Trial · NCT01132690
A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
NCT01132690, Phase 4, Completed, Pfizer; results first posted 2014-11-11
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease. Trials, record NCT01132690. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01132690
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
- NCT number
- NCT01132690
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 11 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Gaucher Disease
- Interventions
- Taliglucerase alfa
- First posted
- 2010-05-28
- Last updated
- 2018-10-05
- Results first posted
- 2014-11-11
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Hemoglobin | Every 3 months for 12 months | median and interquartile range for change from baseline in haemoglobin |
| Secondary | Chitotriosidase | Every 3 months for 12 months | Percent change from baseline in chitotriosidase |
| Secondary | Spleen Volume | Baseline and Month 12 | Spleen volume measured by MRI |
| Secondary | Platelet Count | Baseline and 12 months | Mean and standard deviation of platelet count per cubic mm |
| Secondary | Chemokine (C-C Motif) Ligand 18 (CCL18) | Every 3 months for 12 months | Percent change from baseline in CCL18 |
| Secondary | Liver Volume | Baseline and Month 12 | Liver volume measured by MRI |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Hemoglobin | Every 3 months for 12 months | g/dL | MEDIAN | |
| SECONDARY | Chitotriosidase | Every 3 months for 12 months | Percent Change from Baseline | MEAN | |
| SECONDARY | Spleen Volume | Baseline and Month 12 | mL | MEAN | |
| SECONDARY | Platelet Count | Baseline and 12 months | platelets per cubic mm | MEAN | |
| SECONDARY | Chemokine (C-C Motif) Ligand 18 (CCL18) | Every 3 months for 12 months | Percent Change from Baseline | MEAN | |
| SECONDARY | Liver Volume | Baseline and Month 12 | mL | MEAN |
Source ClinicalTrials.gov
Results by group36
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Hemoglobin | 30 Units/kg | Month 3 | 1.2 | Inter-Quartile Range -0.3 to 1.6 | g/dL |
| Hemoglobin | 60 Units/kg | Month 3 | 0.4 | Inter-Quartile Range -0.1 to 1.1 | g/dL |
| Hemoglobin | 30 Units/kg | Month 6 | 1.1 | Inter-Quartile Range 0.3 to 2.3 | g/dL |
| Hemoglobin | 60 Units/kg | Month 6 | 1.5 | Inter-Quartile Range 1.0 to 1.7 | g/dL |
| Hemoglobin | 30 Units/kg | Month 9 | 0.9 | Inter-Quartile Range 0.5 to 1.9 | g/dL |
| Hemoglobin | 60 Units/kg | Month 9 | 1.5 | Inter-Quartile Range 1.4 to 1.6 | g/dL |
| Hemoglobin | 30 Units/kg | Month 12 | 1.4 | Inter-Quartile Range 0.2 to 2.5 | g/dL |
| Hemoglobin | 60 Units/kg | Month 12 | 1.6 | Inter-Quartile Range 1.4 to 2.0 | g/dL |
| Chitotriosidase | 30 Units/kg | Month 3 | -32.4 | Standard Deviation 23.5 | Percent Change from Baseline |
| Chitotriosidase | 60 Units/kg | Month 3 | -32.7 | Standard Deviation 15.1 | Percent Change from Baseline |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | 30 Units/kg | 6 | |
| Overall Study | STARTED | 60 Units/kg | 5 | |
| Overall Study | COMPLETED | 30 Units/kg | 6 | |
| Overall Study | COMPLETED | 60 Units/kg | 5 | |
| Overall Study | NOT COMPLETED | 30 Units/kg | 0 | |
| Overall Study | NOT COMPLETED | 60 Units/kg | 0 |
Source ClinicalTrials.gov
Adverse events4
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 | VOMITING | Gastrointestinal disorders | 30 Units/kg | 2 | 6 | 2 |
| other | VOMITING | Gastrointestinal disorders | 60 Units/kg | 2 | 5 | 2 |
| serious | GASTROINTESTINAL INFLAMMATION | Gastrointestinal disorders | 30 Units/kg | 0 | 6 | 0 |
| serious | GASTROINTESTINAL INFLAMMATION | Gastrointestinal disorders | 60 Units/kg | 1 | 5 | 1 |
Source ClinicalTrials.gov
Adverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | 30 Units/kg | ||
| Total, all-cause mortality | 60 Units/kg | ||
| Total, other adverse events | 30 Units/kg | 2 | 6 |
| Total, other adverse events | 60 Units/kg | 2 | 5 |
| Total, serious adverse events | 30 Units/kg | 0 | 6 |
| Total, serious adverse events | 60 Units/kg | 1 | 5 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 25453586 | DERIVED |
Source 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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| 30 Units/kg vs 60 Units/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/NCT01132690
GET https://conductscience.org/api/trials/v1/NCT01132690/results?page=2