A Study of Single-dose ALXN2050 in Healthy Adults

Study Overview
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Brief Summary
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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.

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

Conditions
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Healthy

Intervention / Treatment
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* Drug: ALXN2050
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 25 Years
Maximum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

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

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 | Measure Description | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Factor D Inhibitor, Complement, ALXN2050, ACH-0145228, Pharmacokinetics, Pharmacodynamics, Safety