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

NCT07304791 | Recruiting Phase 1

• 1 other identifier: BTX-103-001
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Healthy Subjects; Healthy Participant Study

Keywords

ESCAPE; BEAM-103

Outcome Measures

Primary Outcomes (1)

• To determine the safety and tolerability of single ascending doses of BEAM-103

  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 Outcomes (8)

• Change in Free Stem Cell Factor (SCF) in Peripheral Blood Over Time

  from the start of treatment to the end of study at 5 months

• Change in Tryptase Level in Peripheral Blood Over Time

  from the start of treatment to the end of study at 5 months

• Change in Reticulocyte Count in Peripheral Blood Over Time

  From enrollment to end of study at 5 months

• Change in Neutrophil Count in Peripheral Blood Over Time

  From enrollment to end of study at 5 months

• Maximum Observed Plasma Concentration (Cmax)

  From enrollment to end of study at 5 months

Study Arms (2)

BEAM-103 Drug Product

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

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

Interventions

BEAM-103 DRUG

• BEAM-103 is a monoclonal antibody administered as a single intravenous dose

BEAM-103 Drug Product

Placebo Comparator DRUG

• The matching placebo comparator is administered as a single intravenous dose

Placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Beam Therapeutics Inc.: lead

Study Sites (1)

Clinical Study Site

London, United Kingdom

RECRUITING

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: BASIC SCIENCE
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 29, 2025
• First Posted: December 26, 2025
• Study Start: October 27, 2025
• Primary Completion: January 29, 2026
• Study Completion (Estimated): May 29, 2026
• Last Updated: December 26, 2025

Record last verified: 2025-11

Locations

GB (1)