Trial · NCT01163149
Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
NCT01163149, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2017-09-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP). Trials, record NCT01163149. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01163149
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
- NCT number
- NCT01163149
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 19 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Hypophosphatasia
- Interventions
- asfotase alfa, asfotase alfa
- First posted
- 2010-07-15
- Last updated
- 2019-03-13
- Results first posted
- 2017-09-18
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline to Week 24 for Plasma Pyridoxal-5' Phosphate (PLP) | Baseline, Week 24 | Blood samples were collected to evaluate the effect of asfotase alfa on reduction in plasma pyridoxal-5' phosphate (PLP) |
| Primary | Change From Baseline to Week 24 for Plasma Inorganic Pyrophosphate (PPi) | Baseline, Week 24 | Blood samples were collected to evaluate the effect of asfotase alfa on reduction in plasma inorganic pyrophosphate (PPi) |
| Primary | Safety and Tolerability of Asfotase Alfa | Up to 288 weeks exposure to asfotase alfa | The safety and tolerability of daily subcutaneous (SC) injections of asfotase alfa was assessed by routine monitoring of patients for treatment-emergent adverse events (TEAEs) and injection-associated reactions (IARs). |
| Secondary | Change From Baseline in Bone Mineral Content (BMC) as Measured by Dual-energy X-ray Absorptiometry (DXA) | Baseline, every 24 weeks through Week 96, then every 48 weeks until Week 288. | A DXA scan was performed to evaluate bone mineral content (BMC) of the spine, hip, and whole body during the primary (first 24 weeks) and extension treatment periods (up to 288 weeks). |
| Secondary | Change From Baseline in Bone Mineral Density (BMD) as Measured by Dual-energy X-ray Absorptiometry (DXA) | Baseline, every 24 weeks through Week 96, then every 48 weeks until Week 288. | A DXA scan was performed to evaluate bone bone mineral density (BMD) of the spine, hip, and whole body during the primary (first 24 weeks) and extension treatment periods (up to 288 weeks). |
| Secondary | Change in Walking Ability as Measured by the Six-Minute Walk Test (6MWT) | Baseline, Week 24 (primary treatment period) and up to 288 weeks of asfotase alfa exposure | The patient was instructed to walk the length of a pre-measured hallway for 6 minutes. The primary measurement was distance walked (in meters). |
| Secondary | Change From Baseline in HPP-related Osteomalacia as Measured by Trans-iliac Crest Bone Biopsy: Osteoid Volume/Bone Volume | Baseline, Week 24 (Control group), and Week 48 (Asfotase alfa groups). | A trans-iliac crest bone biopsy was performed to quantify changes from Baseline in histomorphometric parameters relevant for evaluation of osteomalacia severity, including Osteoid Volume/Bone Volume (%). The difference in time under observation between asfotase alfa groups (Week 48) and control group (Week 24) resulted from study design, ie, control subjects transitioned to active treatment after the Week 24 visit. |
| Secondary | Change From Baseline in HPP-related Osteomalacia as Measured by Trans-iliac Crest Bone Biopsy: Osteoid Thickness | Baseline, Week 24 (Control group), and Week 48 (Asfotase alfa groups). | A trans-iliac crest bone biopsy was performed to quantify changes from Baseline in histomorphometric parameters relevant for evaluation of osteomalacia severity, including Osteoid Thickness (um). The difference in time under observation between asfotase alfa groups (Week 48) and control group (Week 24) resulted from study design, ie, control subjects transitioned to active treatment after the Week 24 visit. |
| Secondary | Change From Baseline in HPP-related Osteomalacia as Measured by Trans-iliac Crest Bone Biopsy: Mineralization Lag Time | Baseline, Week 24 (Control group), and Week 48 (Asfotase alfa groups). | A trans-iliac crest bone biopsy was performed to quantify changes from Baseline in histomorphometric parameters relevant for evaluation of osteomalacia severity, including Mineralization Lag Time (days). The difference in time under observation between asfotase alfa groups (Week 48) and control group (Week 24) resulted from study design, ie, control subjects transitioned to active treatment after the Week 24 visit. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline to Week 24 for Plasma Pyridoxal-5' Phosphate (PLP) | Baseline, Week 24 | Full analysis set (intent-to-treat, all randomized patients) | ng/mL | MEAN |
| PRIMARY | Change From Baseline to Week 24 for Plasma Inorganic Pyrophosphate (PPi) | Baseline, Week 24 | Full analysis set (intent-to-treat, all randomized patients) | uM | MEAN |
| PRIMARY | Safety and Tolerability of Asfotase Alfa | Up to 288 weeks exposure to asfotase alfa | Safety Set. Control group and asfotase alfa Cohorts during the primary treatment period (first 24 weeks of study); all patients with asfotase alfa exposure during open-label extension treatment period. | Number of Treatment-Emergent Events | NUMBER |
| SECONDARY | Change From Baseline in Bone Mineral Content (BMC) as Measured by Dual-energy X-ray Absorptiometry (DXA) | Baseline, every 24 weeks through Week 96, then every 48 weeks until Week 288. | Full analysis set (intent-to-treat, all randomized patients) | g | MEAN |
| SECONDARY | Change From Baseline in Bone Mineral Density (BMD) as Measured by Dual-energy X-ray Absorptiometry (DXA) | Baseline, every 24 weeks through Week 96, then every 48 weeks until Week 288. | Full analysis set (intent-to-treat, all randomized patients) | g/cm2 | MEAN |
| SECONDARY | Change in Walking Ability as Measured by the Six-Minute Walk Test (6MWT) | Baseline, Week 24 (primary treatment period) and up to 288 weeks of asfotase alfa exposure | Full analysis set (intent-to-treat, all randomized patients). Among the 6 control subjects, only 4 had both a Baseline and Week 24 value. | meters | MEAN |
| SECONDARY | Change From Baseline in HPP-related Osteomalacia as Measured by Trans-iliac Crest Bone Biopsy: Osteoid Volume/Bone Volume | Baseline, Week 24 (Control group), and Week 48 (Asfotase alfa groups). | Full analysis set (intent-to-treat, all randomized patients) | percentage of volume | MEAN |
| SECONDARY | Change From Baseline in HPP-related Osteomalacia as Measured by Trans-iliac Crest Bone Biopsy: Osteoid Thickness | Baseline, Week 24 (Control group), and Week 48 (Asfotase alfa groups). | Full analysis set (intent-to-treat, all randomized patients) | um | MEAN |
| SECONDARY | Change From Baseline in HPP-related Osteomalacia as Measured by Trans-iliac Crest Bone Biopsy: Mineralization Lag Time | Baseline, Week 24 (Control group), and Week 48 (Asfotase alfa groups). | Full analysis set (intent-to-treat, all randomized patients) | days | MEAN |
Source ClinicalTrials.gov
Results by group240
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline to Week 24 for Plasma Pyridoxal-5' Phosphate (PLP) | 0.3 mg/kg Asfotase Alfa | -254.96 | Standard Deviation 196.206 | ng/mL | |
| Change From Baseline to Week 24 for Plasma Pyridoxal-5' Phosphate (PLP) | 0.5 mg/kg Asfotase Alfa | -564.27 | Standard Deviation 624.009 | ng/mL | |
| Change From Baseline to Week 24 for Plasma Pyridoxal-5' Phosphate (PLP) | Concurrent Control | 3.13 | Standard Deviation 242.721 | ng/mL | |
| Change From Baseline to Week 24 for Plasma Pyridoxal-5' Phosphate (PLP) | Combined Asfotase Alfa Group | -397.72 | Standard Deviation 455.249 | ng/mL | |
| Change From Baseline to Week 24 for Plasma Inorganic Pyrophosphate (PPi) | 0.3 mg/kg Asfotase Alfa | -2.027 | Standard Deviation 1.4381 | uM | |
| Change From Baseline to Week 24 for Plasma Inorganic Pyrophosphate (PPi) | 0.5 mg/kg Asfotase Alfa | -2.185 | Standard Deviation 1.3304 | uM | |
| Change From Baseline to Week 24 for Plasma Inorganic Pyrophosphate (PPi) | Concurrent Control | -1.052 | Standard Deviation 2.9248 | uM | |
| Change From Baseline to Week 24 for Plasma Inorganic Pyrophosphate (PPi) | Combined Asfotase Alfa Group | -2.100 | Standard Deviation 1.3335 | uM | |
| Safety and Tolerability of Asfotase Alfa | 0.3 mg/kg Asfotase Alfa | Any TEAE | 243 | Number of Treatment-Emergent Events | |
| Safety and Tolerability of Asfotase Alfa | 0.5 mg/kg Asfotase Alfa | Any TEAE | 81 | Number of Treatment-Emergent Events |
1 to 10 of 240Source ClinicalTrials.gov
See all 240 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Treatment Period | STARTED | 0.3 mg/kg Asfotase Alfa | 7 | |
| Primary Treatment Period | STARTED | 0.5 mg/kg Asfotase Alfa | 6 | |
| Primary Treatment Period | STARTED | Concurrent Control | 6 | |
| Primary Treatment Period | COMPLETED | 0.3 mg/kg Asfotase Alfa | 7 | |
| Primary Treatment Period | COMPLETED | 0.5 mg/kg Asfotase Alfa | 6 | |
| Primary Treatment Period | COMPLETED | Concurrent Control | 6 | |
| Primary Treatment Period | NOT COMPLETED | 0.3 mg/kg Asfotase Alfa | 0 | |
| Primary Treatment Period | NOT COMPLETED | 0.5 mg/kg Asfotase Alfa | 0 | |
| Primary Treatment Period | NOT COMPLETED | Concurrent Control | 0 | |
| Extension Treatment Period | STARTED | 0.3 mg/kg Asfotase Alfa | 7 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events1,260
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 | 0.3 mg/kg Asfotase Alfa (First 24 Weeks) | 3 | 7 | 61 |
| other | Injection Site Erythema | General disorders | 0.5 mg/kg Asfotase Alfa (First 24 Weeks) | 2 | 6 | 5 |
| other | Injection Site Erythema | General disorders | Concurrent Control (First 24 Weeks) | 0 | 6 | 0 |
| other | Injection Site Erythema | General disorders | Cumulative Exposure to Asfotase Alfa | 13 | 19 | 121 |
| other | Injection Site Haematoma | General disorders | 0.3 mg/kg Asfotase Alfa (First 24 Weeks) | 3 | 7 | 14 |
| other | Injection Site Haematoma | General disorders | 0.5 mg/kg Asfotase Alfa (First 24 Weeks) | 1 | 6 | 6 |
| other | Injection Site Haematoma | General disorders | Concurrent Control (First 24 Weeks) | 0 | 6 | 0 |
| other | Injection Site Haematoma | General disorders | Cumulative Exposure to Asfotase Alfa | 10 | 19 | 33 |
| other | Injection Site Pain | General disorders | 0.3 mg/kg Asfotase Alfa (First 24 Weeks) | 4 | 7 | 20 |
| other | Injection Site Pain | General disorders | 0.5 mg/kg Asfotase Alfa (First 24 Weeks) | 0 | 6 | 0 |
1 to 10 of 1,260Source ClinicalTrials.gov
See all 1,260 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | 0.3 mg/kg Asfotase Alfa (First 24 Weeks) | 0 | 7 |
| Total, all-cause mortality | 0.5 mg/kg Asfotase Alfa (First 24 Weeks) | 0 | 6 |
| Total, all-cause mortality | Concurrent Control (First 24 Weeks) | 0 | 6 |
| Total, all-cause mortality | Cumulative Exposure to Asfotase Alfa | 0 | 19 |
| Total, other adverse events | 0.3 mg/kg Asfotase Alfa (First 24 Weeks) | 7 | 7 |
| Total, other adverse events | 0.5 mg/kg Asfotase Alfa (First 24 Weeks) | 6 | 6 |
| Total, other adverse events | Concurrent Control (First 24 Weeks) | 6 | 6 |
| Total, other adverse events | Cumulative Exposure to Asfotase Alfa | 19 | 19 |
| Total, serious adverse events | 0.3 mg/kg Asfotase Alfa (First 24 Weeks) | 1 | 7 |
| Total, serious adverse events | 0.5 mg/kg Asfotase Alfa (First 24 Weeks) | 1 | 6 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers3
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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