Phase 1 randomized single‑blind placebo‑controlled safety and dosing of BEAM‑103 in healthy adults

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

This study will test a single intravenous dose of BEAM‑103 versus placebo in healthy adults 18–55 to see how safe it is and how the body processes the drug.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 18 Years to 55 Years
• Sex: All
• Sponsor: Beam Therapeutics Inc. (industry)
• Locations: 1 site (London)
• Trial ID: NCT07304791 on ClinicalTrials.gov

What this trial studies

This is a Phase 1, single ascending dose, randomized, single‑blind, placebo‑controlled trial in healthy volunteers that will administer a single IV infusion of BEAM‑103 or placebo and follow participants for up to four months. The trial will collect safety data, pharmacokinetics (how the drug is absorbed, distributed, metabolized, and cleared), pharmacodynamics (biologic effects), hematologic measures, and immunogenicity (antibody responses to the drug). Participants will be screened for normal labs and general good health and must meet narrow BMI and blood count criteria before enrollment. Results will be used to define a therapeutic window and select doses for future patient trials.

Who should consider this trial

Good fit: Ideal candidates are healthy adults aged 18–55 with BMI 18.5–25 kg/m2, normal baseline blood counts and labs, not pregnant or breastfeeding, and without recent investigational drug exposure or major medical conditions.

Not a fit: People with significant medical conditions, abnormal screening labs, outside the age/BMI ranges, pregnant or breastfeeding, or who recently received another investigational agent are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, the trial could identify safe dose ranges and PK/PD profiles that enable later patient trials and safer dosing decisions.

How similar studies have performed: First‑in‑human single ascending dose antibody trials in healthy volunteers are a standard approach and have routinely defined safety, PK/PD, and dosing parameters for later trials.

Eligibility criteria

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.

Where this trial is running

London

• Clinical Study Site — London, United Kingdom (RECRUITING)

Conditions: Healthy Subjects, Healthy Participant Study, ESCAPE, BEAM-103