Trial Outcomes & Findings for Copper Balance in Healthy Participants Administered ALXN1840 (NCT NCT04594252)
NCT ID: NCT04594252
Last Updated: 2024-08-19
Results Overview
Copper balance was defined as the difference in copper input and copper output. A negative copper balance indicated greater copper output than copper intake. Copper input was defined as the sum of all copper input as measured in all food and fluids over the specified period. Copper output was defined as the sum of all copper output as measured in urine and feces over the specified collection period. Baseline was defined as the average of the nonmissing values on or before first study drug administration from Day -4 to Day -1.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
17 participants
Primary outcome timeframe
Baseline, Days 4 to 15
Results posted on
2024-08-19
Participant Flow
A total of 17 participants were enrolled in 2 groups. Group 1 consisted of 6 participants. Group 2, consisting of 11 participants, was initiated after the Safety Review Committee (SRC) reviewed safety information from Group 1. No safety concerns were identified; therefore, no dose adjustments were implemented with Group 2.
Participant milestones
| Measure |
Group 1: ALXN1840
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|
|
Overall Study
STARTED
|
6
|
11
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
6
|
11
|
|
Overall Study
COMPLETED
|
6
|
9
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
Reasons for withdrawal
| Measure |
Group 1: ALXN1840
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|
|
Overall Study
Adverse Event
|
0
|
2
|
Baseline Characteristics
Copper Balance in Healthy Participants Administered ALXN1840
Baseline characteristics by cohort
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total
n=17 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
27.0 years
STANDARD_DEVIATION 6.29 • n=5 Participants
|
29.5 years
STANDARD_DEVIATION 5.61 • n=7 Participants
|
28.6 years
STANDARD_DEVIATION 5.80 • n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Mean Daily Copper Balance
|
0.8538 milligrams (mg)/day
STANDARD_DEVIATION 0.48629 • n=5 Participants
|
0.3317 milligrams (mg)/day
STANDARD_DEVIATION 0.44568 • n=7 Participants
|
0.5160 milligrams (mg)/day
STANDARD_DEVIATION 0.51399 • n=5 Participants
|
|
Mean Daily Molybdenum Balance
|
0.1286 mg/day
STANDARD_DEVIATION 0.04469 • n=5 Participants
|
0.0133 mg/day
STANDARD_DEVIATION 0.05035 • n=7 Participants
|
0.0540 mg/day
STANDARD_DEVIATION 0.07372 • n=5 Participants
|
|
Total Molybdenum Excretion in Feces
|
0.0586 mg
STANDARD_DEVIATION 0.02347 • n=5 Participants
|
0.0724 mg
STANDARD_DEVIATION 0.03023 • n=7 Participants
|
0.0675 mg
STANDARD_DEVIATION 0.02809 • n=5 Participants
|
|
Total Molybdenum Excretion in Urine
|
0.1039 mg
STANDARD_DEVIATION 0.02656 • n=5 Participants
|
0.1198 mg
STANDARD_DEVIATION 0.03546 • n=7 Participants
|
0.1142 mg
STANDARD_DEVIATION 0.03268 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Days 4 to 15Population: Full analysis set included all participants who received at least 1 dose of ALXN1840.
Copper balance was defined as the difference in copper input and copper output. A negative copper balance indicated greater copper output than copper intake. Copper input was defined as the sum of all copper input as measured in all food and fluids over the specified period. Copper output was defined as the sum of all copper output as measured in urine and feces over the specified collection period. Baseline was defined as the average of the nonmissing values on or before first study drug administration from Day -4 to Day -1.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Change From Baseline in Mean Daily Copper Balance Over 2 Weeks of Repeated Daily ALXN1840 Dosing (Over Days 4 to 15)
|
-0.0749 mg/day
Standard Deviation 0.37419
|
0.6901 mg/day
Standard Deviation 0.48176
|
0.4201 mg/day
Standard Deviation 0.57518
|
SECONDARY outcome
Timeframe: Day 4 through Day 15Population: Full analysis set included all participants who received at least 1 dose of ALXN1840.
Copper balance was defined by the difference in copper input and copper output. A negative copper balance indicated greater copper output than copper intake. Copper input was defined as the sum of all copper input as measured in all food and fluids over the specified period. Copper output was defined as the sum of all copper output as measured in urine and feces over the specified collection period.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Mean Daily Copper Balance Over Two Weeks of Repeated ALXN1840 Dosing
|
0.7789 mg/day
Standard Deviation 0.20297
|
1.0218 mg/day
Standard Deviation 0.21279
|
0.9361 mg/day
Standard Deviation 0.23558
|
SECONDARY outcome
Timeframe: Baseline, Days 12 to 15Population: Full analysis set included all participants who received at least 1 dose of ALXN1840.
Molybdenum mass balance was defined as the difference in molybdenum input and molybdenum output. A negative molybdenum balance indicated greater molybdenum output than molybdenum intake. Molybdenum input was defined as the sum of all molybdenum input as measured in all food and fluids over the specified period. Molybdenum output was defined as the sum of all molybdenum output as measured in urine and feces over the specified collection period. Baseline was defined as the average of the nonmissing values on or before first study drug administration from Day -4 to Day -1.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Change From Baseline in Mean Daily Molybdenum Balance at Steady State (Over Days 12 to 15)
|
1.7750 mg/day
Standard Deviation 0.98161
|
0.6189 mg/day
Standard Deviation 1.24337
|
1.0269 mg/day
Standard Deviation 1.26161
|
SECONDARY outcome
Timeframe: Baseline, Days 4 to 15Population: Full analysis set included all participants who received at least 1 dose of ALXN1840.
Molybdenum mass balance was defined as the difference in molybdenum input and molybdenum output. A negative molybdenum balance indicated greater molybdenum output than molybdenum intake. Molybdenum input was defined as the sum of all molybdenum input as measured in all food and fluids over the specified period. Molybdenum output was defined as the sum of all molybdenum output as measured in urine and feces over the specified collection period. Baseline was defined as the average of the nonmissing values on or before first study drug administration from Day -4 to Day -1. Molybdenum excretion for the Day 4 through Day 15 period included data averaged from Day 4 through Day 15.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Change From Baseline in Total Molybdenum Excretion in Urine and Feces Averaged Over 2 Weeks of Dosing (Days 4 to 15)
Urine
|
2.6210 mg
Standard Deviation 0.68681
|
2.5957 mg
Standard Deviation 0.39688
|
2.6046 mg
Standard Deviation 0.49600
|
|
Change From Baseline in Total Molybdenum Excretion in Urine and Feces Averaged Over 2 Weeks of Dosing (Days 4 to 15)
Feces
|
2.0247 mg
Standard Deviation 0.64199
|
1.9586 mg
Standard Deviation 0.32854
|
1.9819 mg
Standard Deviation 0.44421
|
SECONDARY outcome
Timeframe: Day 1 through Day 15Population: Full analysis set included all participants who received at least 1 dose of ALXN1840.
Molybdenum mass balance was defined as the difference in molybdenum input and molybdenum output. A negative molybdenum balance indicated greater molybdenum output than molybdenum intake. Molybdenum input was defined as the sum of all molybdenum input as measured in all food and fluids over the specified period. Molybdenum output was defined as the sum of all molybdenum output as measured in urine and feces over the specified collection period.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Mean Daily Molybdenum Balance Throughout the ALXN1840 Treatment Period (Day 1 Through Day 15)
|
2.5560 mg/day
Standard Deviation 0.73042
|
2.2861 mg/day
Standard Deviation 0.35654
|
2.3813 mg/day
Standard Deviation 0.51367
|
SECONDARY outcome
Timeframe: Days 4 to 15Population: Full analysis set included all participants who received at least 1 dose of ALXN1840.
Copper balance for the Day 4 through Day 15 period included data averaged from Day 4 through Day 15.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Copper Quantified in Food, Drink, Feces, and Urine Averaged Over 2 Weeks of Dosing
Copper Quantified in Food
|
1.5039 mg
Standard Deviation 0.10025
|
1.7178 mg
Standard Deviation 0.12247
|
1.6423 mg
Standard Deviation 0.15366
|
|
Copper Quantified in Food, Drink, Feces, and Urine Averaged Over 2 Weeks of Dosing
Copper Quantified in Drink
|
0.0270 mg
Standard Deviation 0.00120
|
0.0108 mg
Standard Deviation 0.00194
|
0.0165 mg
Standard Deviation 0.00816
|
|
Copper Quantified in Food, Drink, Feces, and Urine Averaged Over 2 Weeks of Dosing
Copper Quantified in Feces
|
0.7373 mg
Standard Deviation 0.23986
|
0.6895 mg
Standard Deviation 0.17102
|
0.7064 mg
Standard Deviation 0.19187
|
|
Copper Quantified in Food, Drink, Feces, and Urine Averaged Over 2 Weeks of Dosing
Copper Quantified in Urine
|
0.0147 mg
Standard Deviation 0.01102
|
0.0173 mg
Standard Deviation 0.00780
|
0.0164 mg
Standard Deviation 0.00881
|
SECONDARY outcome
Timeframe: Day 1 through Day 30Population: Full analysis set included all participants who received at least 1 dose of ALXN1840.
Copper balance for the Day 1 through Day 30 period included data averaged from Day 1 through Day 30.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Copper Quantified in Food, Drink, Feces, and Urine From Day 1 Through Day 30
Copper Quantified in Food
|
1.6117 mg
Standard Deviation 0.12736
|
1.6673 mg
Standard Deviation 0.12779
|
1.6476 mg
Standard Deviation 0.12659
|
|
Copper Quantified in Food, Drink, Feces, and Urine From Day 1 Through Day 30
Copper Quantified in Drink
|
0.0252 mg
Standard Deviation 0.00124
|
0.0124 mg
Standard Deviation 0.00212
|
0.0169 mg
Standard Deviation 0.00658
|
|
Copper Quantified in Food, Drink, Feces, and Urine From Day 1 Through Day 30
Copper Quantified in Feces
|
0.7017 mg
Standard Deviation 0.19009
|
0.6962 mg
Standard Deviation 0.13385
|
0.6982 mg
Standard Deviation 0.14999
|
|
Copper Quantified in Food, Drink, Feces, and Urine From Day 1 Through Day 30
Copper Quantified in Urine
|
0.0192 mg
Standard Deviation 0.01107
|
0.0209 mg
Standard Deviation 0.00655
|
0.0203 mg
Standard Deviation 0.00811
|
SECONDARY outcome
Timeframe: Day 15 (predose and 24 hours postdose)Population: Full analysis set included all participants who received at least 1 dose of ALXN1840. Here, 'number analyzed' signifies participants evaluable for specified categories.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Plasma Total Copper Concentration and Labile Bound Copper (LBC) Concentration
Total Copper Concentration at Day 15 (predose)
|
12.58931 micromoles (μmol)/liter (L)
Standard Deviation 1.636640
|
13.87256 micromoles (μmol)/liter (L)
Standard Deviation 1.627496
|
13.41964 micromoles (μmol)/liter (L)
Standard Deviation 1.700617
|
|
Plasma Total Copper Concentration and Labile Bound Copper (LBC) Concentration
Total Copper Concentration at Day 15 (24 hours postdose)
|
12.56570 micromoles (μmol)/liter (L)
Standard Deviation 1.342502
|
14.49816 micromoles (μmol)/liter (L)
Standard Deviation 1.548441
|
13.77349 micromoles (μmol)/liter (L)
Standard Deviation 1.724230
|
|
Plasma Total Copper Concentration and Labile Bound Copper (LBC) Concentration
LBC Concentration at Day 15 (predose)
|
0.93213 micromoles (μmol)/liter (L)
Standard Deviation 0.262655
|
0.82360 micromoles (μmol)/liter (L)
Standard Deviation 0.160887
|
0.86190 micromoles (μmol)/liter (L)
Standard Deviation 0.201482
|
|
Plasma Total Copper Concentration and Labile Bound Copper (LBC) Concentration
LBC Concentration at Day 15 (24 hours postdose)
|
1.04386 micromoles (μmol)/liter (L)
Standard Deviation 0.233526
|
0.90612 micromoles (μmol)/liter (L)
Standard Deviation 0.264791
|
0.95777 micromoles (μmol)/liter (L)
Standard Deviation 0.254932
|
SECONDARY outcome
Timeframe: Day 1 through Day 30Population: Full analysis set included all participants who received at least 1 dose of ALXN1840.
Molybdenum balance for the Day 1 through Day 30 period included data averaged from Day 1 through Day 30.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Molybdenum Quantified in ALXN1840 Doses Given and in Food, Drink, Feces, And Urine
Molybdenum Quantified in Food
|
0.2618 mg
Standard Deviation 0.02275
|
0.1414 mg
Standard Deviation 0.01123
|
0.1839 mg
Standard Deviation 0.06129
|
|
Molybdenum Quantified in ALXN1840 Doses Given and in Food, Drink, Feces, And Urine
Molybdenum Quantified in Drink
|
0.0014 mg
Standard Deviation 0.00015
|
0.0017 mg
Standard Deviation 0.00048
|
0.0016 mg
Standard Deviation 0.00041
|
|
Molybdenum Quantified in ALXN1840 Doses Given and in Food, Drink, Feces, And Urine
Molybdenum Quantified in Feces
|
1.0575 mg
Standard Deviation 0.29359
|
0.9949 mg
Standard Deviation 0.16082
|
1.0170 mg
Standard Deviation 0.20988
|
|
Molybdenum Quantified in ALXN1840 Doses Given and in Food, Drink, Feces, And Urine
Molybdenum Quantified in Urine
|
1.4016 mg
Standard Deviation 0.35415
|
1.3848 mg
Standard Deviation 0.21874
|
1.3907 mg
Standard Deviation 0.26300
|
|
Molybdenum Quantified in ALXN1840 Doses Given and in Food, Drink, Feces, And Urine
Molybdenum Quantified in ALXN1840 Doses Given
|
3.5933 mg
Standard Deviation 0.02277
|
3.3625 mg
Standard Deviation 0.28101
|
3.4439 mg
Standard Deviation 0.24988
|
SECONDARY outcome
Timeframe: Predose (within 1 hour prior to dosing) through 24 hours postdose on Day 1 and Day 15Population: Pharmacokinetic (PK) analysis set included all participants who had sufficient samples to enable the calculation of PK parameters and provide PK profiles. Here, 'number analyzed' signifies participants evaluable for specified categories.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Cmax of Total Molybdenum at Day 1
|
303.000 nanograms (ng)/mL
Standard Deviation 65.3667
|
291.727 nanograms (ng)/mL
Standard Deviation 100.7324
|
295.706 nanograms (ng)/mL
Standard Deviation 87.7950
|
|
Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Cmax of Total Molybdenum at Day 15
|
358.500 nanograms (ng)/mL
Standard Deviation 53.6945
|
382.333 nanograms (ng)/mL
Standard Deviation 78.2624
|
372.800 nanograms (ng)/mL
Standard Deviation 68.3794
|
|
Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Cmax of PUF Molybdenum at Day 1
|
30.445 nanograms (ng)/mL
Standard Deviation 12.0613
|
27.266 nanograms (ng)/mL
Standard Deviation 12.9396
|
28.388 nanograms (ng)/mL
Standard Deviation 12.3515
|
|
Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Cmax of PUF Molybdenum at Day 15
|
93.717 nanograms (ng)/mL
Standard Deviation 42.9368
|
74.678 nanograms (ng)/mL
Standard Deviation 36.1617
|
82.293 nanograms (ng)/mL
Standard Deviation 38.7152
|
SECONDARY outcome
Timeframe: Predose (within 1 hour prior to dosing) through 24 hours postdose on Day 1 and Day 15Population: PK analysis set included all participants who had sufficient samples to enable the calculation of PK parameters and provide PK profiles. Here, 'number analyzed' signifies participants evaluable for specified categories.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval (AUCtau) of Total Molybdenum and PUF Molybdenum
AUCtau of Total Molybdenum at Day 1
|
4860.289 hours*ng/mL
Standard Deviation 1078.263
|
4439.012 hours*ng/mL
Standard Deviation 1374.869
|
4587.698 hours*ng/mL
Standard Deviation 1260.082
|
|
Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval (AUCtau) of Total Molybdenum and PUF Molybdenum
AUCtau of Total Molybdenum at Day 15
|
6770.905 hours*ng/mL
Standard Deviation 582.770
|
7496.724 hours*ng/mL
Standard Deviation 1499.370
|
7206.397 hours*ng/mL
Standard Deviation 1241.529
|
|
Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval (AUCtau) of Total Molybdenum and PUF Molybdenum
AUCtau of PUF Molybdenum at Day 1
|
356.156 hours*ng/mL
Standard Deviation 133.8174
|
318.123 hours*ng/mL
Standard Deviation 131.9084
|
331.546 hours*ng/mL
Standard Deviation 129.6990
|
|
Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval (AUCtau) of Total Molybdenum and PUF Molybdenum
AUCtau of PUF Molybdenum at Day 15
|
1035.231 hours*ng/mL
Standard Deviation 252.0931
|
913.807 hours*ng/mL
Standard Deviation 379.1750
|
962.376 hours*ng/mL
Standard Deviation 329.6126
|
SECONDARY outcome
Timeframe: Predose (within 1 hour prior to dosing) through 24 hours postdose on Day 1 and Day 15Population: PK analysis set included all participants who had sufficient samples to enable the calculation of PK parameters and provide PK profiles. Here, 'number analyzed' signifies participants evaluable for specified categories.
Outcome measures
| Measure |
Group 1: ALXN1840
n=6 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 Participants
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Observed Concentration at the End of the Dosing Interval (Ctau) of Total Molybdenum and PUF Molybdenum
Ctau of Total Molybdenum at Day 1
|
143.800 ng/mL
Standard Deviation 35.5348
|
140.436 ng/mL
Standard Deviation 53.2088
|
141.624 ng/mL
Standard Deviation 46.5492
|
|
Observed Concentration at the End of the Dosing Interval (Ctau) of Total Molybdenum and PUF Molybdenum
Ctau of Total Molybdenum at Day 15
|
218.833 ng/mL
Standard Deviation 23.0340
|
242.625 ng/mL
Standard Deviation 54.5892
|
232.429 ng/mL
Standard Deviation 44.2488
|
|
Observed Concentration at the End of the Dosing Interval (Ctau) of Total Molybdenum and PUF Molybdenum
Ctau of PUF Molybdenum at Day 1
|
4.655 ng/mL
Standard Deviation 1.1623
|
4.785 ng/mL
Standard Deviation 1.6602
|
4.739 ng/mL
Standard Deviation 1.4660
|
|
Observed Concentration at the End of the Dosing Interval (Ctau) of Total Molybdenum and PUF Molybdenum
Ctau of PUF Molybdenum at Day 15
|
11.060 ng/mL
Standard Deviation 1.3785
|
11.141 ng/mL
Standard Deviation 2.8492
|
11.106 ng/mL
Standard Deviation 2.2591
|
Adverse Events
Group 1: ALXN1840
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Group 2: ALXN1840
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Total (Groups 1 and 2): ALXN1840
Serious events: 0 serious events
Other events: 14 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Group 1: ALXN1840
n=6 participants at risk
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Group 2: ALXN1840
n=11 participants at risk
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
Total (Groups 1 and 2): ALXN1840
n=17 participants at risk
Participants received repeat dose of ALXN1840 tablet administered orally on Day 1 through Day 15.
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
0.00%
0/11 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/6 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
9.1%
1/11 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Gastrointestinal disorders
Mouth ulceration
|
0.00%
0/6 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
9.1%
1/11 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Gastrointestinal disorders
Nausea
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
36.4%
4/11 • Number of events 4 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
29.4%
5/17 • Number of events 5 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
General disorders
Catheter site haematoma
|
0.00%
0/6 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
9.1%
1/11 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Infections and infestations
Folliculitis
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
0.00%
0/11 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
0.00%
0/11 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/6 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
9.1%
1/11 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Investigations
Liver function test abnormal
|
0.00%
0/6 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
18.2%
2/11 • Number of events 2 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
11.8%
2/17 • Number of events 2 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Investigations
Weight decreased
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
0.00%
0/11 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/6 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
9.1%
1/11 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Nervous system disorders
Dizziness
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
0.00%
0/11 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Nervous system disorders
Headache
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
18.2%
2/11 • Number of events 3 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
17.6%
3/17 • Number of events 4 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Psychiatric disorders
Depressed mood
|
0.00%
0/6 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
9.1%
1/11 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Renal and urinary disorders
Renal cyst
|
0.00%
0/6 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
9.1%
1/11 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
66.7%
2/3 • Number of events 2 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
0.00%
0/6 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
22.2%
2/9 • Number of events 2 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
0.00%
0/11 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Skin and subcutaneous tissue disorders
Acne
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
0.00%
0/11 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
0.00%
0/11 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Skin and subcutaneous tissue disorders
Dandruff
|
0.00%
0/6 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
9.1%
1/11 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
9.1%
1/11 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
11.8%
2/17 • Number of events 2 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Skin and subcutaneous tissue disorders
Ingrown hair
|
0.00%
0/6 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
9.1%
1/11 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
16.7%
1/6 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
0.00%
0/11 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
5.9%
1/17 • Number of events 1 • Baseline up to Day 43
Safety set included all participants who received at least 1 dose of ALXN1840.
|
Additional Information
Alexion Pharmaceuticals Inc.
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