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CASPERKID: Study of Exa-cel in Children with Severe Sickle Cell Disease

About this study

This is a clinical trial to learn about the safety and possible side effects of exagamglogene autotemcel (exa-cel).

Exa-cel is investigational. That means the U.S. Food and Drug Administration has not yet approved it. We want to see if a single dose of exa-cel allows your child’s 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 your child.
  2. We send those cells to a lab. We will change (edit) the DNA of a specific gene in your child’s cells in the lab to create the study product (exa-cel). The technology we use to edit the DNA is called CRISPR/Cas9. It cuts the DNA like scissors. The blood stem cells repair the cut DNA using their natural repair tools. We hope this repair will help your child’s body make more hemoglobin F.
  3. Your child will get an autologous bone marrow transplant using their changed cells. This transplant takes place in the hospital. Your child will take medicine to remove their bone marrow cells and create “room” for the edited cells. We will give the modified cells into your child’s vein with an IV.
  4. We hope that once the changed cells are in the body, the red blood cells will not sickle or cause complications of sickle cell disease..

Eligibility overview

  • 2–11 years old
  • Diagnosis of 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 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease

Study goal:

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

Diagnosis:

Sickle cell disease

Age:

2 to 11 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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