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Gene Editing Therapy
BEAM-101 for Sickle Cell Disease (BEACON Trial)
Phase 1 & 2
Recruiting
Research Sponsored by Beam Therapeutics Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group
Summary
This trial is testing a new treatment for sickle cell disease, which is a genetic disorder that causes red blood cells to become deformed and break down. The new treatment is called BEAM-101 and it uses a patient's own stem cells that have been edited to fix the genetic mutation that causes sickle cell disease.
Who is the study for?
This trial is for adults and approved children with severe Sickle Cell Disease (SCD) who've had at least 4 serious pain episodes in the last 2 years despite treatment. Candidates must have specific SCD genotypes and be between 12-35 years old, pending FDA approval for minors. Those with a history of transplants, available sibling donors, stroke, moyamoya syndrome or high fetal hemoglobin levels are excluded.
What is being tested?
BEACON study tests BEAM-101 on patients with severe SCD. It's an early-phase trial to see if one's own genetically edited blood-forming cells can safely treat the disease. Participants receive a single infusion of their modified cells after which their health and disease symptoms are monitored.
What are the potential side effects?
Potential side effects aren't detailed but may include reactions related to cell infusion, body's response to genetic modifications or immune responses against edited cells. Close monitoring will identify any adverse effects due to the novel nature of this therapy.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: BEAM-101Experimental Treatment1 Intervention
BEAM-101 manufactured with autologous CD34+ hematopoietic stem cells collected by plerixafor mobilization and edited ex vivo. No maximum dose has been set for BEAM-101; all of the gene edited cells that pass release specifications will be administered to the patient. BEAM 101 will be administered as a single dose by IV infusion.
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Who is running the clinical trial?
Beam Therapeutics Inc.Lead Sponsor
3 Previous Clinical Trials
1,208 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I have been diagnosed with moyamoya syndrome through a brain scan.I have a sibling who matches and is willing to donate.I have had a stem cell or organ transplant before.I have had a stroke before.My HbF levels are above 20%, with or without hydroxyurea treatment.I have severe sickle cell disease with 4 or more pain crises in the last 2 years despite treatment.I have sickle cell disease with a specific genetic makeup.I am between 18 and 35 years old, or 12 to 35 with special approval.
Research Study Groups:
This trial has the following groups:- Group 1: BEAM-101
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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