Trial Outcomes & Findings for Study of ALXN1850 in Participants With Hypophosphatasia (HPP) (NCT NCT04980248)
NCT ID: NCT04980248
Last Updated: 2024-12-27
Results Overview
TEAEs were defined as any adverse events (AEs) that began or worsened on or after the first dose of treatment until the final follow-up visit. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. TESAEs were defined as any serious AEs that began or worsened on or after the first dose of treatment until the final follow-up visit. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
15 participants
Primary outcome timeframe
Day 1 up to Day 85
Results posted on
2024-12-27
Participant Flow
Participant milestones
| Measure |
Cohort 1
Participants received 15 milligrams (mg) of ALXN1850 as a single intravenous dose, followed by 15 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 2
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Overall Study
STARTED
|
5
|
5
|
5
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
5
|
5
|
5
|
|
Overall Study
COMPLETED
|
5
|
5
|
5
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of ALXN1850 in Participants With Hypophosphatasia (HPP)
Baseline characteristics by cohort
| Measure |
Cohort 1
n=5 Participants
Participants received 15 mg of ALXN1850 as a single intravenous dose, followed by 15 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 2
n=5 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=5 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Total
n=15 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
44.2 years
STANDARD_DEVIATION 12.44 • n=5 Participants
|
37.8 years
STANDARD_DEVIATION 12.52 • n=7 Participants
|
54.6 years
STANDARD_DEVIATION 9.07 • n=5 Participants
|
45.5 years
STANDARD_DEVIATION 12.80 • n=4 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
15 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 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
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
15 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Day 1 up to Day 85Population: The Safety Set included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
TEAEs were defined as any adverse events (AEs) that began or worsened on or after the first dose of treatment until the final follow-up visit. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. TESAEs were defined as any serious AEs that began or worsened on or after the first dose of treatment until the final follow-up visit. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Outcome measures
| Measure |
Cohort 1
n=5 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=5 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=5 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
TEAEs
|
3 Participants
|
5 Participants
|
4 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
TESAEs
|
0 Participants
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Predose, 2, 6 and 12 hours postdose on Days 1, 15, 22 and 29Population: The Pharmacokinetic (PK) Set included all treated participants for whom the PK profile of ALXN1850 was adequately characterized. PK analyses was based upon the study drug received. Here, number analyzed signifies those participants who were evaluable for prespecified categories only.
Outcome measures
| Measure |
Cohort 1
n=5 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=5 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=5 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3
IV Dose
|
5.31 micrograms/milliliter
Geometric Coefficient of Variation 29.2
|
13.5 micrograms/milliliter
Geometric Coefficient of Variation 23.3
|
32.4 micrograms/milliliter
Geometric Coefficient of Variation 31.1
|
|
Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3
SC Dose 1
|
0.871 micrograms/milliliter
Geometric Coefficient of Variation 43.8
|
2.89 micrograms/milliliter
Geometric Coefficient of Variation 59.4
|
8.60 micrograms/milliliter
Geometric Coefficient of Variation 43.5
|
|
Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3
SC Dose 2
|
0.815 micrograms/milliliter
Geometric Coefficient of Variation 52.3
|
2.38 micrograms/milliliter
Geometric Coefficient of Variation 88.8
|
6.33 micrograms/milliliter
Geometric Coefficient of Variation 55.5
|
|
Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3
SC Dose 3
|
0.844 micrograms/milliliter
Geometric Coefficient of Variation 75.2
|
2.41 micrograms/milliliter
Geometric Coefficient of Variation 97.4
|
5.44 micrograms/milliliter
Geometric Coefficient of Variation 145
|
SECONDARY outcome
Timeframe: Predose, 2, 6 and 12 hours postdose on Days 1, 15, 22 and 29Population: The PK Set included all treated participants for whom the PK profile of ALXN1850 was adequately characterized. PK analyses was based upon the study drug received. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure only.
Outcome measures
| Measure |
Cohort 1
n=4 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=5 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=4 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Area Under the Plasma Concentration Versus Time Curve From Time 0 Extrapolated to Infinity Following IV Dose of ALXN1850
|
407 hour*micrograms/milliliter
Geometric Coefficient of Variation 38.3
|
1010 hour*micrograms/milliliter
Geometric Coefficient of Variation 26.0
|
2450 hour*micrograms/milliliter
Geometric Coefficient of Variation 33.6
|
SECONDARY outcome
Timeframe: Predose, 2, 6 and 12 hour postdose on Days 1, 15, 22 and 29Population: The PK Set included all treated participants for whom the PK profile of ALXN1850 was adequately characterized. PK analyses was based upon the study drug received. Here, overall number of participants analyzed signfies those participants who were evaluable for this endpoint only and number analyzed signifies those participants who were evaluable for prespecified categories only.
Outcome measures
| Measure |
Cohort 1
n=5 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=5 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=4 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Following SC Dose 1, SC Dose 2 and SC Dose 3
SC Dose 1
|
115 hour*micrograms/milliliter
Geometric Coefficient of Variation 42.2
|
383 hour*micrograms/milliliter
Geometric Coefficient of Variation 58.1
|
1110 hour*micrograms/milliliter
Geometric Coefficient of Variation 49.6
|
|
Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Following SC Dose 1, SC Dose 2 and SC Dose 3
SC Dose 2
|
111 hour*micrograms/milliliter
Geometric Coefficient of Variation 58.5
|
350 hour*micrograms/milliliter
Geometric Coefficient of Variation 83.3
|
872 hour*micrograms/milliliter
Geometric Coefficient of Variation 65.5
|
|
Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Following SC Dose 1, SC Dose 2 and SC Dose 3
SC Dose 3
|
115 hour*micrograms/milliliter
Geometric Coefficient of Variation 73.9
|
367 hour*micrograms/milliliter
Geometric Coefficient of Variation 95.6
|
881 hour*micrograms/milliliter
Geometric Coefficient of Variation 104
|
SECONDARY outcome
Timeframe: Predose, 2, 6 and 12 hour postdose on Days 1, 15, 22 and 29Population: The PK Set included all treated participants for whom the PK profile of ALXN1850 was adequately characterized. PK analyses was based upon the study drug received. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure only. As pre-specified, data are presented pooled for all participants who received study drug.
Outcome measures
| Measure |
Cohort 1
n=15 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Values of the First SC Versus IV Administration of ALXN1850
15 mg
|
0.286 Ratio
Interval 0.105 to 0.782
|
—
|
—
|
|
Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Values of the First SC Versus IV Administration of ALXN1850
45 mg
|
0.367 Ratio
Interval 0.18 to 0.784
|
—
|
—
|
|
Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Values of the First SC Versus IV Administration of ALXN1850
90 mg
|
0.368 Ratio
Interval 0.0908 to 1.49
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3Population: The Pharmacodynamic (PD) Set included all treated participants for whom the PD profile of ALXN1850 was adequately characterized. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure only. Here, number analyzed signifies those participants who were evaluable for prespecified timepoints only.
Outcome measures
| Measure |
Cohort 1
n=4 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=4 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=4 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Absolute Change From Baseline in Plasma Concentration of Pyridoxal-5' Phosphate (PLP) at Week 1
1 Week post Day 1 IV Dose
|
-18.300 nanogram/milliliter
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
-7.600 nanogram/milliliter
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
-49.460 nanogram/milliliter
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
|
Absolute Change From Baseline in Plasma Concentration of Pyridoxal-5' Phosphate (PLP) at Week 1
1 Week post Day 29 SC Dose 3
|
-97.950 nanogram/milliliter
Standard Deviation 164.0193
|
-38.573 nanogram/milliliter
Standard Deviation 26.4932
|
-28.055 nanogram/milliliter
Standard Deviation 17.3237
|
SECONDARY outcome
Timeframe: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3Population: The PD Set included all treated participants for whom the PD profile of ALXN1850 was adequately characterized. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure only. Number analyzed signifies those participants who were evaluable for the prespecified timepoints only.
Outcome measures
| Measure |
Cohort 1
n=4 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=2 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=1 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Absolute Change From Baseline in Plasma Concentration of Inorganic Pyrophosphate (PPi) at Week 1
1 Week post Day 1 IV Dose
|
-1.620 micromoles (uM)
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
-1.930 micromoles (uM)
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
—
|
|
Absolute Change From Baseline in Plasma Concentration of Inorganic Pyrophosphate (PPi) at Week 1
1 Week post Day 29 SC Dose 3
|
-0.813 micromoles (uM)
Standard Deviation 0.5970
|
-1.505 micromoles (uM)
Standard Deviation 0.1202
|
-2.040 micromoles (uM)
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
SECONDARY outcome
Timeframe: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3Population: The PD Set included all treated participants for whom the PD profile of ALXN1850 was adequately characterized. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure only. Here, number analyzed signifies those participants who were evaluable for prespecified timepoints only.
Outcome measures
| Measure |
Cohort 1
n=4 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=4 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=4 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Absolute Change From Baseline in Plasma Concentration of Pyridoxal Phosphate/ Pyridoxal (PLP/PL) Ratio at Week 1
1 Week post Day 1 IV Dose
|
-8.570 Ratio
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
-16.070 Ratio
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
-15.260 Ratio
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
|
Absolute Change From Baseline in Plasma Concentration of Pyridoxal Phosphate/ Pyridoxal (PLP/PL) Ratio at Week 1
1 Week post Day 29 SC Dose 3
|
-7.620 Ratio
Standard Deviation 5.3878
|
-15.285 Ratio
Standard Deviation 6.2807
|
-8.368 Ratio
Standard Deviation 6.3951
|
SECONDARY outcome
Timeframe: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3Population: The PD Set included all treated participants for whom the PD profile of ALXN1850 was adequately characterized. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure only. Here, number analyzed signifies those participants who were evaluable for prespecified timepoints only.
Outcome measures
| Measure |
Cohort 1
n=4 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=4 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=4 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Percent Change From Baseline in Plasma Concentration of PLP at Week 1
1 Week post Day 1 IV Dose
|
-47.906 percent change
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
-17.512 percent change
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
-86.318 percent change
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
|
Percent Change From Baseline in Plasma Concentration of PLP at Week 1
1 Week post Day 29 SC Dose 3
|
-33.895 percent change
Standard Deviation 42.5254
|
-69.049 percent change
Standard Deviation 16.5385
|
-70.515 percent change
Standard Deviation 23.0447
|
SECONDARY outcome
Timeframe: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3Population: The PD Set included all treated participants for whom the PD profile of ALXN1850 was adequately characterized. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure only. Number analyzed signifies those participants who were evaluable for prespecified timepoints only.
Outcome measures
| Measure |
Cohort 1
n=4 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=2 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=1 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Percent Change From Baseline in Plasma Concentration of PPi at Week 1
1 Week post Day 1 IV Dose
|
-53.821 percent change
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
-70.956 percent change
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
—
|
|
Percent Change From Baseline in Plasma Concentration of PPi at Week 1
1 Week post Day 29 SC Dose 3
|
-32.740 percent change
Standard Deviation 23.0615
|
-55.328 percent change
Standard Deviation 1.7622
|
-71.329 percent change
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
SECONDARY outcome
Timeframe: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3Population: The PD Set included all treated participants for whom the PD profile of ALXN1850 was adequately characterized. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure only. Here, number analyzed signifies those participants who were evaluable for prespecified timepoints only.
Outcome measures
| Measure |
Cohort 1
n=4 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=4 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=4 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Percent Change From Baseline in Plasma Concentration of PLP/PL Ratio Over Time at Week 1
1 Week post Day 1 IV Dose
|
-65.270 percent change
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
-92.944 percent change
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
-91.323 percent change
Standard Deviation NA
Standard deviation could not be calculated as there was only 1 evaluable participant.
|
|
Percent Change From Baseline in Plasma Concentration of PLP/PL Ratio Over Time at Week 1
1 Week post Day 29 SC Dose 3
|
-35.104 percent change
Standard Deviation 15.0384
|
-77.160 percent change
Standard Deviation 16.0636
|
-60.816 percent change
Standard Deviation 49.7578
|
SECONDARY outcome
Timeframe: Baseline up to Day 85Population: The Immunogenicity Analysis Set included all treated participants who received any study drug and who after the first dose had at least one reportable result in the ADA assay.
Outcome measures
| Measure |
Cohort 1
n=5 Participants
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=5 Participants
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=5 Participants
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Number of Participants With Anti-drug Antibody (ADA) Positive and Neutralizing Antibody (NAb) Positive Status
ADA Positive
|
2 Participants
|
0 Participants
|
2 Participants
|
|
Number of Participants With Anti-drug Antibody (ADA) Positive and Neutralizing Antibody (NAb) Positive Status
NAb Positive
|
0 Participants
|
0 Participants
|
0 Participants
|
Adverse Events
Cohort 1
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 2
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Cohort 3
Serious events: 1 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1
n=5 participants at risk
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=5 participants at risk
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=5 participants at risk
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
Other adverse events
| Measure |
Cohort 1
n=5 participants at risk
Participants received 15 mg as a single intravenous dose, followed by 15 mg subcutaneous at 1-week interval for 3 doses.
|
Cohort 2
n=5 participants at risk
Participants received 45 mg of ALXN1850 as a single intravenous dose, followed by 45 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
Cohort 3
n=5 participants at risk
Participants received 90 mg of ALXN1850 as a single intravenous dose, followed by 90 mg of ALXN1850 as a subcutaneous injection at 1-week interval for 3 doses.
|
|---|---|---|---|
|
General disorders
Fatigue
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
General disorders
Injection site bruising
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
General disorders
Injection site erythema
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
General disorders
Injection site haemorrhage
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
General disorders
Injection site pruritus
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
General disorders
Injection site reaction
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
General disorders
Vessel puncture site bruise
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Musculoskeletal and connective tissue disorders
Muscle tightness
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Musculoskeletal and connective tissue disorders
Spinal pain
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Musculoskeletal and connective tissue disorders
Headache
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
40.0%
2/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Musculoskeletal and connective tissue disorders
Dizziness
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Infections and infestations
Coronavirus infection
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
40.0%
2/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Infections and infestations
Vulvovaginal mycotic infection
|
0.00%
0/3 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/3 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Metabolism and nutrition disorders
Hyperphosphataemia
|
40.0%
2/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Blood and lymphatic system disorders
Anaemia
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Blood and lymphatic system disorders
Abdominal pain
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Blood and lymphatic system disorders
Nausea
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Investigations
Blood phosphorus increased
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Investigations
Gamma-glutamyltransferase increased
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
40.0%
2/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Renal and urinary disorders
Hyperphosphaturia
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Cardiac disorders
Dry eye
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Reproductive system and breast disorders
Breast discomfort
|
0.00%
0/3 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/3 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
|
Vascular disorders
Hypertension
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
20.0%
1/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
0.00%
0/5 • Baseline up to Day 85
All-cause mortality is reported for all enrolled participants. Serious adverse events and other adverse events are reported in the Safety Set, which included all participants who received any amount of study drug. Participants were analyzed according to the study drug received.
|
Additional Information
Alexion Pharmaceuticals Inc.
Alexion Pharmaceuticals Inc.
Phone: 855-752-2356
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place