A Study of Single-dose ALXN2050 in Healthy Adults

A Single Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACH-0145228 in Healthy Volunteers

This was a phase 1, first-in-human, single-center, randomized, double-blind (participants and investigator blind, sponsor open) placebo-controlled, single-ascending dose study of ACH-0145228 (ALXN2050) conducted in healthy adult participants.

Study Overview

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

• Study Type: Interventional
• Enrollment (Actual): 28
• Phase: Phase 1

Contacts and Locations

Study Locations

• New Zealand
  • Auckland, New Zealand
    • Clinical Trial Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 21 years to 51 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Key Inclusion Criteria:

• Healthy was defined as having no clinically relevant abnormalities identified by a detailed medical history, physical examination, blood pressure and heart rate measurements, 12-lead ECG, and clinical laboratory tests.
• Had a body mass index of 18 to 30 kilograms (kg)/meter squared with a minimum body weight of 50 kg.
• Female participant of nonchildbearing potential.
• Male participant agreed to abstinence or use of a highly effective form of contraception.

Key Exclusion Criteria:

• Had a history or clinically relevant evidence of significant cardiovascular, pulmonary, hepatic, renal, hematological, gastrointestinal, endocrine, immunologic, dermatologic, neurological, or psychiatric disease.
• Had any condition possibly affecting drug absorption.
• Had a body temperature greater than or equal to 38°Celsius on Day -1 or Day 1, Hour 0; had a history of febrile illness, or other evidence of infection, within 14 days prior to first study drug administration.
• Had a positive urine drug screen at Screening or Day -1; was a current tobacco/nicotine user or smoker; had consumed any alcohol within 72 hours before first study drug administration or had a history of regular alcohol consumption within 6 months of Screening.
• Had participated in a clinical study within 30 days prior to first study drug administration
• Had clinically significant laboratory abnormalities,
• Had donated blood or lost more than 500 milliliters of blood within 3 months prior to first study drug administration; had received a blood transfusion or blood products within 6 months prior to first study drug administration.
• Had a clinically significant history of drug allergy.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1: 40 mg ALXN2050/Placebo; Participants randomized to receive ALXN2050 or placebo on Day 1.	Drug: ALXN2050; Powder-in-capsule (PIC).; Other Names: ACH-0145228 (formerly); ACH-5228; Drug: Placebo; PIC.
Experimental: Cohort 2: 80 mg ALXN2050/Placebo; Participants randomized to receive ALXN2050 or placebo on Day 1.	Drug: ALXN2050; Powder-in-capsule (PIC).; Other Names: ACH-0145228 (formerly); ACH-5228; Drug: Placebo; PIC.
Experimental: Cohort 3: 120 mg ALXN2050/Placebo; Participants randomized to receive ALXN2050 or placebo on Day 1.	Drug: ALXN2050; Powder-in-capsule (PIC).; Other Names: ACH-0145228 (formerly); ACH-5228; Drug: Placebo; PIC.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number Of Participants Experiencing Serious Adverse Events	Day 1 through Day 42
Number Of Participants Experiencing Grade 3 Or 4 Adverse Events (AEs)	Day 1 through Day 42
Number Of Participants Experiencing AEs Leading To Discontinuation From The Study	Day 1 through Day 42
Number Of Participants Experiencing Grade 3 Or 4 Laboratory Abnormalities	Day 1 through Day 42
Number Of Participants Experiencing Treatment-emergent Vital Signs, Physical Examination Results, And Electrocardiogram (ECG) Abnormalities	Day 1 through Day 42

Secondary Outcome Measures

Outcome Measure	Time Frame
Maximum Plasma Concentration (Cmax) Of ALXN2050	Up to 144 hours postdose
Time To Reach The Maximum Plasma Concentration (Tmax) Of ALXN2050	Up to 144 hours postdose
Area Under The Plasma Concentration-time Curve Extrapolated To Infinity (AUC0-inf) Of ALXN2050	Up to 144 hours postdose
Alternative Pathway (AP) Activity As Measured By Wieslab Assay	Up to 144 hours postdose
Plasma Bb Fragment Of Complement Factor B Concentration Over Time	Up to 144 hours postdose

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals
• Collaborators: Achillion, a wholly owned subsidiary of Alexion

Study record dates

Study Major Dates

• Study Start (Actual): November 28, 2017
• Primary Completion (Actual): April 13, 2018
• Study Completion (Actual): April 13, 2018

Study Registration Dates

• First Submitted: September 9, 2021
• First Submitted That Met QC Criteria: September 9, 2021
• First Posted (Actual): September 17, 2021

Study Record Updates

• Last Update Posted (Actual): September 17, 2021
• Last Update Submitted That Met QC Criteria: September 9, 2021
• Last Verified: September 1, 2021

More Information

Terms related to this study

• Keywords: Safety; Pharmacokinetics; Complement; Pharmacodynamics; Factor D Inhibitor; ALXN2050; ACH-0145228
• Other Study ID Numbers: ACH228-001; ACTRN12617001521314 (Other Identifier: Australian New Zealand Clinical Trials Registry); U1111-1203-1371 (Other Identifier: Universal Trial Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No