A Study of Single-dose ALXN2050 in Healthy Adults

NCT05047458 | Completed Phase 1

• 3 other identifiers: ACH228-001ACTRN12617001521314U1111-1203-1371
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Healthy

Keywords

Factor D Inhibitor; Complement; ALXN2050; ACH-0145228; Pharmacokinetics; Pharmacodynamics; Safety

Outcome Measures

Primary Outcomes (5)

• 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 Outcomes (5)

• 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

Study Arms (3)

Cohort 1: 40 mg ALXN2050/Placebo

EXPERIMENTAL

Participants randomized to receive ALXN2050 or placebo on Day 1.

Drug: ALXN2050; Drug: Placebo

Cohort 2: 80 mg ALXN2050/Placebo

EXPERIMENTAL

Participants randomized to receive ALXN2050 or placebo on Day 1.

Drug: ALXN2050; Drug: Placebo

Cohort 3: 120 mg ALXN2050/Placebo

EXPERIMENTAL

Participants randomized to receive ALXN2050 or placebo on Day 1.

Drug: ALXN2050; Drug: Placebo

Interventions

ALXN2050 DRUG

Powder-in-capsule (PIC).

Also known as: ACH-0145228 (formerly), ACH-5228

Cohort 1: 40 mg ALXN2050/Placebo; Cohort 2: 80 mg ALXN2050/Placebo; Cohort 3: 120 mg ALXN2050/Placebo

Placebo DRUG

PIC.

Cohort 1: 40 mg ALXN2050/Placebo; Cohort 2: 80 mg ALXN2050/Placebo; Cohort 3: 120 mg ALXN2050/Placebo

Eligibility Criteria

• Age: 25 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead
• Achillion, a wholly owned subsidiary of Alexion: collaborator

Study Sites (1)

Clinical Trial Site

Auckland, New Zealand

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: September 9, 2021
• First Posted: September 17, 2021
• Study Start: November 28, 2017
• Primary Completion: April 13, 2018
• Study Completion: April 13, 2018
• Last Updated: September 17, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will share

Locations

NZ (1)