Trial · NCT01203826

Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)

NCT01203826, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2017-07-26

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP). Trials, record NCT01203826. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01203826

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Trial

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

Title: Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
NCT number: NCT01203826
Status: Completed
Phases: Phase 2
Enrollment: 12 participants, actual
Lead sponsor: Alexion Pharmaceuticals, Inc.
Conditions: Hypophosphatasia (HPP)
Interventions: Asfotase Alfa
First posted: 2010-09-16
Last updated: 2019-03-13
Results first posted: 2017-07-26

Source ClinicalTrials.gov

Registered outcomes1

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

List	Outcome	Time frame	Description
Primary	Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09.	At least 72 months of treatment with asfotase alfa	Evaluation of radiographic change in rickets severity (as assessed by skeletal radiographs of the hands/wrists and knees) from the Baseline of Study ENB-006-09 (NCT00952484) to the End of Study (EOS) visit in Study ENB-008-10 using an ordinal RGI-C scale score. 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 points will be pre-treatment (Baseline from Study ENB-006-09) to the last radiographic assessment in Study ENB-008-10, which represents at least 72 months of treatment.

Source ClinicalTrials.gov

Outcomes1

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09.	At least 72 months of treatment with asfotase alfa	Each visit in Study ENB-008-10 was calculated relative to the start of exposure to asfotase alfa in Study ENB-006-09 (NCT00952484); 24 weeks are added to each visit in Study ENB-008-10. Results shown are for the last assessment in Study ENB-008-10 and represent at least 72 months of treatment with asfotase alfa.	units on a scale	MEDIAN

Source ClinicalTrials.gov

Results by group1

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

Outcome	Group	Category	Value	Spread	Units
Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09.	Asfotase Alfa Combined		2.83	Full Range 2.0 to 3.0	units on a scale

Source ClinicalTrials.gov

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	2 mg/kg Asfotase Alfa	6
Overall Study	STARTED	3 mg/kg Asfotase Alfa	6
Overall Study	COMPLETED	2 mg/kg Asfotase Alfa	6
Overall Study	COMPLETED	3 mg/kg Asfotase Alfa	6
Overall Study	NOT COMPLETED	2 mg/kg Asfotase Alfa	0
Overall Study	NOT COMPLETED	3 mg/kg Asfotase Alfa	0

Source ClinicalTrials.gov

Adverse events324

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	2 mg/kg Asfotase Alfa	4	6	18
other	Injection site erythema	General disorders	3 mg/kg Asfotase Alfa	6	6	27
other	Injection site erythema	General disorders	Combined Asfotase Alfa Group	10	12	45
other	Injection site macule	General disorders	2 mg/kg Asfotase Alfa	4	6	28
other	Injection site macule	General disorders	3 mg/kg Asfotase Alfa	5	6	31
other	Injection site macule	General disorders	Combined Asfotase Alfa Group	9	12	59
other	Injection site hypertrophy	General disorders	2 mg/kg Asfotase Alfa	4	6	14
other	Injection site hypertrophy	General disorders	3 mg/kg Asfotase Alfa	4	6	13
other	Injection site hypertrophy	General disorders	Combined Asfotase Alfa Group	8	12	27
other	Injection site atrophy	General disorders	2 mg/kg Asfotase Alfa	3	6	10

1 to 10 of 324Source ClinicalTrials.gov

See all 324 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	2 mg/kg Asfotase Alfa	0	6
Total, all-cause mortality	3 mg/kg Asfotase Alfa	0	6
Total, all-cause mortality	Combined Asfotase Alfa Group	0	12
Total, other adverse events	2 mg/kg Asfotase Alfa	6	6
Total, other adverse events	3 mg/kg Asfotase Alfa	6	6
Total, other adverse events	Combined Asfotase Alfa Group	12	12
Total, serious adverse events	2 mg/kg Asfotase Alfa	0	6
Total, serious adverse events	3 mg/kg Asfotase Alfa	0	6
Total, serious adverse events	Combined Asfotase Alfa Group	0	12

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
27699270	DERIVED	PubMed
32417537	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/NCT01203826

GET https://conductscience.org/api/trials/v1/NCT01203826/events?page=2

API reference For AI assistants