Trial · NCT04980248
Study of ALXN1850 in Participants With Hypophosphatasia (HPP)
NCT04980248, Phase 1, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2024-12-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of ALXN1850 in Participants With Hypophosphatasia (HPP). Trials, record NCT04980248. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04980248
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of ALXN1850 in Participants With Hypophosphatasia (HPP)
- NCT number
- NCT04980248
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 15 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Hypophosphatasia
- Interventions
- ALXN1850
- First posted
- 2021-07-28
- Last updated
- 2024-12-27
- Results first posted
- 2024-12-27
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) | Day 1 up to Day 85 | 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. |
| Secondary | Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3 | Predose, 2, 6 and 12 hours postdose on Days 1, 15, 22 and 29 | |
| Secondary | Area Under the Plasma Concentration Versus Time Curve From Time 0 Extrapolated to Infinity Following IV Dose of ALXN1850 | Predose, 2, 6 and 12 hours postdose on Days 1, 15, 22 and 29 | |
| Secondary | 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 | Predose, 2, 6 and 12 hour postdose on Days 1, 15, 22 and 29 | |
| Secondary | 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 | Predose, 2, 6 and 12 hour postdose on Days 1, 15, 22 and 29 | |
| Secondary | Absolute Change From Baseline in Plasma Concentration of Pyridoxal-5' Phosphate (PLP) at Week 1 | Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3 | |
| Secondary | Absolute Change From Baseline in Plasma Concentration of Inorganic Pyrophosphate (PPi) at Week 1 | Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3 | |
| Secondary | Absolute Change From Baseline in Plasma Concentration of Pyridoxal Phosphate/ Pyridoxal (PLP/PL) Ratio at Week 1 | Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3 | |
| Secondary | Percent Change From Baseline in Plasma Concentration of PLP at Week 1 | Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3 | |
| Secondary | Percent Change From Baseline in Plasma Concentration of PPi at Week 1 | Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) | Day 1 up to Day 85 | The Safety Set included all participants who received any amount of study drug. Participants were analyzed according to the study drug received. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3 | Predose, 2, 6 and 12 hours postdose on Days 1, 15, 22 and 29 | 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. | micrograms/milliliter | GEOMETRIC_MEAN |
| SECONDARY | Area Under the Plasma Concentration Versus Time Curve From Time 0 Extrapolated to Infinity Following IV Dose of ALXN1850 | Predose, 2, 6 and 12 hours postdose on Days 1, 15, 22 and 29 | 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. | hour*micrograms/milliliter | GEOMETRIC_MEAN |
| SECONDARY | 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 | Predose, 2, 6 and 12 hour postdose on Days 1, 15, 22 and 29 | 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. | hour*micrograms/milliliter | GEOMETRIC_MEAN |
| SECONDARY | 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 | Predose, 2, 6 and 12 hour postdose on Days 1, 15, 22 and 29 | 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. | Ratio | GEOMETRIC_LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change From Baseline in Plasma Concentration of Pyridoxal-5' Phosphate (PLP) at Week 1 | Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3 | 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. | nanogram/milliliter | MEAN |
| SECONDARY | Absolute Change From Baseline in Plasma Concentration of Inorganic Pyrophosphate (PPi) at Week 1 | Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3 | 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. | micromoles (uM) | MEAN |
| SECONDARY | Absolute Change From Baseline in Plasma Concentration of Pyridoxal Phosphate/ Pyridoxal (PLP/PL) Ratio at Week 1 | Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3 | 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. | Ratio | MEAN |
| SECONDARY | Percent Change From Baseline in Plasma Concentration of PLP at Week 1 | Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3 | 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. | percent change | MEAN |
| SECONDARY | Percent Change From Baseline in Plasma Concentration of PPi at Week 1 | Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3 | 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. | percent change | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group73
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) | Cohort 1 | TEAEs | 3 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) | Cohort 2 | TEAEs | 5 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) | Cohort 3 | TEAEs | 4 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) | Cohort 1 | TESAEs | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) | Cohort 2 | TESAEs | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) | Cohort 3 | TESAEs | 1 | Participants | |
| Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3 | Cohort 1 | IV Dose | 5.31 | Geometric Coefficient of Variation 29.2 | micrograms/milliliter |
| Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3 | Cohort 2 | IV Dose | 13.5 | Geometric Coefficient of Variation 23.3 | micrograms/milliliter |
| Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3 | Cohort 3 | IV Dose | 32.4 | Geometric Coefficient of Variation 31.1 | micrograms/milliliter |
| Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3 | Cohort 1 | SC Dose 1 | 0.871 | Geometric Coefficient of Variation 43.8 | micrograms/milliliter |
1 to 10 of 73Source ClinicalTrials.gov
See all 73 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1 | 5 | |
| Overall Study | STARTED | Cohort 2 | 5 | |
| Overall Study | STARTED | Cohort 3 | 5 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cohort 1 | 5 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cohort 2 | 5 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cohort 3 | 5 | |
| Overall Study | COMPLETED | Cohort 1 | 5 | |
| Overall Study | COMPLETED | Cohort 2 | 5 | |
| Overall Study | COMPLETED | Cohort 3 | 5 | |
| Overall Study | NOT COMPLETED | Cohort 1 | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events93
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Fatigue | General disorders | Cohort 1 | 1 | 5 | |
| other | Fatigue | General disorders | Cohort 2 | 1 | 5 | |
| other | Fatigue | General disorders | Cohort 3 | 0 | 5 | |
| other | Injection site bruising | General disorders | Cohort 1 | 1 | 5 | |
| other | Injection site bruising | General disorders | Cohort 2 | 1 | 5 | |
| other | Injection site bruising | General disorders | Cohort 3 | 0 | 5 | |
| other | Injection site erythema | General disorders | Cohort 1 | 0 | 5 | |
| other | Injection site erythema | General disorders | Cohort 2 | 1 | 5 | |
| other | Injection site erythema | General disorders | Cohort 3 | 1 | 5 | |
| other | Injection site haemorrhage | General disorders | Cohort 1 | 0 | 5 |
1 to 10 of 93Source ClinicalTrials.gov
See all 93 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1 | 0 | 5 |
| Total, all-cause mortality | Cohort 2 | 0 | 5 |
| Total, all-cause mortality | Cohort 3 | 0 | 5 |
| Total, other adverse events | Cohort 1 | 3 | 5 |
| Total, other adverse events | Cohort 2 | 5 | 5 |
| Total, other adverse events | Cohort 3 | 4 | 5 |
| Total, serious adverse events | Cohort 1 | 0 | 5 |
| Total, serious adverse events | Cohort 2 | 0 | 5 |
| Total, serious adverse events | Cohort 3 | 1 | 5 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39135540 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04980248
GET https://conductscience.org/api/trials/v1/NCT04980248/results?page=2