Trial · NCT01176266
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
NCT01176266, Phase 2, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2018-02-26
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP). Trials, record NCT01176266. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01176266
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
- NCT number
- NCT01176266
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 69 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Hypophosphatasia
- Interventions
- asfotase alfa
- First posted
- 2010-08-05
- Last updated
- 2019-03-13
- Results first posted
- 2018-02-26
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Effect of Asfotase Alfa Treatment on Skeletal Manifestations of Hypophosphatasia (HPP) | From Baseline to Week 24 | The effect of asfotase alfa treatment on skeletal manifestations of HPP (i.e., change in rickets severity) was measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale. Skeletal radiographs obtained at Week 24 were compared with skeletal radiographs obtained before initiation of treatment. The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP-associated rickets) to +3 (indicative of complete or near complete healing of HPP-associated rickets). |
| Primary | Safety and Tolerability of Repeated Subcutaneous (SC) Injections of Asfotase Alfa | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Safety and tolerability of repeated subcutaneous (SC) injections of asfotase alfa for all treated patients was assessed by the number of patients with 1 or more treatment-emergent adverse event. |
| Secondary | Effect of Asfotase Alfa Treatment on Skeletal Manifestations of Hypophosphatasia (HPP) | Up to 72 Months or regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | The effect of asfotase alfa treatment on skeletal manifestations of HPP (i.e., change in rickets severity) was measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale. Skeletal radiographs obtained at the patient's last assessment were compared with skeletal radiographs obtained before initiation of treatment. The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP-associated rickets) to +3 (indicative of complete or near complete healing of HPP-associated rickets). |
| Secondary | Effect of Asfotase Alfa Treatment on Ventilator-free Survival (Week 312) | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | For patients who were not on respiratory support at the time of enrollment, the Kaplan-Meier estimate of ventilator-free survival at the end of the study |
| Secondary | Effect of Asfotase Alfa Treatment on Respiratory Function | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Effect of asfotase alfa treatment on respiratory function as measured by the shift in proportion of patients requiring respiratory support at their last assessment compared with Baseline. |
| Secondary | Effect of Asfotase Alfa Treatment on Physical Growth - Length/Height Z-scores Change From Baseline to Last Obtained Value | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Effect of asfotase alfa treatment on physical growth as measured by change from Baseline to last assessment for each patient in length/height Z-scores |
| Secondary | Effect of Asfotase Alfa Treatment on Physical Growth - Weight Z-scores Change From Baseline to Last Obtained Value | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Effect of asfotase alfa treatment on physical growth as measured by change from Baseline to last assessment for each patient in weight Z-scores |
| Secondary | Effect of Asfotase Alfa on Biomarkers - Plasma Inorganic Pyrophosphate (PPi) Change From Baseline to Last Obtained Value | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Effect of asfotase alfa on PPi as measured by change from Baseline to last assessment for each patient |
| Secondary | Effect of Asfotase Alfa on Biomarkers - Plasma Pyridoxal-5' Phosphate (PLP) Change From Baseline to Last Obtained Value | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Effect of asfotase alfa on PLP as measured by change from Baseline to last assessment for each patient |
| Secondary | Effect of Asfotase Alfa on Serum Parathyroid Hormone (PTH) - Change From Baseline to Last Obtained Value | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Effect of asfotase alfa on serum PTH as measured by change from Baseline to last assessment for each patient |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Effect of Asfotase Alfa Treatment on Skeletal Manifestations of Hypophosphatasia (HPP) | From Baseline to Week 24 | Full Analysis Set | scores on a scale | MEDIAN |
| PRIMARY | Safety and Tolerability of Repeated Subcutaneous (SC) Injections of Asfotase Alfa | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Full Analysis Set | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Effect of Asfotase Alfa Treatment on Skeletal Manifestations of Hypophosphatasia (HPP) | Up to 72 Months or regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Full Analysis Set | scores on a scale | MEDIAN |
| SECONDARY | Effect of Asfotase Alfa Treatment on Ventilator-free Survival (Week 312) | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Patients on respiratory support at Baseline were excluded from the analysis. | Probability | NUMBER |
| SECONDARY | Effect of Asfotase Alfa Treatment on Respiratory Function | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Full Analysis Set | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Effect of Asfotase Alfa Treatment on Physical Growth - Length/Height Z-scores Change From Baseline to Last Obtained Value | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Full Analysis Set | Z-score | MEDIAN |
| SECONDARY | Effect of Asfotase Alfa Treatment on Physical Growth - Weight Z-scores Change From Baseline to Last Obtained Value | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Full Analysis Set | Z-score | MEDIAN |
| SECONDARY | Effect of Asfotase Alfa on Biomarkers - Plasma Inorganic Pyrophosphate (PPi) Change From Baseline to Last Obtained Value | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Full Analysis Set | uM | MEDIAN |
| SECONDARY | Effect of Asfotase Alfa on Biomarkers - Plasma Pyridoxal-5' Phosphate (PLP) Change From Baseline to Last Obtained Value | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Full Analysis Set | ng/mL | MEDIAN |
| SECONDARY | Effect of Asfotase Alfa on Serum Parathyroid Hormone (PTH) - Change From Baseline to Last Obtained Value | Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years). | Full Analysis Set | pmol/L | MEDIAN |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group32
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Effect of Asfotase Alfa Treatment on Skeletal Manifestations of Hypophosphatasia (HPP) | Asfotase Alfa | 2.00 | Full Range -1.67 to 3.00 | scores on a scale | |
| Safety and Tolerability of Repeated Subcutaneous (SC) Injections of Asfotase Alfa | Asfotase Alfa | 69 | Participants | ||
| Effect of Asfotase Alfa Treatment on Skeletal Manifestations of Hypophosphatasia (HPP) | Asfotase Alfa | 2.33 | Full Range -2.67 to 3.00 | scores on a scale | |
| Effect of Asfotase Alfa Treatment on Ventilator-free Survival (Week 312) | Asfotase Alfa | 0.81 | Probability | ||
| Effect of Asfotase Alfa Treatment on Respiratory Function | Baseline | No respiratory support | 45 | Participants | |
| Effect of Asfotase Alfa Treatment on Respiratory Function | Asfotase Alfa - Without Respiratory Support at Baseline | No respiratory support | 43 | Participants | |
| Effect of Asfotase Alfa Treatment on Respiratory Function | Asfotase Alfa - With Respiratory Support at Baseline | No respiratory support | 11 | Participants | |
| Effect of Asfotase Alfa Treatment on Respiratory Function | Baseline | Supplemental oxygen | 6 | Participants | |
| Effect of Asfotase Alfa Treatment on Respiratory Function | Asfotase Alfa - Without Respiratory Support at Baseline | Supplemental oxygen | 1 | Participants | |
| Effect of Asfotase Alfa Treatment on Respiratory Function | Asfotase Alfa - With Respiratory Support at Baseline | Supplemental oxygen | 2 | Participants |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Asfotase Alfa | 69 | |
| Overall Study | COMPLETED | Asfotase Alfa | 60 | |
| Overall Study | NOT COMPLETED | Asfotase Alfa | 9 |
Source ClinicalTrials.gov
Adverse events237
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 | Nasopharyngitis | Infections and infestations | Asfotase Alfa | 18 | 69 | 52 |
| other | Upper respiratory tract infection | Infections and infestations | Asfotase Alfa | 17 | 69 | 31 |
| other | Gastroenteritis | Infections and infestations | Asfotase Alfa | 16 | 69 | 20 |
| other | Respiratory tract infection | Infections and infestations | Asfotase Alfa | 16 | 69 | 62 |
| other | Ear infection | Infections and infestations | Asfotase Alfa | 13 | 69 | 18 |
| other | Rhinitis | Infections and infestations | Asfotase Alfa | 10 | 69 | 17 |
| other | Bronchitis | Infections and infestations | Asfotase Alfa | 9 | 69 | 16 |
| other | Urinary tract infection | Infections and infestations | Asfotase Alfa | 8 | 69 | 8 |
| other | Influenza | Infections and infestations | Asfotase Alfa | 7 | 69 | 8 |
| other | Pneumonia | Infections and infestations | Asfotase Alfa | 7 | 69 | 9 |
1 to 10 of 237Source ClinicalTrials.gov
See all 237 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Asfotase Alfa | 9 | 69 |
| Total, other adverse events | Asfotase Alfa | 68 | 69 |
| Total, serious adverse events | Asfotase Alfa | 50 | 69 |
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 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.
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