Trial Outcomes & Findings for Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants (NCT NCT05641311)

NCT ID: NCT05641311

Last Updated: 2023-11-07

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

24 participants

Primary outcome timeframe

Day 1 through Day 11 of Dosing Periods 1 and 2

Results posted on

2023-11-07

Participant Flow

For this study, Japanese participants were defined as those whose parents and grandparents were both Japanese and who had spent less than 5 years outside of Japan.

A total of 24 participants were screened, all of whom were enrolled and received ALXN1840 in the study at 1 study site. Of the 24 enrolled, 12 were Japanese, therefore, they were allocated to Cohort 1, and 12 were non-Japanese, therefore, they were allocated to Cohort 2. All participants were included in all analysis sets.

Participant milestones

Participant milestones
Measure	Cohort 1: Japanese Participants All Japanese participants received a single dose of ALXN1840 15 milligrams (mg) in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2.	Cohort 2: Non-Japanese Participants All non-Japanese participants received a single dose of ALXN1840 15 mg in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2.
Dosing Period 1 STARTED	12	12
Dosing Period 1 Received At Least 1 Dose Of Study Drug	12	12
Dosing Period 1 COMPLETED	11	12
Dosing Period 1 NOT COMPLETED	1	0
Dosing Period 2 STARTED	11	12
Dosing Period 2 Received At Least 1 Dose Of Study Drug	11	12
Dosing Period 2 COMPLETED	11	12
Dosing Period 2 NOT COMPLETED	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1: Japanese Participants All Japanese participants received a single dose of ALXN1840 15 milligrams (mg) in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2.	Cohort 2: Non-Japanese Participants All non-Japanese participants received a single dose of ALXN1840 15 mg in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2.
Dosing Period 1 Adverse Event	1	0

Baseline Characteristics

Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1: Japanese Participants n=12 Participants All Japanese participants received a single dose of ALXN1840 15 mg in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2.	Cohort 2: Non-Japanese Participants n=12 Participants All non-Japanese participants received a single dose of ALXN1840 15 mg in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2.	Total n=24 Participants Total of all reporting groups
Age, Continuous	31.7 years STANDARD_DEVIATION 5.91 • n=99 Participants	29.5 years STANDARD_DEVIATION 6.78 • n=107 Participants	30.6 years STANDARD_DEVIATION 6.32 • n=206 Participants
Sex: Female, Male Female	5 Participants n=99 Participants	5 Participants n=107 Participants	10 Participants n=206 Participants
Sex: Female, Male Male	7 Participants n=99 Participants	7 Participants n=107 Participants	14 Participants n=206 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	12 Participants n=99 Participants	12 Participants n=107 Participants	24 Participants n=206 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Asian	12 Participants n=99 Participants	1 Participants n=107 Participants	13 Participants n=206 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) White	0 Participants n=99 Participants	11 Participants n=107 Participants	11 Participants n=206 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants

PRIMARY outcome

Timeframe: Day 1 through Day 11 of Dosing Periods 1 and 2

Population: The PK population consisted of all participants who had sufficient plasma samples to enable the calculation of PK parameters of at least area under the curve (AUC) for at least one period.

Outcome measures

Outcome measures
Measure	Cohort 1: Japanese Participants n=12 Participants All Japanese participants received a single dose of ALXN1840 15 mg in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2.	Cohort 2: Non-Japanese Participants n=12 Participants All non-Japanese participants received a single dose of ALXN1840 15 mg in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2.
Area Under The Plasma Concentration Versus Time Curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) Of Plasma Total Molybdenum After Each Dose Dosing Period 1: ALXN1840 15 mg	10158.9753 hours* nanograms/milliliters Standard Deviation 1427.41833	9221.0132 hours* nanograms/milliliters Standard Deviation 3413.75213
Area Under The Plasma Concentration Versus Time Curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) Of Plasma Total Molybdenum After Each Dose Dosing Period 2: ALXN1840 60 mg	16178.7885 hours* nanograms/milliliters Standard Deviation 6103.53245	20911.3717 hours* nanograms/milliliters Standard Deviation 11491.5869

Adverse Events

Cohort 1: Japanese Participants

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Cohort 2: Non-Japanese Participants

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Alexion Pharmaceuticals Inc.

Phone: +1 855-752-2356

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Cohort 1: Japanese Participants n=12 participants at risk All Japanese participants received a single dose of ALXN1840 15 mg in Dosing Period 1 and received a single dose of ALXN1840 60 mg in Dosing Period 2.	Cohort 2: Non-Japanese Participants n=12 participants at risk All non-Japanese participants received a single dose of 15 mg ALXN1840 in Dosing Period 1 and a single dose of 60 mg ALXN1840 in Dosing Period 2.
Nervous system disorders Dizziness	16.7% 2/12 • Number of events 2 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Nervous system disorders Headache	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	25.0% 3/12 • Number of events 3 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Nervous system disorders Presyncope	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Nervous system disorders Paraesthesia	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Nervous system disorders Syncope	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Gastrointestinal disorders Nausea	16.7% 2/12 • Number of events 3 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Gastrointestinal disorders Haemorrhoids	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
General disorders Medical device site reaction	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
General disorders Non-cardiac chest pain	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Infections and infestations Oral herpes	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Infections and infestations Urinary tract infection	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Investigations Liver function test abnormal	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Musculoskeletal and connective tissue disorders Pain in extremity	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Reproductive system and breast disorders Vaginal discharge	20.0% 1/5 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	0.00% 0/5 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.
Skin and subcutaneous tissue disorders Ingrowing nail	8.3% 1/12 • Number of events 1 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.	0.00% 0/12 • Baseline up to end of study visit (up to Day 29) The safety population consisted of all participants who received at least 1 dose of the study drug. All-Cause Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected by their assigned cohort, regardless of their dose level.