BEAM-101 for Sickle Cell Disease
(BEACON Trial)
Recruiting in Palo Alto (17 mi)
+19 other locations
Age: 18 - 65
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Recruiting
Sponsor: Beam Therapeutics Inc.
Must be taking: Hydroxyurea
Disqualifiers: Moyamoya syndrome, Stroke, others
No Placebo Group
Trial Summary
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
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.
12345Eligibility 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.
I am between 18 and 35 years old, or 12 to 35 with special approval.
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.
+2 more
Trial 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
Participant Groups
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.
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.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Henry Ford Cancer CenterDetroit, MI
University Hospitals Cleveland Medical CenterCleveland, OH
Columbia University Irving Medical CenterNew York, NY
University of MinnesotaMinneapolis, MN
More Trial Locations
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Who Is Running the Clinical Trial?
Beam Therapeutics Inc.Lead Sponsor
References
Safety of hydroxyurea in children with sickle cell anemia: results of the HUG-KIDS study, a phase I/II trial. Pediatric Hydroxyurea Group. [2021]Previous studies have determined the short-term toxicity profile, laboratory changes, and clinical efficacy associated with hydroxyurea (HU) therapy in adults with sickle cell anemia. The safety and efficacy of this agent in pediatric patients with sickle cell anemia has not been determined. Children with sickle cell anemia, age 5 to 15 years, were eligible for this multicenter Phase I/II trial. HU was started at 15 mg/kg/d and escalated to 30 mg/kg/d unless the patient experienced laboratory toxicity. Patients were monitored by 2-week visits to assess compliance, toxicity, clinical adverse events, growth parameters, and laboratory efficacy associated with HU treatment. Eighty-four children were enrolled between December 1994 and March 1996. Sixty-eight children reached maximum tolerated dose (MTD) and 52 were treated at MTD for 1 year. Significant hematologic changes included increases in hemoglobin concentration, mean corpuscular volume, mean corpuscular hemoglobin, and fetal hemoglobin parameters, and decreases in white blood cell, neutrophil, platelet, and reticulocyte counts. Laboratory toxicities typically were mild, transient, and were reversible upon temporary discontinuation of HU. No life-threatening clinical adverse events occurred and no child experienced growth failure. This Phase I/II trial shows that HU therapy is safe for children with sickle cell anemia when treatment was directed by a pediatric hematologist. HU in children induces similar laboratory changes as in adults. Phase III trials to determine if HU can prevent chronic organ damage in children with sickle cell anemia are warranted.
Hydroxyurea in children: present and future. [2013]Children with sickle cell anemia provide the best opportunity to assess the efficacy of hydroxyurea (HU) in preventing complications and progressive organ damage. The possibility of treating infants with sickle cell disease (SCD) to inhibit the development of organ dysfunction may be the most important future use of HU. The possibility even exists that instituting HU in the neonate may stop the fetal-to-adult globin chain switch and thus markedly change the clinical phenotype of SCD. Recent data suggest HU may also be especially beneficial in children not only by increasing hemoglobin F (HbF), but also by altering the adhesive receptors expressed on red blood cells and vascular endothelium, further increasing the possibility that vasculopathy can be prevented. Six pediatric trials that included small numbers of severely ill patients have been reported recently. All patients received relatively standard HU doses. All studies reported a significant improvement in HbF and mean corpuscular volume and a mild to marked increase in hemoglobin. The clinical response to HU in children with SCD seems to be consistent. The National Institutes of Health pediatric multicenter trial should help answer the question of short-term HU toxicity; however, questions remain concerning long-term risks, such as carcinogenesis, gametogenesis, marrow toxicity, growth retardation, and chromosomal damage. Long-term studies are needed to answer these questions. The future treatment of most children with SCD with HU alone or in combination with other agents looks promising, and long-term trials are warranted.
Process and strategies for patient engagement and outreach in the Sickle Cell Disease (SCD) community to promote clinical trial participation. [2023]Sickle cell disease (SCD) is the most common inherited blood disorder in the United States. The Cure Sickle Cell Initiative (CureSCi) was created by the National Heart, Lung and Blood Institute (NHLBI) to improve the lives of people with SCD by accelerating the advancement of safe and promising genetic therapies, engaging the SCD community and healthcare providers, and encouraging collaboration among stakeholders. CureSCi is a collaborative, patient-focused research effort that includes patients at every level of the Initiative. Patient engagement is a key component, particularly during the development of clinical trials.
Clinical complications in severe pediatric sickle cell disease and the impact of hydroxyurea. [2022]More evidence of the safety and effectiveness of hydroxyurea (HU) in community-based cohorts of pediatric patients with sickle cell disease (SCD) are needed. The association of HU with organ-specific clinical complications and adverse events is examined herein.
Use of hydroxyurea in children with sickle cell disease: what comes next? [2017]Hydroxyurea (HU) is the first drug that, under well-organized clinical trials, has shown the potential for altering significantly the clinical severity of sickle cell disease (SCD). The placebo-controlled trial of HU in adult SS hemoglobinopathy patients (the Multicenter Study of Hydroxyurea in Sickle Cell Anemia) reported in May 1995 that HU therapy reduced significantly the frequencies of severe pain episodes, acute chest syndrome, and transfusion. Despite these impressive results, no guidelines have been developed to direct clinicians on the use of HU in adult SCD patients. Small-scale phase II studies in children have reported increases in fetal hemoglobin (HbF) and F-cell levels in response to HU therapy. A larger phase II study, the Pediatric Hydroxyurea Study Group (HUG-KIDS), is under way and is expected to be completed by March 1998. The need for a large-scale placebo-controlled trial in children will be doubtful if no unusual short-term toxicity is demonstrated by HUG-KIDS. Guidelines regarding patient selection, dosing schedules, treatment goals, and short- and long-term monitoring parameters need to be established. The case is made of the organization of a clinical network to register and follow SCD patients treated with HU for long-term toxicity.