Current Environment:

Warning

Recall Alert

There is a voluntary recall of Potassium Chloride Extended-Release Capsules. Learn more

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:

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.
Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes.
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:

HbF levels >20%, obtained at the time of screening on or off hydroxyurea therapy
Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation
Available and willing matched sibling donor
Definitive diagnosis of moyamoya syndrome based on screening brain MRA
History of overt stroke

Intervention

Intervention Type

Intervention Name

Biological

BEAM-101

Phase

Phase 1, Phase 2

Gender

All

Min Age

18 Years

Max Age

35 Years

Download Date

May 17, 2024

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

Medical Information

857-327-8641

clinicalinfo@beamtx.com

For more information on this trial, visit clinicaltrials.gov.

Contact

For more information and to contact the study team:

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease NCT05456880 Medical Information 857-327-8641 clinicalinfo@beamtx.com