A Study of Multiple Doses of ALXN2050 in Healthy Adults

NCT05047484 | Completed Phase 1

• 1 other identifier: ACH228-002
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

Brief Summary

This was a Phase 1, placebo-controlled, randomized, double-blind (participant and investigator blind, sponsor open), multiple-ascending dose study conducted in healthy participants to demonstrate the safety and tolerability and to evaluate the pharmacokinetics and pharmacodynamics of ACH-0145228 (ALXN2050).

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 (8)

• Maximum Steady-state Plasma Concentration (Cmax,ss) Of Multiple-dose ALXN2050

  Up to 168 hours postdose

• Time To Reach Maximum Steady-state Plasma Concentration (Tmax,ss) Of Multiple-dose ALXN2050

  Up to 168 hours postdose

• Area Under The Plasma Concentration Versus Time Curve Over The Dosing Interval (AUCtau) Of Multiple-dose ALXN2050

  Up to 168 hours postdose

• Maximum Plasma Concentration (Cmax) Of Single-dose ALXN2050

  Up to 72 hours postdose

• Time To Reach Maximum Plasma Concentration (Tmax) Of Single-dose ALXN2050

  Up to 72 hours postdose

Study Arms (6)

Cohort 1: 40 mg ALXN2050/Placebo

EXPERIMENTAL

Participants randomized to receive ALXN2050 or placebo twice daily (BID) on Day 1 through Day 14 in a fasted state.

Drug: ALXN2050; Drug: Placebo

Cohort 2: 80 mg ALXN2050/Placebo

EXPERIMENTAL

Participants randomized to receive ALXN2050 or placebo BID on Day 1 through Day 14 in a fasted state.

Drug: ALXN2050; Drug: Placebo

Cohort 3: 120 mg ALXN2050/Placebo

EXPERIMENTAL

Participants randomized to receive ALXN2050 or placebo BID on Day 1 through Day 14 in a fasted state.

Drug: ALXN2050; Drug: Placebo

Cohort 4: 200 mg ALXN2050/Placebo

EXPERIMENTAL

Participants randomized to receive ALXN2050 or placebo BID on Day 1 through Day 14 in a fasted state.

Drug: ALXN2050; Drug: Placebo

Cohort 5: 120 mg ALXN2050/Placebo

EXPERIMENTAL

Participants randomized to receive a single dose of ALXN2050 or placebo on Day 1 in a fed state.

Drug: ALXN2050; Drug: Placebo

Cohort 6: 240 mg ALXN2050/Placebo

EXPERIMENTAL

Participants randomized to receive a single dose of ALXN2050 or placebo on Day 1 in a fasted state.

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; Cohort 4: 200 mg ALXN2050/Placebo; Cohort 5: 120 mg ALXN2050/Placebo; Cohort 6: 240 mg ALXN2050/Placebo

Placebo DRUG

PIC.

Cohort 1: 40 mg ALXN2050/Placebo; Cohort 2: 80 mg ALXN2050/Placebo; Cohort 3: 120 mg ALXN2050/Placebo; Cohort 4: 200 mg ALXN2050/Placebo; Cohort 5: 120 mg ALXN2050/Placebo; Cohort 6: 240 mg ALXN2050/Placebo

Eligibility Criteria

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

You may qualify if:

• Was overtly healthy as determined by medical evaluation including detailed medical history, physical examination, blood pressure and heart rate measurements, 12-lead ECG, and clinical laboratory tests.
• Had a body weight of at least 50 kilograms (kg) and body mass index within the range of 18 to 30 kg/meter squared (inclusive).
• Male participants were eligible to participate if they agreed to abstinence or use of a highly effective method of contraception.
• Female participants must have been of nonchildbearing potential.

You may not qualify if:

• Had a history or clinically relevant evidence of current cardiovascular, pulmonary, hepatic, renal, hematological, gastrointestinal, endocrine, immunologic, dermatologic, neurological, or psychiatric disorders or conditions capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study intervention; or interfering with the interpretation of data.
• 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 sensitivity to any of the study interventions, or components thereof, or drug or other allergy that contraindicated participation in the study.
• Donated blood or lost more than 500 milliliters of blood within 3 months prior to first study drug administration, or received a blood transfusion or blood products within 6 months prior to first study drug administration.
• Current enrollment or past participation within the last 30 days before study drug administration in any clinical study involving an investigational study intervention or any other type of medical research
• Had clinically significant laboratory abnormalities.
• Positive urine drug screen at Screening or Day -1; was a current tobacco/nicotine user or smoker; consumed any alcohol within 72 hours before first study drug administration or had a history of regular alcohol consumption within 6 months of screening.

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: January 7, 2019
• Primary Completion: July 23, 2019
• Study Completion: July 23, 2019
• Last Updated: September 17, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will share

Locations

NZ (1)