Safety, PK/PD, and Immunogenicity Study of SC ALXN2030 in Healthy Participants

A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Subcutaneous ALXN2030 in Healthy Participants

The primary purpose of this study is to assess the safety and tolerability of single ascending doses of ALXN2030 in healthy participants.

Study Overview

• Status: Active, not recruiting
• Conditions: Healthy
• Intervention / Treatment: Drug: ALXN2030; Drug: Placebo

Detailed Description

Approximately 48 healthy adult participants (36 participants will be on ALXN2030 and 12 participants will be on placebo) are expected to be enrolled.

• Study Type: Interventional
• Enrollment (Actual): 48
• Phase: Phase 1

Contacts and Locations

Study Locations

• United Kingdom
  • Harrow, United Kingdom, HA1 3UJ
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 60 years (Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

• Healthy participants
• QTcF ≤ 450 msec at Screening and on admission (ie, on Day -1).
• Participants of Japanese descent are defined as: First generation (born to 2 Japanese parents and 4 Japanese grandparents).
• Participants of Japanese descent must be between 20 and 60 years of age.
• BMI within the range 18-32kg/m2 (inclusive) at Screening.

Exclusion Criteria:

• Current or recurrent disease
• Current or relevant history of physical or psychiatric illness.
• Any other significant disease or disorder that, in the opinion of the Investigator, may put the participant at risk.
• Female participants who are pregnant or breastfeeding.
• Major surgery or hospitalization within 90 days prior to dosing on Day1.
• History of allergy or hypersensitivity to an oligonucleotide or GalNAc moiety or any excipients of ALXN2030.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Single Group Assignment
• Masking: Quadruple

Number of Arms

6

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ALXN2030 Dose A; ALXN2030 will be administered as a single dose.	Drug: ALXN2030; ALXN2030 will be administered subcutaneously as a single dose either as a manual SC injection or as an SC infusion via a syringe pump.
Experimental: ALXN2030 Dose B; ALXN2030 will be administered as a single dose.	Drug: ALXN2030; ALXN2030 will be administered subcutaneously as a single dose either as a manual SC injection or as an SC infusion via a syringe pump.
Experimental: ALXN2030 Dose C; ALXN2030 will be administered as a single dose.	Drug: ALXN2030; ALXN2030 will be administered subcutaneously as a single dose either as a manual SC injection or as an SC infusion via a syringe pump.
Experimental: ALXN2030 Dose D; ALXN2030 will be administered as a single dose.	Drug: ALXN2030; ALXN2030 will be administered subcutaneously as a single dose either as a manual SC injection or as an SC infusion via a syringe pump.
Experimental: ALXN2030 Dose E; ALXN2030 will be administered as a single dose.	Drug: ALXN2030; ALXN2030 will be administered subcutaneously as a single dose either as a manual SC injection or as an SC infusion via a syringe pump.
Placebo Comparator: Placebo; Placebo will be administered as a single dose.	Drug: Placebo; Placebo will be administered subcutaneously as a single dose either as a manual SC injection or as an SC infusion via a syringe pump.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)	Day 1 through through study completion, an average of 1 year

Secondary Outcome Measures

Outcome Measure	Time Frame
Maximum Observed Plasma Concentration (Cmax)	Days 1 (predose; end of infusion (EOI); and 0.25, 0.5, 1, 2, 4, 6, 8, and 12 hours post-EOI), 2, 3, 5, 8, 15, 22, 29, 36, 43, 50, 57, 71, 85, 99, and 127
Time to Maximum Observed Plasma Concentration (Tmax)	Days 1 (predose; EOI; and 0.25, 0.5, 1, 2, 4, 6, 8, and 12 hours post-EOI), 2, 3, 5, 8, 15, 22, 29, 36, 43, 50, 57, 71, 85, 99, and 127
Area Under the Plasma Concentration Versus Time Curve From Time 0 (Dosing) to the Last Quantifiable Concentration (AUCt)	Days 1 (predose; EOI; and 0.25, 0.5, 1, 2, 4, 6, 8, and 12 hours post-EOI), 2, 3, 5, 8, 15, 22, 29, 36, 43, 50, 57, 71, 85, 99, and 127
Change from Baseline in Serum Complement Functional Activity	Baseline (Day 1) and Day 127
Change from Baseline in Plasma Concentration of Complement Component 3 (C3) Protein	Baseline (Day 1) and study completion, an average of 1 year
Number of Participants With Treatment-Emergent Antidrug Antibodies (ADAs)	Days 1 through study completion, an average of 1 year

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): August 16, 2022
• Primary Completion (Actual): October 9, 2025
• Study Completion (Estimated): October 14, 2026

Study Registration Dates

• First Submitted: August 12, 2022
• First Submitted That Met QC Criteria: August 12, 2022
• First Posted (Actual): August 15, 2022

Study Record Updates

• Last Update Posted (Actual): April 9, 2026
• Last Update Submitted That Met QC Criteria: April 7, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Other Study ID Numbers: ALXN2030-HV-101

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