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Clinical Trials/NCT07304791
NCT07304791
Recruiting
Phase 1

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

Beam Therapeutics Inc.1 site in 1 country30 target enrollmentStarted: October 27, 2025Last updated:
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InterventionsBEAM-103Placebo Comparator

Overview

Phase
Phase 1
Status
Recruiting
Enrollment
30
Locations
1
Primary Endpoint
To determine the safety and tolerability of single ascending doses of BEAM-103
OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Overview

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

Study Design

Study Type
Interventional
Allocation
Randomized
Intervention Model
Sequential
Primary Purpose
Basic Science
Masking
Single (Participant)

Eligibility Criteria

Ages
18 Years to 55 Years (Adult)
Sex
All
Accepts Healthy Volunteers
Yes

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.

Arms & Interventions

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.

Intervention: BEAM-103 (Drug)

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.

Intervention: Placebo Comparator (Drug)

Outcomes

Primary Outcomes

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

Secondary Outcomes

  • 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)
  • Area Under the Plasma Concentration-Time Curve to Last Quantifiable Concentration (AUClast)(From enrollment to end of study at 5 months)
  • Area Under the Plasma Concentration-Time Curve to Infinity (AUC∞)(From enrollment to end of study at 5 months)
  • Terminal Elimination Half-Life (t½)(From enrollment to end of study at 5 months)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (1)

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