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

Exploring BEAM-101 for severe sickle cell disease safety and effectiveness.

Recruiting
18-35 years
All
Phase 1/2

BEACON Study is researching a new treatment called BEAM-101 for people with severe Sickle Cell Disease (SCD). SCD is a condition where red blood cells, which carry oxygen, are shaped like a sickle instead of being round. This can cause pain and other health problems. BEAM-101 uses a special kind of cell (CD34+ HSPCs) from the patient’s own body, changes them, and puts them back to help treat the disease. The study is open-label, meaning everyone knows what treatment is being given, and single-arm, meaning everyone gets the same treatment. It is being conducted at multiple centers.

To join, you should:

  • Be between ages 12 to 35.
  • Have severe SCD with certain types of the disease.
  • Have had frequent painful events (at least 4 in the last 2 years) despite treatment.

Some reasons you might not join:

  • High levels of fetal hemoglobin (HbF) at the time of screening.
  • Previous transplants or available sibling donor.
  • History of stroke or certain brain conditions.

The study focuses on safety (no serious side effects) and efficacy (how well it works).

Study details
    Sickle Cell Disease

NCT05456880

Beam Therapeutics Inc.

19 January 2025

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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