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BHEEM: Study of BEAM-101 in Patients with Severe Sickle Cell Disease

About this study

This is a clinical trial to learn about the safety and possible side effects of BEAM-101 in patients with severe sickle cell disease (SCD).

BEAM-101 is investigational. That means the U.S. Food and Drug Administration (FDA) has not yet approved it. We want to see if a single dose of BEAM-101 helps your body to:

  • Increase the amount of a type of hemoglobin called hemoglobin F. Hemoglobin is a protein in red blood cells that delivers oxygen to the body’s cells
  • Decrease the effects of SCD

Main steps of the study:

  1. We collect bone marrow cells (stem cells) from you.
  2. We send those cells to a lab. In the lab, we change (edit) the DNA of a specific gene in your cells. This creates a study product called BEAM-101. The technology used to edit the DNA is called base editing. It introduces certain changes to the DNA. We hope this repair will help your body make more hemoglobin F.
  3. You will get an autologous bone marrow transplant using your changed cells. This transplant takes place in the hospital. You will take medicine to remove your bone marrow cells and create “room” for the edited cells. We will put the modified cells into your vein with an IV.
  4. We hope that once the changed cells are in the body, the red blood cells will no longer sickle or cause complications of sickle cell disease.

Eligibility overview

  • 18–35 years old
  • Diagnosed with severe sickle cell disease

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The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.

Overview

Full 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) to Increase Fetal Hemoglobin (HbF) Production in Patients with Severe Sickle Cell Disease

Study goal:

The main goal of this study is to see if a single dose of BEAM-101 allows your body to increase the amount of hemoglobin F while reducing the painful effects of sickle cell disease.

Diagnosis:

Sickle cell disease

Age:

18 to 35 years old

Clinical trials categories:

Blood Disorders Sickle Cell Disease

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

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