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NCT Number: NCT07304791

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

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

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Key information

Conditions

Healthy Subjects Healthy Participant Study

Age range

18 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

BEAM-103

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

placebo comparator

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

Primary outcomes

  1. 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

  1. 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)

  2. 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)

  3. 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)

  4. 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)

  5. 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

  6. 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

  7. 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

  8. 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

Sponsors and collaborators

Lead sponsor

Beam Therapeutics Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Dec 26, 2025
Registry last updated
Dec 26, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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