BEAM-101 for Sickle Cell Disease

(BEACON Trial)

Recruiting at 20 trial locations

Overseen By Medical Information

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Beam Therapeutics Inc.

Must be taking: Hydroxyurea

Disqualifiers: Moyamoya syndrome, Stroke, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 1 day

15 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

The trial aims to test the safety and effectiveness of BEAM-101, a new treatment for individuals with severe sickle cell disease. BEAM-101 employs a special type of stem cell therapy to reduce the painful episodes and complications associated with this condition. The trial targets those who have experienced at least four severe pain crises in the past two years, despite using other treatments like hydroxyurea. Participants will receive a single dose of this experimental treatment through an IV. As a Phase 1 trial, the research focuses on understanding how BEAM-101 functions in people, offering participants the opportunity to be among the first to receive this innovative treatment.

Do I need to stop my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. However, it mentions that participants must have experienced severe symptoms despite receiving hydroxyurea or other supportive care, which suggests you may continue some treatments.

Is there any evidence suggesting that BEAM-101 is likely to be safe for humans?

Research has shown that BEAM-101, a treatment for sickle cell disease, has been studied for safety in humans. Early results indicate that BEAM-101 is generally well-tolerated. Some patients have experienced mild to moderate side effects, while serious side effects have been rare.

The treatment uses a patient's own stem cells, modified to increase fetal hemoglobin. This special type of hemoglobin helps prevent red blood cells from sickling, aiming to reduce sickle cell symptoms. Studies have not yet set a maximum dose, using all safely modified cells available for each patient.

Due to limited data, the trial remains in a phase where researchers closely monitor BEAM-101 for safety. This phase typically assesses the safety of a new treatment and identifies potential risks. While BEAM-101 appears promising, ongoing research will continue to evaluate its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

BEAM-101 is unique because it uses gene-edited autologous hematopoietic stem cells, offering a personalized approach for treating Sickle Cell Disease. Unlike standard treatments like hydroxyurea or blood transfusions, BEAM-101 aims to address the root cause by editing the patient's own stem cells to produce healthy red blood cells. Researchers are excited about its potential to provide a long-term, possibly one-time solution through a single IV infusion, which could significantly improve quality of life for patients.

What evidence suggests that BEAM-101 might be an effective treatment for Sickle Cell Disease?

Research has shown that BEAM-101, the investigational treatment in this trial, holds promise for treating severe sickle cell disease (SCD). In a study with 17 patients, all experienced a significant increase in Hemoglobin F (HbF), a blood protein that helps reduce SCD symptoms, with levels rising above 60%. Meanwhile, Hemoglobin S (HbS), the abnormal protein causing sickle cell problems, dropped to less than 40%. These changes suggest that BEAM-101 can effectively reduce sickle cell issues. The treatment uses genetically modified cells to achieve these results, offering hope for people with severe SCD. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

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.

Exclusion 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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of autologous CD34+ base edited hematopoietic stem cells (BEAM-101) administered by IV infusion

1 day

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

What Are the Treatments Tested in This Trial?

Interventions

• BEAM-101

Trial Overview 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.

How Is the Trial Designed?

Group I: BEAM-101 Experimental Treatment 1 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.

Who Is Running the Clinical Trial?

Beam Therapeutics Inc.

Lead Sponsor

Trials

5

Recruited

1,200+

Published Research Related to This Trial

Hydroxyurea (HU) has been shown to significantly reduce severe pain episodes, acute chest syndrome, and the need for blood transfusions in adult patients with sickle cell disease, based on a well-organized clinical trial.

Despite its efficacy, there are currently no established guidelines for the use of HU in adult sickle cell disease patients, highlighting the need for further research and the development of treatment protocols.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Use of hydroxyurea in children with sickle cell disease: what comes next? Ohene-Frempong, K., Smith-Whitley, K.[2017]

A Phase I/II trial involving 84 children aged 5 to 15 years with sickle cell anemia demonstrated that hydroxyurea (HU) therapy is safe, with no life-threatening adverse events and no instances of growth failure.

HU treatment led to significant positive changes in blood parameters, such as increased hemoglobin and fetal hemoglobin levels, while laboratory toxicities were generally mild and reversible, indicating its potential efficacy in managing sickle cell anemia in pediatric patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety of hydroxyurea in children with sickle cell anemia: results of the HUG-KIDS study, a phase I/II trial. Pediatric Hydroxyurea Group. Kinney, TR., Helms, RW., O'Branski, EE., et al.[2021]

In a study of 523 pediatric patients with sickle cell disease, those treated with hydroxyurea (HU) were more likely to experience organ-specific complications, such as cardiovascular, hepatic, renal, and pulmonary issues, compared to those not treated with HU.

Despite the increased likelihood of complications in the HU-treated group, the study concluded that HU is not associated with the development of serious adverse events, suggesting it can be safely administered to severely ill children with sickle cell disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical complications in severe pediatric sickle cell disease and the impact of hydroxyurea. Tripathi, A., Jerrell, JM., Stallworth, JR.[2022]

Hydroxyurea (HU) shows promise in treating children with sickle cell anemia by significantly increasing fetal hemoglobin (HbF) levels and improving overall hemoglobin, which may help prevent complications and organ damage.

While short-term use of HU appears safe and effective based on recent pediatric trials, concerns about long-term risks such as cancer and growth issues highlight the need for further studies to ensure its safety over time.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hydroxyurea in children: present and future. Vichinsky, EP.[2013]

The Cure Sickle Cell Initiative (CureSCi) actively engages individuals with sickle cell disease (SCD) in the development of genetic therapy clinical trials, with about 50% of patients having previously participated in a trial, highlighting the importance of patient involvement.

Barriers to participation identified by patients include transportation, childcare, and trust in the healthcare system, which have informed recommendations for improving clinical trial design and communication strategies within the initiative.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Process and strategies for patient engagement and outreach in the Sickle Cell Disease (SCD) community to promote clinical trial participation. Byrnes, C., Botello-Harbaum, M., Clemons, T., et al.[2023]

Citations

1. investors.beamtx.com investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-new-data-beacon-phase-12-clinical-0

Beam Therapeutics Announces New Data from BEACON ... Updated Data from 17 Patients Consistent with Previously Presented Data; All Patients Treated with BEAM-101 Achieved Hemoglobin F (HbF) ...

2. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/513/530865/Initial-Results-from-the-BEACON-Clinical-Study-A

Initial Results from the BEACON Clinical Study: A Phase 1/2 ... A Phase 1/2 study evaluating the safety and efficacy of a single dose of autologous CD34+ base edited hematopoietic stem cells (BEAM-101) in patients with ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05456880

NCT05456880 | BEACON: A Study Evaluating the Safety ... BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease (BEACON). ClinicalTrials.gov ID NCT05456880.

4. investors.beamtx.com investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-highlight-new-data-beam-101-program-sickle

Beam Therapeutics to Highlight New Data from BEAM-101 ... BEAM-101 is an investigational genetically modified cell therapy for the treatment of severe sickle cell disease (SCD).

5. cgtlive.com cgtlive.com/view/beam-base-editing-hsc-therapy-beam-101-raises-hbf-levels-patients-sickle-cell-disease

Beam's Base-Editing HSC Therapy BEAM-101 Raises HbF ... All 17 patients treated with BEAM-101 achieved significant increases in HbF levels, exceeding 60%, and a reduction in HbS to less than 40%.

6. astctjournal.org astctjournal.org/article/S2666-6367(25)00062-4/fulltext

Safety and Efficacy of Autologous CD34+ Base Edited ... BEAM-101 is an investigational base edited autologous cell therapy for the treatment (tx) of SCD through upregulation of anti-sickling fetal hemoglobin (HbF).