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

Phase 1RecruitingNCT07304791

Beam Therapeutics Inc.30 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

BASIC_SCIENCE

Masking

SINGLE

Enrollment

Conditions

Healthy Subjects Healthy Participant Study

Interventions

Eligibility

Sex: ALLMin age: 18 YearsMax age: 55 YearsHealthy volunteers:

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

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

Time frame: from enrollment to the end of study at 5 months

Secondary Outcomes

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

Concentration of SCF in peripheral blood measured in nanograms per milliliter (mL)

Time frame: from the start of treatment to the end of study at 5 months

Change in Tryptase Level in Peripheral Blood Over Time

Serum tryptase concentration measured in nanograms per milliliter (ng/mL)

Time frame: from the start of treatment to the end of study at 5 months

Change in Reticulocyte Count in Peripheral Blood Over Time

Absolute reticulocyte count measured in cells per microliter (cells/µL)