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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 month 6 post beam-101 treatment to month 24 as compared to baseline

Awards & highlights

BEACON Trial 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.Check my eligibility

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.See study design

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.

BEACON Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ month 6 post beam-101 treatment to month 24 as compared to baseline

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~month 6 post beam-101 treatment to month 24 as compared to baseline

This trial's timeline: 3 weeks for screening, Varies for treatment, and month 6 post beam-101 treatment to month 24 as compared to baseline for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline

Proportion of patients with successful neutrophil engraftment

Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)

+3 more

Secondary outcome measures

Change in RBC transfusions per month and per year for SCD-related indications

Change in annualized duration of hospitalizations for VOCs

Change in annualized number of hospitalizations for VOCs

+9 more

BEACON 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.

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Beam Therapeutics Inc.Lead Sponsor

2 Previous Clinical Trials

1,102 Total Patients Enrolled

Media Library

BEAM-101 (Gene Editing Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05456880 — Phase 1 & 2

Sickle Cell Disease Research Study Groups: BEAM-101

Sickle Cell Disease Clinical Trial 2023: BEAM-101 Highlights & Side Effects. Trial Name: NCT05456880 — Phase 1 & 2

BEAM-101 (Gene Editing Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05456880 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

In what capacity is this clinical experiment recruiting participants?

"Affirmative. The information hosted on clinicaltrials.gov suggests that this medical experiment is actively recruiting participants. It was initially posted in August of 2022 and subsequently edited in November of the same year, with 15 patients required to enroll from two sites."

Answered by AI

Are there any enrollment opportunities remaining in this medical experiment?

"Indeed, the clinicaltrials.gov registry indicates that this study is actively enrolling participants, with 15 individuals sought from two trial sites since 8/30/2022. The listing was last modified on 11/14/2022"

Answered by AI

To what demographic is this trial open?

"This clinical trial is admitting 15 people, aged between 18 and 35 years old with anemia caused by sickle cell. Additionally, enrollees must conform to the following qualifications: they should be at least 12 years of age but not exceed 35; have a documented diagnosis of βS/βS, βS/β0 or βS/β+ genotypes; and display evidence of four severe VOCs in 24 months prior to screening despite receiving hydroxyurea or other supportive care treatments."

Answered by AI

Is this study enrolling participants aged 35 and above?

"To be a eligible participant in this clinical trial, one must fall between 18 and 35 years of age. For children or seniors there are additional studies with 131 trials for the former and 85 for the latter."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2022. "BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05456880.

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