Clinical trial NCT05456880: BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
Official title: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients With Sickle Cell Disease and Severe Vaso-Occlusive Crises (Beacon Trial)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Sickle Cell Disease
Keywords: Gene Editing
Sickle Cell
Severe Sickle Cell
Lead sponsor: Beam Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2022-07-13
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Maximum age (years): 35
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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


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

Primary outcomes:
measure: Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline
timeFrame: 6 months to time of analysis as compared to baseline
measure: Proportion of patients with successful neutrophil engraftment
timeFrame: BEAM-101 administration to month 24
measure: Time to neutrophil engraftment
timeFrame: BEAM-101 administration to month 24
measure: Time to platelet engraftment
timeFrame: BEAM-101 administration to month 24
measure: Transplant-related mortality within 100 days after beam-101 treatment
timeFrame: BEAM-101 administration to day 100
measure: Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)
timeFrame: BEAM-101 administration through month 24
Secondary outcomes:
measure: Proportion of patients experiencing at least 75% reduction in annualized rate of severe VOCs
timeFrame: Month 6 post BEAM-101 treatment to month 24 as compared to baseline
measure: Proportion of patients experiencing no severe VOCs
timeFrame: 6 months to time of analysis as compared to baseline
measure: Change in annualized number of hospitalizations for VOCs
timeFrame: Month 6 post BEAM-101 treatment to month 24 as compared to baseline
measure: Change in annualized duration of hospitalizations for VOCs
timeFrame: Month 6 post BEAM-101 treatment to month 24 as compared to baseline
measure: Change in RBC transfusions per month and per year for SCD-related indications
timeFrame: Month 2 post BEAM-101 treatment to month 24 as compared to baseline
measure: Change in total Hgb (g/dL) concentration over time
timeFrame: Baseline to month 24
measure: Proportion of patients with HbF ≥30%, for at least 3 months
timeFrame: Month 6 post BEAM-101 treatment to month 24 as compared to baseline
measure: Change in lactate dehydrogenase (LDH) over time
timeFrame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline
measure: Change in total bilirubin over time
timeFrame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline
measure: Change in free Hgb over time
timeFrame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline
measure: Change in haptoglobin over time
timeFrame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline
measure: Change in reticulocyte count over time
timeFrame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital at Standford
state: California
city: Jacksonville
country: United States
facility: Mayo Clinic Florida
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital
state: Georgia
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Detroit
country: United States
facility: Henry Ford Cancer Center
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine in St. Louis
state: Missouri
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
city: Cleveland
country: United States
facility: The Cleveland Clinic Foundation
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Memphis
country: United States
facility: St Jude Children's Research Hospital
state: Tennessee
city: Nashville
country: United States
facility: The Children's Hospital at TriStar Centennial
state: Tennessee
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
References: