birmingham, Alabama, United States

Outcomes

Primary Outcomes

Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline

Time frame: 6 months to time of analysis as compared to baseline

Proportion of patients with successful neutrophil engraftment

Time frame: BEAM-101 administration to month 24

Time to neutrophil engraftment

Time frame: BEAM-101 administration to month 24

Time to platelet engraftment

Time frame: BEAM-101 administration to month 24

Transplant-related mortality within 100 days after beam-101 treatment

Time frame: BEAM-101 administration to day 100

Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)

Time frame: BEAM-101 administration through month 24

Secondary Outcomes

Proportion of patients experiencing at least 75% reduction in annualized rate of severe VOCs

Time frame: Month 6 post BEAM-101 treatment to month 24 as compared to baseline

Proportion of patients experiencing no severe VOCs

Time frame: 6 months to time of analysis as compared to baseline

Change in annualized number of hospitalizations for VOCs

Time frame: Month 6 post BEAM-101 treatment to month 24 as compared to baseline

Change in annualized duration of hospitalizations for VOCs

Data from ClinicalTrials.gov

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