Healthy
Conditions
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.
Interventions
DRUGALXN2030
ALXN2030 will be administered subcutaneously as a single dose either as a manual SC injection or as an SC infusion via a syringe pump.
DRUGPlacebo
Placebo will be administered subcutaneously as a single dose either as a manual SC injection or as an SC infusion via a syringe pump.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
Healthy volunteers
Yes
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.
Design outcomes
Primary
| 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
| 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 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 |
| Number of Participants With Treatment-Emergent Antidrug Antibodies (ADAs) | Days 1 through study completion, an average of 1 year |
Countries
United Kingdom
Outcome results
None listed