Trial · NCT00952484
Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
NCT00952484, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2011-07-26
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP). Trials, record NCT00952484. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00952484
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
- NCT number
- NCT00952484
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 13 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Hypophosphatasia (HPP)
- Interventions
- asfotase alfa, asfotase alfa
- First posted
- 2009-08-06
- Last updated
- 2019-04-01
- Results first posted
- 2011-07-26
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Rickets Severity on Skeletal Radiographs From Baseline to Week 24 as Measured by the Radiographic Global Impression of Change (RGI-C) Scale | Baseline and Week 24 | A 7-point RGI-C (radiographic global impression of change) score was used to rate change in rickets severity. Only those patients with a minimum score of +2 indicating substantial healing of rickets) were considered responders. Three pediatric radiologists not affiliated with the conduct of the study performed the ratings. |
| Secondary | Change in Osteomalacia - Osteoid Thickness (as Measured by Trans-iliac Crest Bone Biopsy) | Baseline and Week 24 | Change from Baseline to Week 24 in osteoid thickness. |
| Secondary | Change in Osteomalacia - Osteoid Volume/Bone Volume (as Measured by Trans-iliac Crest Bone Biopsy) | Baseline and Week 24 | Change from Baseline to Week 24 in osteoid volume/bone volume (%), calculated as the absolute difference of the Baseline and Week 24 percentages. |
| Secondary | Change in Osteomalacia - Mineralization Lag Time (as Measured by Trans-iliac Crest Bone Biopsy) | Baseline and Week 24 | Change from Baseline to Week 24 in mineralization lag time. |
| Secondary | Change in Height (Z-scores) | Baseline and Week 24 | Change from Baseline to Week 24 in Height Z-Score. Height Z-Scores assigned based on Centers for Disease Control (CDC) growth charts and methodology. |
| Secondary | Change in Biomarkers of Asfotase Alfa Activity as Measured by Plasma Inorganic Pyrophosphate (PPi) | Baseline and Week 24 | Change from Baseline to Week 24 in Plasma PPi |
| Secondary | Change in Biomarkers of Asfotase Alfa Activity as Measured by Pyridoxal-5'-Phosphate (PLP) | Baseline and Week 24 | Change from Baseline to Week 24 in Plasma PLP |
| Secondary | Maximum Serum Concentration of Asfotase Alfa (Cmax). | Study Week 1 (0 to 48 hours post-dose) | Maximum serum concentration observed following single dose of asfotase alfa. |
| Secondary | Time at Maximum Serum Concentration of Asfotase Alfa (Tmax) | Study Week 1 (0 to 48 hours post-dose) | Maximum serum concentration observed following single dose of asfotase alfa. |
| Secondary | Area Under Serum Concentration-time Curve to Last Measurable Concentration of Asfotase Alfa (AUCt) | Study Week 1 (0 to 48 hours post-dose) | Area under serum concentration-time curve to last measurable concentration following single dose of asfotase alfa. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Rickets Severity on Skeletal Radiographs From Baseline to Week 24 as Measured by the Radiographic Global Impression of Change (RGI-C) Scale | Baseline and Week 24 | ITT population, which included randomized patients that received treatment with asfotase alfa, even if discontinued or lost to follow-up. The last assessment prior to Week 24 is used for missing Week 24 data; patients with no post-baseline assessments imputed as having no change. A historical control group is used for comparison. | units on a scale | MEDIAN |
| SECONDARY | Change in Osteomalacia - Osteoid Thickness (as Measured by Trans-iliac Crest Bone Biopsy) | Baseline and Week 24 | ITT population, which included all randomized patients that received any treatment with asfotase alfa (2 mg/kg or 3 mg/kg thrice weekly), even if they discontinued or were lost to follow-up during the conduct of the clinical trial. Data imputation was not performed. | um | MEAN |
| SECONDARY | Change in Osteomalacia - Osteoid Volume/Bone Volume (as Measured by Trans-iliac Crest Bone Biopsy) | Baseline and Week 24 | ITT population, which included all randomized patients that received any treatment with asfotase alfa (2 mg/kg or 3 mg/kg thrice weekly), even if they discontinued or were lost to follow-up during the conduct of the clinical trial. Data imputation was not performed. | percentage points | MEAN |
| SECONDARY | Change in Osteomalacia - Mineralization Lag Time (as Measured by Trans-iliac Crest Bone Biopsy) | Baseline and Week 24 | ITT population, which included all randomized patients that received any treatment with asfotase alfa (2 mg/kg or 3 mg/kg thrice weekly), even if they discontinued or were lost to follow-up during the conduct of the clinical trial. Data imputation was not performed. | days | MEAN |
| SECONDARY | Change in Height (Z-scores) | Baseline and Week 24 | ITT population, which included all randomized patients that received any treatment with asfotase alfa (2 mg/kg or 3 mg/kg thrice weekly), even if they discontinued or were lost to follow-up during the conduct of the clinical trial. Data imputation was not performed. | Height Z score | MEAN |
| SECONDARY | Change in Biomarkers of Asfotase Alfa Activity as Measured by Plasma Inorganic Pyrophosphate (PPi) | Baseline and Week 24 | ITT population, which included all randomized patients that received any treatment with asfotase alfa (2 mg/kg or 3 mg/kg thrice weekly), even if they discontinued or were lost to follow-up during the conduct of the clinical trial. Data imputation was not performed. | uM | MEAN |
| SECONDARY | Change in Biomarkers of Asfotase Alfa Activity as Measured by Pyridoxal-5'-Phosphate (PLP) | Baseline and Week 24 | ITT population, which included all randomized patients that received any treatment with asfotase alfa (2 mg/kg or 3 mg/kg thrice weekly), even if they discontinued or were lost to follow-up during the conduct of the clinical trial. Data imputation was not performed. | ng/mL | MEAN |
| SECONDARY | Maximum Serum Concentration of Asfotase Alfa (Cmax). | Study Week 1 (0 to 48 hours post-dose) | ITT population which included all randomized patients that received any treatment with asfotase alfa (2 mg/kg or 3 mg/kg thrice weekly). | U/L | MEAN |
| SECONDARY | Time at Maximum Serum Concentration of Asfotase Alfa (Tmax) | Study Week 1 (0 to 48 hours post-dose) | ITT population which included all randomized patients that received any treatment with asfotase alfa (2 mg/kg or 3 mg/kg thrice weekly). | hours | MEAN |
| SECONDARY | Area Under Serum Concentration-time Curve to Last Measurable Concentration of Asfotase Alfa (AUCt) | Study Week 1 (0 to 48 hours post-dose) | ITT population which included all randomized patients that received any treatment with asfotase alfa (2 mg/kg or 3 mg/kg thrice weekly). | h*U/L | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Rickets Severity on Skeletal Radiographs From Baseline to Week 24 as Measured by the Radiographic Global Impression of Change (RGI-C) Scale | Historical Controls | 0 | Full Range -1.3 to 2.0 | units on a scale | |
| Change in Rickets Severity on Skeletal Radiographs From Baseline to Week 24 as Measured by the Radiographic Global Impression of Change (RGI-C) Scale | Asfotase Alfa Combined | 2.00 | Full Range 0.0 to 2.3 | units on a scale | |
| Change in Osteomalacia - Osteoid Thickness (as Measured by Trans-iliac Crest Bone Biopsy) | Asfotase Alfa Combined | -3.858 | Standard Deviation 4.2784 | um | |
| Change in Osteomalacia - Osteoid Volume/Bone Volume (as Measured by Trans-iliac Crest Bone Biopsy) | Asfotase Alfa Combined | -4.225 | Standard Deviation 7.5582 | percentage points | |
| Change in Osteomalacia - Mineralization Lag Time (as Measured by Trans-iliac Crest Bone Biopsy) | Asfotase Alfa Combined | 20.392 | Standard Deviation 208.9814 | days | |
| Change in Height (Z-scores) | Asfotase Alfa Combined | 0.01 | Standard Deviation 0.141 | Height Z score | |
| Change in Biomarkers of Asfotase Alfa Activity as Measured by Plasma Inorganic Pyrophosphate (PPi) | Asfotase Alfa Combined | -1.883 | Standard Deviation 0.7285 | uM | |
| Change in Biomarkers of Asfotase Alfa Activity as Measured by Pyridoxal-5'-Phosphate (PLP) | Asfotase Alfa Combined | -164.533 | Standard Deviation 121.4840 | ng/mL | |
| Maximum Serum Concentration of Asfotase Alfa (Cmax). | 2 mg/kg Asfotase Alfa | 566 | Standard Deviation 120 | U/L | |
| Maximum Serum Concentration of Asfotase Alfa (Cmax). | 3 mg/kg Asfotase Alfa | 1260 | Standard Deviation 439 | U/L |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | 2 mg/kg Asfotase Alfa | 6 | |
| Overall Study | STARTED | 3 mg/kg Asfotase Alfa | 7 | |
| Overall Study | STARTED | Historical Control | 16 | |
| Overall Study | COMPLETED | 2 mg/kg Asfotase Alfa | 6 | |
| Overall Study | COMPLETED | 3 mg/kg Asfotase Alfa | 6 | |
| Overall Study | COMPLETED | Historical Control | 16 | |
| Overall Study | NOT COMPLETED | 2 mg/kg Asfotase Alfa | 0 | |
| Overall Study | NOT COMPLETED | 3 mg/kg Asfotase Alfa | 1 | |
| Overall Study | NOT COMPLETED | Historical Control | 0 |
Source ClinicalTrials.gov
Adverse events122
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 | Injection site erythema | General disorders | Asfotase Alfa 2 mg/kg | 5 | 6 | 13 |
| other | Injection site erythema | General disorders | Asfotase Alfa 3 mg/kg | 7 | 7 | 16 |
| other | Injection site discoloration | General disorders | Asfotase Alfa 2 mg/kg | 4 | 6 | 4 |
| other | Injection site discoloration | General disorders | Asfotase Alfa 3 mg/kg | 4 | 7 | 4 |
| other | Injection site pruritis | General disorders | Asfotase Alfa 2 mg/kg | 4 | 6 | 8 |
| other | Injection site pruritis | General disorders | Asfotase Alfa 3 mg/kg | 3 | 7 | 3 |
| other | Injection site pain | General disorders | Asfotase Alfa 2 mg/kg | 3 | 6 | 5 |
| other | Injection site pain | General disorders | Asfotase Alfa 3 mg/kg | 3 | 7 | 5 |
| other | Injection site swelling | General disorders | Asfotase Alfa 2 mg/kg | 1 | 6 | 3 |
| other | Injection site swelling | General disorders | Asfotase Alfa 3 mg/kg | 2 | 7 | 2 |
1 to 10 of 122Source ClinicalTrials.gov
See all 122 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Asfotase Alfa 2 mg/kg | ||
| Total, all-cause mortality | Asfotase Alfa 3 mg/kg | ||
| Total, other adverse events | Asfotase Alfa 2 mg/kg | 6 | 6 |
| Total, other adverse events | Asfotase Alfa 3 mg/kg | 7 | 7 |
| Total, serious adverse events | Asfotase Alfa 2 mg/kg | 0 | 6 |
| Total, serious adverse events | Asfotase Alfa 3 mg/kg | 0 | 7 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Asfotase Alfa 2 mg/kg vs Asfotase Alfa 3 mg/kg: serious adverse events |
Source Trial comparisons
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/NCT00952484
GET https://conductscience.org/api/trials/v1/NCT00952484/events?page=2