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

Overview

Información sobre este estudio

The purpose of this study is to evaluate the safety and efficacy of a single dose of ex vivo base-edited, autologous CD34+ human hematopoietic stem and progenitor cells (HSPCs) in patients with SCD and severe VOCs. Like allogenic HSCT, these therapies are designed to replace the patient’s bone marrow (BM) with hematopoietic stem cells (HSCs) that encode nonsickling globins. However, as an autologous therapy, BEAM-101 (the IMP in this study) has the potential advantages of eliminating the morbidity and mortality of graft-versus-host disease (GVHD) and greatly broadening access to the majority of patients who lack a matched sibling donor.

BEAM-101 utilizes base editing to introduce specific point mutations that are designed to induce the expression of therapeutic, nonsickling hemoglobin (Hb). BEAM-101 introduces point mutations in the HBG1 and HBG2 promoters, which mimic naturally occurring mutations that disrupt binding of B-cell lymphoma/leukemia 11A gene (BCL11A), a transcriptional repressor of fetal hemoglobin (HbF). Individuals with these mutations have the condition known as hereditary persistence of fetal hemoglobin (HPFH) and continue to have nonsickling HbF expression throughout their life.

Elegibilidad para la participación

Los requisitos de elegibilidad de los participantes incluyen la edad, el sexo, el tipo y el estadio de la enfermedad, y los problemas de salud o tratamientos previos. Las pautas difieren de un estudio a otro e identifican quiénes pueden o no pueden participar. No hay garantía de que cada persona elegible que desee participar en un ensayo se inscribirá. Comunícate con el equipo del estudio para analizar la elegibilidad del estudio y la posible participación.

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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 Magnetic Resonance Angiography (MRA).
  • History of overt stroke.

Eligibility last updated 4/27/23. Questions regarding updates should be directed to the study team contact.

 

Sedes participantes de Mayo Clinic

Los estatus de los estudios cambian con frecuencia. Comunícate con el equipo del estudio para obtener la información más actualizada acerca de la posibilidad de participar.

Sede de Mayo Clinic Estatus Contacto

Jacksonville, Fla.

Investigador principal de Mayo Clinic

Ernesto Ayala, M.D.

Abierto para la inscripción

Contact information:

Clinical Studies Unit

(904) 953-2255

More information

Publicaciones

Publications are currently not available

Otros temas en investigación

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CLS-20552763

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