This is a Phase 1, Randomized, Single-blind, Placebo-controlled Study to Assess the Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of BEAM-103 in Healthy Subjects

December 12, 2025 updated by: Beam Therapeutics Inc.

A Randomized, Single-Blind, Placebo-Controlled, Phase 1 Single Ascending Dose Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of BEAM-103 in Healthy Subjects

The purpose of this Phase I single ascending dose study is to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of BEAM-103 in healthy subjects between the ages of 18 and 55. The main questions the study aims to answer are:

  • Safety and tolerability of BEAM-103
  • The pharmacokinetic (PK) profile and pharmacodynamic (PD) characteristics of EAM-103
  • The effect of BEAM-103 on hematologic parameters
  • To assess the immunogenicity of BEAM-103

Researchers will determine and establish the therapeutic window of the antibody and select optimal doses for future trials in patients.

Subjects will:

  • Be asked to participate in the study for a duration of 4-5 months total
  • Be asked to sign informed consent
  • Be assessed for eligibility
  • Provide medical and medication history
  • Receive a single intravenous infusion of BEAM-103 or placebo on study Day 1 according to their assigned cohort
  • Be followed up to 4 months after infusion

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
30

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

Yes

Description

Inclusion Criteria:

  • Ages 18 to ≤55 years
  • Body mass index (BMI) of 18.5 to 25 kg/m2
  • Morning neutrophil count ≥2.6 × 109 cells/L for Black/African American subjects or ≥3.5 ×109 cells/L for Caucasian/Other subjects
  • Baseline hemoglobin value of ≥14 g/dL for males and ≥12 g/dL for females
  • Baseline platelet count of >150 × 109/L.
  • Subjects must be in general good health without significant medical conditions (based on physical exam, electrocardiogram (ECG), and laboratory test results with no clinically significant deviations), per the investigator's assessment

Exclusion Criteria:

  • Known hypersensitivity to any component of the investigational medicinal product (IMP).
  • Participation in another clinical trial involving treatment with an investigational agent within 90 days of informed consent is prohibited.
  • Positive serum pregnancy test or breastfeeding at screening (female participants).
  • Live virus vaccination within 4 weeks prior to signing informed consent.
  • Any severe or uncontrolled medical condition (eg. severe asthma or severe peanut allergy) that in the opinion of the investigator would put the subject at undue medical risk or impair the ability to interpret study results.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Basic Science
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: BEAM-103 Drug Product
BEAM-103 is a monoclonal antibody that will be administered in single dose ascending dose cohorts as an intravenous infusion through the arm.
Drug: BEAM-103
• BEAM-103 is a monoclonal antibody administered as a single intravenous dose
Placebo Comparator: Placebo
Normal Saline Solution will be administered as a matching place in single dose ascending dose cohorts as an intravenous infusion through the arm.
Drug: Placebo Comparator
• The matching placebo comparator is administered as a single intravenous dose

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
To determine the safety and tolerability of single ascending doses of BEAM-103
Time Frame: from enrollment to the end of study at 5 months
Incidence of treatment-emergent AEs, serious AEs, severe AEs, AEs related to BEAM-103
from enrollment to the end of study at 5 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change in Free Stem Cell Factor (SCF) in Peripheral Blood Over Time
Time Frame: from the start of treatment to the end of study at 5 months
Concentration of SCF in peripheral blood measured in nanograms per milliliter (mL)
from the start of treatment to the end of study at 5 months
Change in Tryptase Level in Peripheral Blood Over Time
Time Frame: from the start of treatment to the end of study at 5 months
Serum tryptase concentration measured in nanograms per milliliter (ng/mL)
from the start of treatment to the end of study at 5 months
Change in Reticulocyte Count in Peripheral Blood Over Time
Time Frame: From enrollment to end of study at 5 months
Absolute reticulocyte count measured in cells per microliter (cells/µL)
From enrollment to end of study at 5 months
Change in Neutrophil Count in Peripheral Blood Over Time
Time Frame: From enrollment to end of study at 5 months
Absolute neutrophil count measured in cells per microliter (cells/µL)
From enrollment to end of study at 5 months
Maximum Observed Plasma Concentration (Cmax)
Time Frame: From enrollment to end of study at 5 months
Cmax will be calculated as the highest observed plasma/blood/serum concentration following drug administration
From enrollment to end of study at 5 months
Area Under the Plasma Concentration-Time Curve to Last Quantifiable Concentration (AUClast)
Time Frame: From enrollment to end of study at 5 months
AUClast will be calculated from time zero to the time of the last quantifiable concentration
From enrollment to end of study at 5 months
Area Under the Plasma Concentration-Time Curve to Infinity (AUC∞)
Time Frame: From enrollment to end of study at 5 months
AUC∞ will be calculated as AUClast plus Clast divided by λz
From enrollment to end of study at 5 months
Terminal Elimination Half-Life (t½)
Time Frame: From enrollment to end of study at 5 months
t½ will be estimated from the terminal elimination phase of the plasma concentration-time curve
From enrollment to end of study at 5 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Beam Therapeutics Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 27, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 29, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 29, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 29, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 12, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 26, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 26, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 12, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2025

More Information

Terms related to this study

Keywords

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • BTX-103-001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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