PK Study of Gefurulimab SC in Healthy Chinese Adult Participants

A Phase 1 Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Immunogenicity of Gefurulimab (ALXN1720) in Healthy Chinese Adults

The primary objective of this study is to characterize the PK properties of a single dose of gefurulimab in healthy Chinese participants.

Study Overview

• Status: Completed
• Conditions: Healthy
• Intervention / Treatment: Drug: gefurulimab

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 1

Contacts and Locations

Study Locations

• China
  • Shanghai, China, 201107
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

• Participants must be 18 to 45 years of age, inclusive, at the time of signing the informed consent.
• Body weight within ≥ 45 kg to < 110 kg and BMI within the range 18 to 30 kg/m2 (inclusive) at Screening and Admission (Day -1).
• Participants who are healthy as determined by medical evaluation with no clinically significant or relevant abnormalities as determined by medical history, physical examination, vital signs, 12-lead ECG, and clinical laboratory evaluation.
• QTcF ≤ 450 msec for male participants and ≤ 460 msec for female participants at Screening and prior to dosing on Day 1.
• Documented vaccination against N meningitidis serotypes A, C, W-135, Y at least 28 days and not more than 3 years prior to dosing on Day 1.

Exclusion Criteria:

• History of any Neisseria meningitidis infection.
• History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, or neurological disorders.
• Abnormal blood pressure as determined by the Investigator.
• History of latent or active TB (Tuberculosis) or exposure to endemic areas.
• Active systemic bacterial, viral, or fungal infection within 14 days prior to dosing.
• History of latent or active TB or exposure to endemic areas within 8 weeks prior to the Screening Visit confirmed by QuantiFERON®-TB test.
• Significant allergies to humanized biologics.
• Clinically significant multiple or severe drug allergies, intolerance to topical corticosteroids, or severe post-treatment hypersensitivity reactions.
• History of clinically significant allergic reaction (eg, anaphylaxis or angioedema) to any product (eg, food, pharmaceutical).
• History of allergy or intolerance to penicillin or cephalosporin.
• Lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.
• Current or chronic history of liver disease.
• Evidence of human immunodeficiency virus (HIV) infection (positive HIV type 1 or type 2 antibody).
• Evidence of hepatitis B infection (positive hepatitis B surface antigen [HBsAg] or positive total hepatitis B core antibody [HBcAb] with negative surface antibody [anti-HBs]), or hepatitis C viral infection (positive HCV RNA).
• Female participants who have a positive pregnancy test at Screening or Admission.
• Positive prestudy drug/alcohol screen; positive result may be repeated once.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: gefurulimab; Participants will receive a single SC dose of gefurulimab in the abdomen on Day 1.	Drug: gefurulimab; Participants will receive gefurulimab subcutaneously (SC).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Maximum Observed Serum Concentration (Cmax) of Gefurulimab	Day 1 predose through Day 92 postdose
Area Under the Plasma Concentration Versus Time Curve From Time 0 Extrapolated to Infinity (AUC0-inf) of Gefurulimab	Day 1 predose through Day 92 postdose

Secondary Outcome Measures

Outcome Measure	Time Frame
Serum Free Complement Component 5 (C5) Concentration	Day 1 predose through Day 92 postdose
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)	Day 1 through Day 92
Number of Participants With Anti-drug Antibodies (ADAs)	Day 1 through Day 92
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Associated With Prefilled Syringe With Needle Safety Device (PFS-SD)	Day 1 through Day 92
Number of Participants With Successful and Failed Dose Administrations	Day 1 through Day 92

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): October 28, 2024
• Primary Completion (Actual): March 19, 2025
• Study Completion (Actual): March 19, 2025

Study Registration Dates

• First Submitted: November 5, 2024
• First Submitted That Met QC Criteria: November 5, 2024
• First Posted (Actual): November 6, 2024

Study Record Updates

• Last Update Posted (Actual): May 1, 2025
• Last Update Submitted That Met QC Criteria: April 30, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Keywords: Chinese; gefurulimab
• Other Study ID Numbers: D6780C00003

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No