Trial · NCT00553631
Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
NCT00553631, Phase 3, Completed, Shire; results first posted 2011-01-04
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease. Trials, record NCT00553631. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00553631
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
- NCT number
- NCT00553631
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 34 participants, actual
- Lead sponsor
- Shire
- Conditions
- Gaucher Disease, Type 1
- Interventions
- velaglucerase alfa, imiglucerase
- First posted
- 2007-11-05
- Last updated
- 2021-06-08
- Other registries' IDs
- eudract:2007-002840-21
- Results first posted
- 2011-01-04
- Limitations and caveats
- Participants aged 2-4 years: 4 participants (23.5%) in imiglucerase group and 0 participants in GA-GCB group.
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group. | Baseline to Month 9 | |
| Secondary | Change From Baseline to Month 9 in Platelet Counts for Each Treatment Group. | Baseline to Month 9 | Values shown are observed change from Baseline to Month 9. |
| Secondary | Change From Baseline to Month 9 in Normalized Liver Volume (Percent (%) Body Weight) for Each Treatment Group. | Baseline to Month 9 | Values shown are observed change from Baseline to Month 9. Measured by Magnetic resonance imaging (MRI). Liver volume has been normalized for percent (%) body weight for each treatment arm. Liver size relative to body weight = (Liver volume \[cubic centimeter (cc)\]/Body weight \[kg\]\*1000. |
| Secondary | Change From Baseline to Month 9 in Normalized Spleen Volume (Percent (%) Body Weight) for Each Treatment Group. | Baseline to Month 9 | Values shown are observed change from Baseline to month 9. Measured by Magnetic resonance imaging (MRI). Spleen volume was normalized for percent (%) of body weight for each treatment arm. Spleen size relative to body weight=(Spleen volume \[cc\]/Body weight \[kg\])\*100. |
| Secondary | Change From Baseline to Month 9 in Plasma Chitotriosidase for Each Treatment Group. | Baseline to Month 9. | Values shown are observed change from Baseline to Month 9. Units of measure is defined as nanomole per milliliter per hour. |
| Secondary | Change From Baseline to Month 9 in Plasma Chemokine (C-C Motif) Ligand 18 (CCL18) for Each Treatment Group. | Baseline to Month 9 | Values shown are observed change from Baseline to Month 9. |
| Secondary | Number of Participants Who Developed Antibody for Each Treatment Group. | Baseline to Month 9 | Measure type is actual number of participants who developed antibodies to treatment; GA-GCB or imiglucerase. Antibody detection was based upon serum samples collected at various time points throughout the study. Serum samples were screened using an enzyme-linked immunosorbent assay (ELISA) and positive antibody confirmation was determined using a radioimmunoprecipitation assay (RIP); positive samples were also tested for enzyme neutralizing activity. Participant samples were compared to internal assay controls (positive/negative), positive samples were determined based upon individual assay criteria. |
| Secondary | Time to Response- Comparison of GA-GCB and Imiglucerase on the Earliest Time to Respond as Assessed Via Hemoglobin Concentration | Response rate at Month 9 compared to Baseline | Time to response was defined as a ≥ 1 g/dL improvement in hemoglobin levels relative to Baseline. Units (%) correlates to the percentage of participants who had a change of ≥ 1 g/dL improvement in hemoglobin levels relative to Baseline during their participation in the study. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group. | Baseline to Month 9 | Intent-to-treat (ITT) population comprised of all randomized participants who received at least 1 full or partial dose of study drug. | gram per deciliter (g/dl) | MEAN |
| SECONDARY | Change From Baseline to Month 9 in Platelet Counts for Each Treatment Group. | Baseline to Month 9 | ITT population. | 10^9 per liter (10^9/L) | MEAN |
| SECONDARY | Change From Baseline to Month 9 in Normalized Liver Volume (Percent (%) Body Weight) for Each Treatment Group. | Baseline to Month 9 | ITT population. | cubic centimeter (cm^3) | MEAN |
| SECONDARY | Change From Baseline to Month 9 in Normalized Spleen Volume (Percent (%) Body Weight) for Each Treatment Group. | Baseline to Month 9 | ITT population. Number of participants analyzed signifies participants evaluable for this outcome. Ten participants in each treatment group underwent splenectomy, and therefore, were excluded from the analysis. | cm^3 | MEAN |
| SECONDARY | Change From Baseline to Month 9 in Plasma Chitotriosidase for Each Treatment Group. | Baseline to Month 9. | ITT population. Number of participants analyzed signifies participants evaluable for this outcome. Chitotriosidase levels were measured in 10 participants in the velaglucerase alfa group and 11 participants in the imiglucerase group. | nanomole/milliliter/hour (nmol/mL/h) | MEAN |
| SECONDARY | Change From Baseline to Month 9 in Plasma Chemokine (C-C Motif) Ligand 18 (CCL18) for Each Treatment Group. | Baseline to Month 9 | ITT population. | nanogram per milliliter (ng/mL) | MEAN |
| SECONDARY | Number of Participants Who Developed Antibody for Each Treatment Group. | Baseline to Month 9 | Safety population comprised of all randomized participants who received at least 1 full or partial dose of study drug. | participants | NUMBER |
| SECONDARY | Time to Response- Comparison of GA-GCB and Imiglucerase on the Earliest Time to Respond as Assessed Via Hemoglobin Concentration | Response rate at Month 9 compared to Baseline | ITT population | Percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group. | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 1.624 | Standard Error 0.223 | gram per deciliter (g/dl) | |
| Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group. | Imiglucerase | 1.488 | Standard Error 0.281 | gram per deciliter (g/dl) | |
| Change From Baseline to Month 9 in Platelet Counts for Each Treatment Group. | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 110.41 | Standard Error 17.159 | 10^9 per liter (10^9/L) | |
| Change From Baseline to Month 9 in Platelet Counts for Each Treatment Group. | Imiglucerase | 144.38 | Standard Error 22.76 | 10^9 per liter (10^9/L) | |
| Change From Baseline to Month 9 in Normalized Liver Volume (Percent (%) Body Weight) for Each Treatment Group. | Gene-Activated Human Glucocerebrosidase (GA-GCB) | -1.31 | Standard Error 0.347 | cubic centimeter (cm^3) | |
| Change From Baseline to Month 9 in Normalized Liver Volume (Percent (%) Body Weight) for Each Treatment Group. | Imiglucerase | -1.10 | Standard Error 0.182 | cubic centimeter (cm^3) | |
| Change From Baseline to Month 9 in Normalized Spleen Volume (Percent (%) Body Weight) for Each Treatment Group. | Gene-Activated Human Glucocerebrosidase (GA-GCB) | -1.34 | Standard Error 0.424 | cm^3 | |
| Change From Baseline to Month 9 in Normalized Spleen Volume (Percent (%) Body Weight) for Each Treatment Group. | Imiglucerase | -2.46 | Standard Error 0.966 | cm^3 | |
| Change From Baseline to Month 9 in Plasma Chitotriosidase for Each Treatment Group. | Gene-Activated Human Glucocerebrosidase (GA-GCB) | -34711.9 | Standard Error 6887.77 | nanomole/milliliter/hour (nmol/mL/h) | |
| Change From Baseline to Month 9 in Plasma Chitotriosidase for Each Treatment Group. | Imiglucerase | -35109.5 | Standard Error 7310.22 | nanomole/milliliter/hour (nmol/mL/h) |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 17 | |
| Overall Study | STARTED | Imiglucerase | 17 | |
| Overall Study | COMPLETED | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 16 | |
| Overall Study | COMPLETED | Imiglucerase | 16 | |
| Overall Study | NOT COMPLETED | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 1 | |
| Overall Study | NOT COMPLETED | Imiglucerase | 1 |
Source ClinicalTrials.gov
Adverse events234
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 | Thrombocythaemia | Blood and lymphatic system disorders | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 1 | 17 | 1 |
| other | Thrombocythaemia | Blood and lymphatic system disorders | Imiglucerase | 1 | 17 | 1 |
| other | Hypersensitivity | Immune system disorders | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 2 | 17 | 2 |
| other | Hypersensitivity | Immune system disorders | Imiglucerase | 0 | 17 | 0 |
| other | Headache | Nervous system disorders | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 3 | 17 | 7 |
| other | Headache | Nervous system disorders | Imiglucerase | 3 | 17 | 6 |
| other | Paraesthesia | Nervous system disorders | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 2 | 17 | 2 |
| other | Paraesthesia | Nervous system disorders | Imiglucerase | 1 | 17 | 1 |
| other | Dry eye | Eye disorders | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 1 | 17 | 1 |
| other | Dry eye | Eye disorders | Imiglucerase | 0 | 17 | 0 |
1 to 10 of 234Source ClinicalTrials.gov
See all 234 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Gene-Activated Human Glucocerebrosidase (GA-GCB) | ||
| Total, all-cause mortality | Imiglucerase | ||
| Total, other adverse events | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 16 | 17 |
| Total, other adverse events | Imiglucerase | 16 | 17 |
| Total, serious adverse events | Gene-Activated Human Glucocerebrosidase (GA-GCB) | 3 | 17 |
| Total, serious adverse events | Imiglucerase | 0 | 17 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 27839979 | 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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Gene-Activated Human Glucocerebrosidase (GA-GCB) vs Imiglucerase: Number of Participants Who Developed Antibody for Each Treatment Group. | Trial comparisons |
| Gene-Activated Human Glucocerebrosidase (GA-GCB) vs Imiglucerase: serious adverse events | 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/NCT00553631
GET https://conductscience.org/api/trials/v1/NCT00553631/events?page=2