BEAM-101 for Sickle Cell Disease

(BEACON Trial)

Recruiting at 19 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

Trial Summary

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.

How is the drug BEAM-101 different from other treatments for sickle cell disease?

BEAM-101 is a novel treatment for sickle cell disease that likely involves genetic therapy, which is different from traditional treatments like hydroxyurea that primarily focus on increasing fetal hemoglobin levels to reduce symptoms. BEAM-101's unique approach may offer a more targeted and potentially curative option compared to existing therapies. ^{1 2 3 4 5}

What is the purpose of this trial?

This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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) 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]

References

1. United States 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. [2021]

2. United States pubmed.ncbi.nlm.nih.gov

Hydroxyurea in children: present and future. [2013]

3. United States 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. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Clinical complications in severe pediatric sickle cell disease and the impact of hydroxyurea. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Use of hydroxyurea in children with sickle cell disease: what comes next? [2017]