Trial · NCT02797821

Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)

NCT02797821, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2018-07-10

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP). Trials, record NCT02797821. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02797821

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
NCT number: NCT02797821
Status: Completed
Phases: Phase 2
Enrollment: 27 participants, actual
Lead sponsor: Alexion Pharmaceuticals, Inc.
Conditions: Hypophosphatasia
Interventions: Asfotase alfa
First posted: 2016-06-14
Last updated: 2019-09-17
Results first posted: 2018-07-10

Source ClinicalTrials.gov

Registered outcomes2

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change In Plasma PPi From Baseline To Pre-3rd Dose At Week 9	Baseline to Week 9	Plasma PPi concentrations were determined using a specific enzyme-catalyzed reaction with a radiolabelled marker in a 3-step process. Baseline plasma PPi values were calculated by averaging pre-dose values from samples collected during the Run-in Period at -168, -156, -24, -12, and 0 hours before Baseline. Week 9 plasma PPi values were calculated using blood samples collected before administration of the 3rd dose. The analysis was a restricted maximum likelihood (REML)-based repeated measures mixed model with treatment, visit, sex, Baseline PPi, Baseline weight group (≥ median versus \< median), and study drug lot assignment as factors, and an unstructured covariance structure for within-participant correlation. Per inclusion criteria, participants had to have had a Screening PPi concentration of ≥3.9 micromolar (μM). Three participants (1 in each group) had Screening PPi concentrations of ≥3.9 μM, but Baseline PPi values ranged between 3.5 to 3.8 μM.
Secondary	Change In Plasma PLP From Baseline To Pre-3rd Dose At Week 9	Baseline to Week 9	Plasma PLP was quantified using liquid chromatography/mass spectrometry. Baseline plasma PLP values were calculated by averaging the pre-dose PLP values from blood samples collected during the Run-in Period at -168, -156, -24, -12, and 0 hours before Baseline. Week 9 PLP values were calculated using blood samples collected before the administration of the 3rd dose. The analysis was a REML-based repeated measures mixed model with treatment, visit, sex, Baseline PPi, Baseline weight group (≥ median versus \< median) and study drug lot assignment as factors, and an unstructured covariance structure for within-participant correlation.

Source ClinicalTrials.gov

Outcomes2

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change In Plasma PPi From Baseline To Pre-3rd Dose At Week 9	Baseline to Week 9	FAS: randomized participants who received ≥1 dose of study drug and had ≥1 pretreatment and ≥1 on-treatment PPi result.	μM	LEAST_SQUARES_MEAN
SECONDARY	Change In Plasma PLP From Baseline To Pre-3rd Dose At Week 9	Baseline to Week 9	FAS: randomized participants who received ≥1 dose of study drug and had ≥1 pretreatment and ≥1 on-treatment PPi result.	ng/mL	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group6

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Change In Plasma PPi From Baseline To Pre-3rd Dose At Week 9	Asfotase Alfa 0.5 mg/kg Dose	-2.604	Standard Error 0.2399	μM
Change In Plasma PPi From Baseline To Pre-3rd Dose At Week 9	Asfotase Alfa 2.0 mg/kg Dose	-3.797	Standard Error 0.1949	μM
Change In Plasma PPi From Baseline To Pre-3rd Dose At Week 9	Asfotase Alfa 3.0 mg/kg Dose	-4.484	Standard Error 0.2120	μM
Change In Plasma PLP From Baseline To Pre-3rd Dose At Week 9	Asfotase Alfa 0.5 mg/kg Dose	-303.955	Standard Error 9.4075	ng/mL
Change In Plasma PLP From Baseline To Pre-3rd Dose At Week 9	Asfotase Alfa 2.0 mg/kg Dose	-333.447	Standard Error 8.1486	ng/mL
Change In Plasma PLP From Baseline To Pre-3rd Dose At Week 9	Asfotase Alfa 3.0 mg/kg Dose	-338.002	Standard Error 85145	ng/mL

Source ClinicalTrials.gov

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Asfotase Alfa 0.5 mg/kg Dose	8
Overall Study	STARTED	Asfotase Alfa 2.0 mg/kg Dose	10
Overall Study	STARTED	Asfotase Alfa 3.0 mg/kg Dose	9
Overall Study	Received at Least 1 Dose of Study Drug	Asfotase Alfa 0.5 mg/kg Dose	8
Overall Study	Received at Least 1 Dose of Study Drug	Asfotase Alfa 2.0 mg/kg Dose	10
Overall Study	Received at Least 1 Dose of Study Drug	Asfotase Alfa 3.0 mg/kg Dose	9
Overall Study	COMPLETED	Asfotase Alfa 0.5 mg/kg Dose	8
Overall Study	COMPLETED	Asfotase Alfa 2.0 mg/kg Dose	10
Overall Study	COMPLETED	Asfotase Alfa 3.0 mg/kg Dose	9
Overall Study	NOT COMPLETED	Asfotase Alfa 0.5 mg/kg Dose	0

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events111

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
other	Fatigue	General disorders	Asfotase Alfa 0.5 mg/kg Dose	3	8
other	Fatigue	General disorders	Asfotase Alfa 2.0 mg/kg Dose	4	10
other	Fatigue	General disorders	Asfotase Alfa 3.0 mg/kg Dose	3	9
other	Injection site erythema	General disorders	Asfotase Alfa 0.5 mg/kg Dose	2	8
other	Injection site erythema	General disorders	Asfotase Alfa 2.0 mg/kg Dose	3	10
other	Injection site erythema	General disorders	Asfotase Alfa 3.0 mg/kg Dose	4	9
other	Injection site reaction	General disorders	Asfotase Alfa 0.5 mg/kg Dose	1	8
other	Injection site reaction	General disorders	Asfotase Alfa 2.0 mg/kg Dose	3	10
other	Injection site reaction	General disorders	Asfotase Alfa 3.0 mg/kg Dose	4	9
other	Injection site pain	General disorders	Asfotase Alfa 0.5 mg/kg Dose	2	8

1 to 10 of 111Source ClinicalTrials.gov

See all 111 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Asfotase Alfa 0.5 mg/kg Dose	0	8
Total, all-cause mortality	Asfotase Alfa 2.0 mg/kg Dose	0	10
Total, all-cause mortality	Asfotase Alfa 3.0 mg/kg Dose	0	9
Total, other adverse events	Asfotase Alfa 0.5 mg/kg Dose	8	8
Total, other adverse events	Asfotase Alfa 2.0 mg/kg Dose	10	10
Total, other adverse events	Asfotase Alfa 3.0 mg/kg Dose	9	9
Total, serious adverse events	Asfotase Alfa 0.5 mg/kg Dose	0	8
Total, serious adverse events	Asfotase Alfa 2.0 mg/kg Dose	0	10
Total, serious adverse events	Asfotase Alfa 3.0 mg/kg Dose	0	9

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
32987199	DERIVED	PubMed
33822385	DERIVED	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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT02797821

GET https://conductscience.org/api/trials/v1/NCT02797821/events?page=2

API reference For AI assistants