Clinical trial NCT05501717: Safety, PK/PD, and Immunogenicity Study of SC ALXN2030 in Healthy Participants
Official title: A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Subcutaneous ALXN2030 in Healthy Participants
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
Conditions: Healthy
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2022-08-15
Enrollment: 48
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
timeFrame: Day 1 through through study completion, an average of 1 year
Secondary outcomes:
measure: Maximum Observed Plasma Concentration (Cmax)
timeFrame: 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
measure: Time to Maximum Observed Plasma Concentration (Tmax)
timeFrame: 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
measure: Area Under the Plasma Concentration Versus Time Curve From Time 0 (Dosing) to the Last Quantifiable Concentration (AUCt)
timeFrame: 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
measure: Change from Baseline in Plasma Concentration of Complement Component 3 (C3) Protein
timeFrame: Baseline (Day 1) and study completion, an average of 1 year
measure: Change from Baseline in Serum Complement Functional Activity
timeFrame: Baseline (Day 1) and Day 127
measure: Number of Participants With Treatment-Emergent Antidrug Antibodies (ADAs)
timeFrame: Days 1 through study completion, an average of 1 year
Other outcomes:
Locations:
city: Harrow
country: United Kingdom
facility: Research Site
Study officials:
References: