Trial · NCT01074944
Registered outcomes36
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 36
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | PAP: Percentage of Participants Who Remained Stable for 52 Weeks During the PAP | PAP Baseline up to the end of PAP (Week 52) | Participants were considered as stable if they met all of the following criteria: 1) no more than 2 bone crisis during PAP (with no more than 1 bone crisis during either the first 6 months or the later 6 months of the period), and were free of other clinically symptomatic bone disease during the entire 52-week PAP; 2) hemoglobin level not decreased \>1.5 g/dL from Baseline for PAP; 3) platelet count not decreased \>25% from Baseline for PAP; 4) spleen volume (in multiples of normal \[MN\]) did not increase \>25% from Baseline for PAP; 5) liver volume (in MN) did not increase \>20% from Baseline for PAP. Baseline for PAP was defined as the last assessment prior to randomization. |
| Secondary | PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52 | Baseline, Week 26, Week 52 | |
| Secondary | PAP: Mean Platelet Count at Baseline, Weeks 26, 52 | Baseline, Week 26, Week 52 | |
| Secondary | PAP: Mean Spleen Volume at Baseline, Weeks 26, 52 | Baseline, Week 26, Week 52 | |
| Secondary | PAP: Mean Liver Volume at Baseline, Weeks 26, 52 | Baseline, Week 26 and Week 52 | |
| Secondary | PAP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26 and Week 52 | Baseline, Week 26, Week 52 | Chitotriosidase biomarker was assayed from plasma. |
| Secondary | PAP: Mean Biomarker (GL-1 on DBS) Value at Baseline, Week 26 and Week 52 | Baseline, Week 26 and week 52 | GL-1 on DBS biomarker was assayed from dried blood spot (DBS). |
| Secondary | PAP: Mean Biomarker Macrophage Inflammatory Protein-1 Beta (MIP1-beta) Value at Baseline, Weeks 26, 52 | Baseline, Week 26, Week 52 | MIP1-beta biomarker was assayed from plasma. |
| Secondary | PAP: Bone Mineral Density (BMD) at Baseline and Week 52 | Baseline, Week 52 | BMD measurements of the spine and bilateral femur were acquired by dual-energy x-ray absorptiometry (DXA) scan. |
| Secondary | PAP: Total T-Scores for BMD at Baseline and Week 52 | Baseline, Week 52 | Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). |
| Secondary | PAP: Total Z-scores for BMD at Baseline and Week 52 | Baseline, Week 52 | Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score \>-2) and below normal (score \<=-2). |
| Secondary | PAP: Number of Participants With Mobility Status Asessments (MS) at Baseline, Weeks 26, and 52. | Baseline, Week 26 and Week 52 | Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. |
| Secondary | PAP: Number of Participants With Bone Crises at Baseline, Weeks 26 and 52 | Baseline, Week 26, and Week 52 | Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, and 2= 2 bone crises during the assessment period. In this outcome, number of participants with different bone crises levels at specified time points were reported. |
| Secondary | PAP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Weeks 26 and 52 | Baseline, Week 26, and Week 52 | Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain during the past 4 weeks. In this outcome, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. |
| Secondary | PAP: Total Bone Marrow Burden Score (BMB) at Baseline and Week 52 | Baseline, Week 52 | BMB Score was measured using magnetic resonance imaging (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 (no abnormalities) -16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. |
| Secondary | LIP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26, 52 and 78 | Baseline, Week 26, Week, 52, and Week 78 | |
| Secondary | LIP: Mean Platelet Count at Baseline, Weeks 26, 52 and 78 | Baseline, Week 26, Week 52, Week 78 | |
| Secondary | LIP: Mean Liver Volume at Baseline, Weeks 26, 52 and 78 | Baseline, Week 26, Week 52, Week 78 | |
| Secondary | LIP: Mean Spleen Volume at Baseline, Weeks 26, 52 and 78 | Baseline, Week 26, Week 52, Week 78 | |
| Secondary | LIP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26, 52, and 78 | Baseline, Week 26, Week 52 and Week 78 | Chitotriosidase biomarker was assayed from plasma. |
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
Read this list from software.
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