Trial · NCT01842841
Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease
NCT01842841, Phase 3, Completed, Shire; results first posted 2015-12-14
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease. Trials, record NCT01842841. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01842841
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease
- NCT number
- NCT01842841
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 5 participants, actual
- Lead sponsor
- Shire
- Conditions
- Gaucher Disease
- Interventions
- velaglucerase alfa
- First posted
- 2013-04-30
- Last updated
- 2021-06-14
- Results first posted
- 2015-12-14
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) | From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) | An AE was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered related to investigational product. A SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. An infusion-related AE was defined as an AE that started either during or within 12 hours after the start of the infusion and that was judged as possibly or probably related to investigational product. |
| Primary | Number of Participants Using Concomitant Medication | From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) | |
| Primary | Number of Participants With Abnormal and Clinically Significant Laboratory Test Results | From Week 65 until the end of study (Week 155) | Laboratory test results were considered abnormal and clinically significant at the discretion of the investigator. |
| Primary | Number of Participants With Positive Anti-Velaglucerase Alfa Antibodies | From Week 65 until the end of study (Week 155) | Serum samples were collected for all participants for determination of anti-velaglucerase alfa antibodies every 12 weeks. |
| Secondary | Change From Baseline in Hemoglobin Concentration at Week 101 | Baseline, Week 101 | Baseline was the modified baseline hemoglobin concentration, the average of the values from screening, baseline, and Week 1 Day 1 from Study HGT-GCB-087 (NCT01614574). |
| Secondary | Change From Baseline in Platelet Count at Week 101 | Baseline, Week 101 | Baseline was the modified baseline platelet count, the average of the values from screening, baseline, and Week 1 Day 1 from Study HGT-GCB-087 (NCT01614574). |
| Secondary | Change From Baseline in Liver Volume Normalized to Body Weight at Week 103 | Baseline, Week 103 | Liver volume was measured using magnetic resonance imaging (MRI). Liver volume measurements were normalized to the percentage of body weight. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. |
| Secondary | Change From Baseline in Spleen Volume Normalized to Body Weight at Week 103 | Baseline, Week 103 | Spleen volume was measured using MRI. Spleen volume measurements were normalized to the percentage of body weight. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. |
| Secondary | Change From Baseline in Bone Mineral Density (BMD) at Week 103: Z Score | Baseline, Week 103 | BMD was measured by dual energy x-ray absorptiometry (DXA) for lumbar spine and femurs. To ensure standardization and allow for comparisons of BMD, results were converted to standardized Z-scores (matched for age and gender). Z-scores express the BMD as the number of standard deviations (SDs) above or below the average BMD of a healthy participant of the same age and gender. Statistical analysis plan only required summarization if greater than (\>) 50 percent (%) of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. |
| Secondary | Change From Baseline in Bone Mineral Density (BMD) at Week 103: T-Score | Baseline, Week 103 | BMD was measured by DXA for lumbar spine and femurs. To ensure standardization and allow for comparisons of BMD, results were converted to standardized T-scores; normal values were used from databases from Hologic based on standard criteria. T-scores are the number of SDs above or below the average for a young adult at peak BMD. Statistical analysis plan only required summarization if \>50% of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) | From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) | Safety population. | participants | NUMBER |
| PRIMARY | Number of Participants Using Concomitant Medication | From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) | Safety population. | participants | NUMBER |
| PRIMARY | Number of Participants With Abnormal and Clinically Significant Laboratory Test Results | From Week 65 until the end of study (Week 155) | Safety population. | participants | NUMBER |
| PRIMARY | Number of Participants With Positive Anti-Velaglucerase Alfa Antibodies | From Week 65 until the end of study (Week 155) | Safety population. | participants | NUMBER |
| SECONDARY | Change From Baseline in Hemoglobin Concentration at Week 101 | Baseline, Week 101 | Safety population | gram per deciliter (g/dL) | MEAN |
| SECONDARY | Change From Baseline in Platelet Count at Week 101 | Baseline, Week 101 | Safety population | *10^9 platelets per liter | MEAN |
| SECONDARY | Change From Baseline in Liver Volume Normalized to Body Weight at Week 103 | Baseline, Week 103 | Safety population | Percentage of body weight | MEAN |
| SECONDARY | Change From Baseline in Spleen Volume Normalized to Body Weight at Week 103 | Baseline, Week 103 | Safety population | Percentage of body weight | MEAN |
| SECONDARY | Change From Baseline in Bone Mineral Density (BMD) at Week 103: Z Score | Baseline, Week 103 | Data was not reported as there were lesser than (\<) 50% of participants with evaluable data. | ||
| SECONDARY | Change From Baseline in Bone Mineral Density (BMD) at Week 103: T-Score | Baseline, Week 103 | Data was not reported as there were \<50% of participants with evaluable data. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) | Velaglucerase Alfa (VPRIV®) | Study drug-related AEs | 2 | participants | |
| Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) | Velaglucerase Alfa (VPRIV®) | Infusion-related AEs | 0 | participants | |
| Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) | Velaglucerase Alfa (VPRIV®) | Serious AEs | 2 | participants | |
| Number of Participants Using Concomitant Medication | Velaglucerase Alfa (VPRIV®) | 5 | participants | ||
| Number of Participants With Abnormal and Clinically Significant Laboratory Test Results | Velaglucerase Alfa (VPRIV®) | 1 | participants | ||
| Number of Participants With Positive Anti-Velaglucerase Alfa Antibodies | Velaglucerase Alfa (VPRIV®) | 0 | participants | ||
| Change From Baseline in Hemoglobin Concentration at Week 101 | Velaglucerase Alfa (VPRIV®) | 0.22 | Standard Deviation 0.817 | gram per deciliter (g/dL) | |
| Change From Baseline in Platelet Count at Week 101 | Velaglucerase Alfa (VPRIV®) | 9.8 | Standard Deviation 13.14 | *10^9 platelets per liter | |
| Change From Baseline in Liver Volume Normalized to Body Weight at Week 103 | Velaglucerase Alfa (VPRIV®) | 0.01 | Standard Deviation 0.207 | Percentage of body weight | |
| Change From Baseline in Spleen Volume Normalized to Body Weight at Week 103 | Velaglucerase Alfa (VPRIV®) | 0.04 | Standard Deviation 0.089 | Percentage of body weight |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Velaglucerase Alfa (VPRIV®) | 5 | |
| Overall Study | COMPLETED | Velaglucerase Alfa (VPRIV®) | 5 | |
| Overall Study | NOT COMPLETED | Velaglucerase Alfa (VPRIV®) | 0 |
Source ClinicalTrials.gov
Adverse events40
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 | Hypoacusis | Ear and labyrinth disorders | Velaglucerase Alfa (VPRIV®) | 1 | 5 | 2 |
| other | Retinal detachment | Eye disorders | Velaglucerase Alfa (VPRIV®) | 1 | 5 | 2 |
| other | Vitreous opacities | Eye disorders | Velaglucerase Alfa (VPRIV®) | 1 | 5 | 2 |
| other | Abdominal pain | Gastrointestinal disorders | Velaglucerase Alfa (VPRIV®) | 1 | 5 | 1 |
| other | Abdominal pain upper | Gastrointestinal disorders | Velaglucerase Alfa (VPRIV®) | 2 | 5 | 2 |
| other | Constipation | Gastrointestinal disorders | Velaglucerase Alfa (VPRIV®) | 1 | 5 | 1 |
| other | Enteritis | Gastrointestinal disorders | Velaglucerase Alfa (VPRIV®) | 1 | 5 | 1 |
| other | Chest pain | General disorders | Velaglucerase Alfa (VPRIV®) | 1 | 5 | 1 |
| other | Adenoiditis | Infections and infestations | Velaglucerase Alfa (VPRIV®) | 1 | 5 | 1 |
| other | Gastroenteritis | Infections and infestations | Velaglucerase Alfa (VPRIV®) | 2 | 5 | 2 |
1 to 10 of 40Source ClinicalTrials.gov
See all 40 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Velaglucerase Alfa (VPRIV®) | ||
| Total, other adverse events | Velaglucerase Alfa (VPRIV®) | 5 | 5 |
| Total, serious adverse events | Velaglucerase Alfa (VPRIV®) | 2 | 5 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 27241455 | 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 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/NCT01842841
GET https://conductscience.org/api/trials/v1/NCT01842841/events?page=2