Trial · NCT01205152
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
NCT01205152, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2017-11-17
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP). Trials, record NCT01205152. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01205152
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
- NCT number
- NCT01205152
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 10 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Hypophosphatasia
- Interventions
- asfotase alfa
- First posted
- 2010-09-20
- Last updated
- 2019-03-13
- Results first posted
- 2017-11-17
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Long-term Tolerability of Subcutaneous (SC) Asfotase Alfa | 84 months | Outcome measure is the number of patients with 1 or more treatment-emergent adverse event. The time period is from Baseline in the ENB-003-08 study to the end of the ENB-003-08 study. |
| Primary | Long-term Efficacy of Asfotase Alfa in Treating Rickets in Infants and Young Children With Hypophosphatasia (HPP). | Up to 90 Months | Outcome measure is the evaluation of radiographic change in rickets severity 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 (Baseline in Study ENB-002-08 \[NCT00744042\]). 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). The time period is pre-dose (Baseline from ENB-002-08 study) to the last assessment for each patient in the ENB-003-08 study, which represents up to 90 months of exposure for the combined studies. |
| Secondary | Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Plasma Inorganic Pyrophosphate (PPi) Levels | Up to 90 Months | Outcome measure is the change from Baseline in plasma inorganic pyrophosphate (PPi) levels. The time period is pre-dose (Baseline from the ENB-002-08 study \[NCT00744042\]) to the last assessment for each patient in the ENB-003-08 study, which represents up to 90 months of exposure for the combined studies. |
| Secondary | Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Pyridoxal-5-phosphate (PLP) Levels | Up to 90 Months | Outcome measure is the change from Baseline in pyridoxal-5-phosphate (PLP) levels. The time period is pre-dose (Baseline from the ENB-002-08 study \[NCT00744042\]) to the last assessment for each patient in the ENB-003-08 study, which represents up to 90 months of exposure for the combined studies. |
| Secondary | Effect of SC Asfotase Alfa on Growth: Weight Z-scores | Up to 90 Months | Outcome measure is the change from Baseline in Z-scores for weight. The time period is pre-dose (Baseline from the ENB-002-08 study \[NCT00744042\]) to the last assessment in the ENB-003-08 study, which represents up to 90 months of exposure in the combined studies. |
| Secondary | Effect of SC Asfotase Alfa on Growth: Height/Length Z-scores | Up to 90 Months | Outcome measure is the change from Baseline in Z-scores for height/length. The time period is pre-dose (Baseline from the ENB-002-08 study \[NCT00744042\]) to the last assessment in the ENB-003-08 study, which represents up to 90 months of exposure in the combined studies. |
| Secondary | Effect of SC Asfotase Alfa on Respiratory Function | Up to 90 Months | Outcome measure is the shift in the proportion of patients requiring respiratory support at their last assessment in Study ENB-003-08 compared with Baseline. The time period is pre-dose (Baseline from the ENB-002-08 study \[NCT00744042\]) to the last assessment in the ENB-003-08 study, which represents up to 90 months of exposure in the combined studies. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Long-term Tolerability of Subcutaneous (SC) Asfotase Alfa | 84 months | Full Analysis Set (All 10 patients enrolled in Study ENB-003-08) | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Long-term Efficacy of Asfotase Alfa in Treating Rickets in Infants and Young Children With Hypophosphatasia (HPP). | Up to 90 Months | Full Analysis Set (All 10 patients enrolled in Study ENB-003-08) | scores on a scale | MEDIAN |
| SECONDARY | Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Plasma Inorganic Pyrophosphate (PPi) Levels | Up to 90 Months | Full Analysis Set (All 10 patients enrolled in Study ENB-003-08). Change from Baseline could not be calculated for 3 patients due to non-evaluable samples at Baseline. | uM | MEDIAN |
| SECONDARY | Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Pyridoxal-5-phosphate (PLP) Levels | Up to 90 Months | Full Analysis Set (All 10 patients enrolled in Study ENB-003-08). Change from Baseline could not be calculated for 2 patients because of non-evaluable samples at Baseline. | ng/mL | MEDIAN |
| SECONDARY | Effect of SC Asfotase Alfa on Growth: Weight Z-scores | Up to 90 Months | Full Analysis Set (All 10 patients enrolled in Study ENB-003-08) | Z-score | MEDIAN |
| SECONDARY | Effect of SC Asfotase Alfa on Growth: Height/Length Z-scores | Up to 90 Months | Full Analysis Set (All 10 patients enrolled in Study ENB-003-08) | Z-score | MEDIAN |
| SECONDARY | Effect of SC Asfotase Alfa on Respiratory Function | Up to 90 Months | Full Analysis Set (All 10 patients enrolled in Study ENB-003-08) | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Long-term Tolerability of Subcutaneous (SC) Asfotase Alfa | Asfotase Alfa | 10 | Participants | ||
| Long-term Efficacy of Asfotase Alfa in Treating Rickets in Infants and Young Children With Hypophosphatasia (HPP). | Asfotase Alfa | 2.00 | Full Range 2.00 to 3.00 | scores on a scale | |
| Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Plasma Inorganic Pyrophosphate (PPi) Levels | Asfotase Alfa | -2.460 | Full Range -9.73 to 2.72 | uM | |
| Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Pyridoxal-5-phosphate (PLP) Levels | Asfotase Alfa | -266.200 | Full Range -844.30 to 184.00 | ng/mL | |
| Effect of SC Asfotase Alfa on Growth: Weight Z-scores | Asfotase Alfa | 2.43 | Full Range -2.9 to 5.2 | Z-score | |
| Effect of SC Asfotase Alfa on Growth: Height/Length Z-scores | Asfotase Alfa | 1.93 | Full Range -3.2 to 4.6 | Z-score | |
| Effect of SC Asfotase Alfa on Respiratory Function | Asfotase Alfa (Baseline ENB-002-08) | No respiratory support | 6 | Participants | |
| Effect of SC Asfotase Alfa on Respiratory Function | Asfotase Alfa (Last Assessment) | No respiratory support | 9 | Participants | |
| Effect of SC Asfotase Alfa on Respiratory Function | Asfotase Alfa (Baseline ENB-002-08) | Supplemental oxygen | 0 | Participants | |
| Effect of SC Asfotase Alfa on Respiratory Function | Asfotase Alfa (Last Assessment) | Supplemental oxygen | 0 | Participants |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 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 | 10 | |
| Overall Study | COMPLETED | Asfotase Alfa | 9 | |
| Overall Study | NOT COMPLETED | Asfotase Alfa | 1 |
Source ClinicalTrials.gov
Adverse events222
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 | Anaemia | Blood and lymphatic system disorders | Asfotase Alfa | 1 | 10 | 1 |
| other | Leukopenia | Blood and lymphatic system disorders | Asfotase Alfa | 1 | 10 | 1 |
| other | Microcytosis | Blood and lymphatic system disorders | Asfotase Alfa | 1 | 10 | 1 |
| other | Neutropenia | Blood and lymphatic system disorders | Asfotase Alfa | 1 | 10 | 1 |
| other | Craniosynostosis | Congenital, familial and genetic disorders | Asfotase Alfa | 2 | 10 | 5 |
| other | Deafness | Ear and labyrinth disorders | Asfotase Alfa | 1 | 10 | 1 |
| other | Eustachian tube dysfunction | Ear and labyrinth disorders | Asfotase Alfa | 1 | 10 | 1 |
| other | Middle ear disorder | Ear and labyrinth disorders | Asfotase Alfa | 1 | 10 | 1 |
| other | Otorrhoea | Ear and labyrinth disorders | Asfotase Alfa | 1 | 10 | 1 |
| other | Tympanic membrane perforation | Ear and labyrinth disorders | Asfotase Alfa | 1 | 10 | 1 |
1 to 10 of 222Source ClinicalTrials.gov
See all 222 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Asfotase Alfa | 1 | 10 |
| Total, other adverse events | Asfotase Alfa | 10 | 10 |
| Total, serious adverse events | Asfotase Alfa | 9 | 10 |
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.
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/NCT01205152
GET https://conductscience.org/api/trials/v1/NCT01205152/events?page=2