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

NCT05501717 | Active Not Recruiting Phase 1

• 1 other identifier: ALXN2030-HV-101
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Healthy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• 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 Plasma Concentration of Complement Component 3 (C3) Protein

  Baseline (Day 1) and study completion, an average of 1 year

• Change from Baseline in Serum Complement Functional Activity

  Baseline (Day 1) and Day 127

Study Arms (6)

ALXN2030 Dose A

EXPERIMENTAL

ALXN2030 will be administered as a single dose.

Drug: ALXN2030

ALXN2030 Dose B

EXPERIMENTAL

ALXN2030 will be administered as a single dose.

Drug: ALXN2030

ALXN2030 Dose C

EXPERIMENTAL

ALXN2030 will be administered as a single dose.

Drug: ALXN2030

ALXN2030 Dose D

EXPERIMENTAL

ALXN2030 will be administered as a single dose.

Drug: ALXN2030

ALXN2030 Dose E

EXPERIMENTAL

ALXN2030 will be administered as a single dose.

Drug: ALXN2030

Placebo

PLACEBO COMPARATOR

Placebo will be administered as a single dose.

Drug: Placebo

Interventions

ALXN2030 DRUG

ALXN2030 will be administered subcutaneously as a single dose either as a manual SC injection or as an SC infusion via a syringe pump.

ALXN2030 Dose A; ALXN2030 Dose B; ALXN2030 Dose C; ALXN2030 Dose D; ALXN2030 Dose E

Placebo DRUG

Placebo will be administered subcutaneously as a single dose either as a manual SC injection or as an SC infusion via a syringe pump.

Placebo

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (1)

Research Site

Harrow, HA1 3UJ, United Kingdom

Location

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 12, 2022
• First Posted: August 15, 2022
• Study Start: August 16, 2022
• Primary Completion: October 9, 2025
• Study Completion (Estimated): October 14, 2026
• Last Updated: April 9, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

GB (1)