Safety, PK/PD, and Immunogenicity Study of SC ALXN2030 in Healthy Participants
- Registration Number
- NCT05501717
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 48
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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ALXN2030 Dose C ALXN2030 ALXN2030 will be administered as a single dose. ALXN2030 Dose E ALXN2030 ALXN2030 will be administered as a single dose. Placebo Placebo Placebo will be administered as a single dose. ALXN2030 Dose A ALXN2030 ALXN2030 will be administered as a single dose. ALXN2030 Dose B ALXN2030 ALXN2030 will be administered as a single dose. ALXN2030 Dose D ALXN2030 ALXN2030 will be administered as a single dose.
- Primary Outcome Measures
Name Time Method 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
Name Time Method 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
Trial Locations
- Locations (1)
Research Site
🇬🇧Harrow, United Kingdom