Study of ALXN2050 in Healthy Adult Participants of Japanese Descent

NCT04952545 | Completed Phase 1 FDA Drug

• 1 other identifier: ALXN2050-HV-113
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Healthy

Keywords

ALXN2050; Pharmacokinetics; Pharmacodynamics; Japanese Descent

Outcome Measures

Primary Outcomes (7)

• 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 Outcomes (2)

• Alternative Pathway Activity As Measured By Wieslab Assay

  Up to 72 hours postdose

• Complement Factor B Fraction b Levels

  Up to 72 hours postdose

Study Arms (4)

Cohort 1: ALXN2050 (Dose 1)

EXPERIMENTAL

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

Cohort 1: Placebo (Dose 1)

EXPERIMENTAL

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

Cohort 2: ALXN2050 (Dose 2)

EXPERIMENTAL

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

Cohort 2: Placebo (Dose 2)

EXPERIMENTAL

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

Interventions

ALXN2050 DRUG

Oral tablet.

Also known as: ACH-0145228 (formerly)

Cohort 1: ALXN2050 (Dose 1); Cohort 2: ALXN2050 (Dose 2)

Placebo DRUG

Oral tablet.

Cohort 1: Placebo (Dose 1); Cohort 2: Placebo (Dose 2)

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (1)

Clinical Trial Site

Tempe, Arizona, 85283, United States

Location

MeSH Terms

Interventions

rhoA GTP-Binding Protein

Intervention Hierarchy (Ancestors)

rho GTP-Binding Proteins; Monomeric GTP-Binding Proteins; GTP-Binding Proteins; GTP Phosphohydrolases; Acid Anhydride Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Carrier Proteins; Proteins; Amino Acids, Peptides, and Proteins; Intracellular Signaling Peptides and Proteins

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 28, 2021
• First Posted: July 7, 2021
• Study Start: July 9, 2021
• Primary Completion: September 7, 2021
• Study Completion: September 7, 2021
• Last Updated: December 9, 2022

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)