Study of ALXN2050 in Healthy Adult Participants of Japanese Descent

A Phase 1 Study to Determine the Safety, Tolerability, and Pharmacokinetics of ALXN2050 in Healthy Participants of Japanese Descent

This is a Phase 1 bridging study being conducted to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ALXN2050 after both single- and multiple-dosing in healthy participants of Japanese descent.

Study Overview

• Status: Completed
• Conditions: Healthy
• Intervention / Treatment: Drug: ALXN2050; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Tempe, Arizona, United States, 85283
      • Clinical Trial Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Key Inclusion Criteria:

• Medically healthy with no clinically significant or relevant abnormalities as determined by medical history, physical or neurological examination, vital signs, 12-lead electrocardiogram, screening clinical laboratory profiles.

• Participants must be of Japanese descent defined as:

  • First generation (born to 2 Japanese parents and 4 Japanese grandparents);
  • Born in Japan, and not have lived outside Japan for greater than 5 years;
  • Lifestyle, including diet, must not have significantly changed since leaving Japan.
• Participants must be able to speak, read, and understand the Japanese and English languages.
• Body mass index within the range 18.5 to 30.0 kilograms (kg)/meter squared, inclusive, with a minimum body weight of 50.0 kg at Screening.

Key Exclusion Criteria:

• History of any medical or psychiatric condition or disease that, in the opinion of the Investigator or designee, might limit the participant's ability to complete or participate in this clinical study, confound the results of the study, or pose an additional risk to the participant by their participation in the study.
• History of significant multiple and/or severe allergies.
• Any previous procedure that could alter absorption or excretion of orally administered drugs.
• Participation in another investigational drug or investigational device study within 5 half-lives (if known) or 30 days prior to the first dose of study intervention, whichever is longer.
• Body temperature ≥ 38.0°Celcius, at Screening or prior to the first dose of study intervention.
• History of drug or alcohol abuse within 2 years prior to the first dose of study intervention or positive drugs-of-abuse or alcohol screen at Screening or Day -1; current tobacco/nicotine users or smokers or a positive cotinine test at Screening.
• Donation of whole blood from 3 months prior to the first dose of study intervention, or of plasma from 30 days prior to the first dose of study intervention; receipt of blood products within 6 months prior to the first dose of study intervention.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1: ALXN2050 (Dose 1); Participants will receive ALXN2050 (Dose 1) as follows under fasting conditions: 120-milligrams (mg) single dose, 3-day washout, then 120-mg twice daily (BID) dosing.	Drug: ALXN2050; Oral tablet.; Other Names: ACH-0145228 (formerly)
Experimental: Cohort 1: Placebo (Dose 1); Participants will receive placebo (Dose 1) as follows under fasting conditions: 120-mg placebo single dose, 3-day washout, then 120-mg placebo BID dosing.	Drug: Placebo; Oral tablet.
Experimental: Cohort 2: ALXN2050 (Dose 2); Participants will receive ALXN2050 (Dose 2) as follows under fasting conditions: 180-mg single dose, 3-day washout, then 180-mg BID dosing.	Drug: ALXN2050; Oral tablet.; Other Names: ACH-0145228 (formerly)
Experimental: Cohort 2: Placebo (Dose 2); Participants will receive placebo (Dose 2) as follows under fasting conditions: 180-mg placebo single dose, 3-day washout, then 180-mg placebo BID dosing.	Drug: Placebo; Oral tablet.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number Of Participants With Treatment-emergent Adverse Events	see Time Frame - defined	Day 1 (after first dose) through follow-up (7 +/- 2 days after final dose)
Area Under The Concentration-time Curve From Time Zero To Infinity (AUCinf) For Single-dose ALXN2050	see Time Frame - defined	Up to 72 hours postdose
Maximum Plasma Concentration (Cmax) For Single-dose ALXN2050	see Time Frame - defined	Up to 72 hours postdose
Time To Maximum Plasma Concentration (Tmax) For Single-dose ALXN2050	see Time Frame - defined	Up to 72 hours postdose
Area Under The Concentration-time Curve From Time Zero To The 12-hour Time Point (AUC0-12) For Multiple-dose ALXN2050	see Time Frame - defined	Up to 72 hours postdose
Cmax For Multiple-dose ALXN2050	see Time Frame - defined	Up to 72 hours postdose
Tmax For Multiple-dose ALXN2050	see Time Frame - defined	Up to 72 hours postdose

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Alternative Pathway Activity As Measured By Wieslab Assay	see Time Frame - defined	Up to 72 hours postdose
Complement Factor B Fraction b Levels	see Time Frame - defined	Up to 72 hours postdose

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): July 9, 2021
• Primary Completion (Actual): September 7, 2021
• Study Completion (Actual): September 7, 2021

Study Registration Dates

• First Submitted: June 28, 2021
• First Submitted That Met QC Criteria: June 28, 2021
• First Posted (Actual): July 7, 2021

Study Record Updates

• Last Update Posted (Estimate): December 9, 2022
• Last Update Submitted That Met QC Criteria: December 7, 2022
• Last Verified: December 1, 2022

More Information

Terms related to this study

• Keywords: Pharmacokinetics; Pharmacodynamics; ALXN2050; Japanese Descent
• Other Study ID Numbers: ALXN2050-HV-113

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No