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

Phase 1Active Not RecruitingNCT05501717

Alexion Pharmaceuticals, Inc.48 enrolled

Overview

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

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

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Healthy

Interventions

ALXN2030DRUG

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

PlaceboDRUG

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

Eligibility

Sex: ALLMin age: 18 YearsMax age: 60 YearsHealthy volunteers:

Medical Language ↔ Plain English

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.

Locations (1)

Research Site

Harrow, United Kingdom

Outcomes

Primary Outcomes

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)

Time frame: Day 1 through through study completion, an average of 1 year

Secondary Outcomes

Maximum Observed Plasma Concentration (Cmax)

Time frame: 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)

Time frame: 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)

Time frame: 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

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

Change from Baseline in Serum Complement Functional Activity

Time frame: Baseline (Day 1) and Day 127

Number of Participants With Treatment-Emergent Antidrug Antibodies (ADAs)

Time frame: Days 1 through study completion, an average of 1 year

Data from ClinicalTrials.gov

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