Trial · NCT01842841
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 16 of 16
Download this page as JSON| 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. |
| Secondary | Change From Baseline in Bone Marrow Burden (BMB) Score at Week 103 | Baseline, Week 103 | BMB Score was measured using MRI, range from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0-16 points. A higher BMB score signified more severe bone marrow involvement. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. |
| Secondary | Change From Baseline in Growth Velocity at Week 101 : Height Z-Score | Baseline, Week 101 | The Z-score, or Standard Deviation Score, is a measure of number of SDs above or below the average BMD of a healthy participant of the same age and gender. World Health Organization 2007 growth reference data were used for Z-score calculation. 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. |
| Secondary | Change From Baseline in Skeletal Age at Week 103: Z-Score | Baseline, Week 103 | Skeletal age was measured via radiography (X-ray) of the left hand and wrist by the method of Greulich and Pyle. The Z-score, or Standard Deviation Score, is a measure of number of SDs above or below the average BMD of a healthy participant of the same age and gender. 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. |
| Secondary | Change From Baseline in Plasma Chitotriosidase Levels at Week 101 | Baseline, Week 101 | Plasma chitotriosidase activity levels were measured using an enzymatic assay with 4-methylumbelliferyl-deoxychitobiose as a substrate. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. |
| Secondary | Number of Participants With Change From Baseline in Neurological Status at Week 103 | Baseline, Week 103 | Neurological status was considered normal or abnormal based on investigator's discretion. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. |
| Secondary | Change From Baseline in Chemokine [C-C Motif] Ligand 18 (CCL18) Levels at Week 101 | Baseline, Week 101 | Plasma CCL18 concentrations were measured using a time-resolved fluorescence assay. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
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
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