Trial · NCT03485677
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 11 of 11
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Assessment of pharmacokinetic (PK) parameter of eliglustat: Cmax | Weeks 2, 13, 26 and 52 | Maximum concentration (Cmax) of eliglustat in plasma |
| Primary | Assessment of PK parameter of eliglustat: AUC | Weeks 2 and 52 | Area under the plasma eliglustat concentration-time curve (AUC) |
| Primary | Adverse Events | Up to Week 364 | Number of adverse events in pediatric patients |
| Secondary | Change in hemoglobin level | Baseline and Week 52 | Absolute change from baseline for hemoglobin (g/dL) (Cohort 1 patients) |
| Secondary | Change in platelet count | Baseline and Week 52 | Percent change from baseline for platelet count (Cohort 1 patients) |
| Secondary | Change in liver volume | Baseline and Week 52 | Percent change from baseline for liver volume (Cohort 1 patients) |
| Secondary | Change in spleen volume | Baseline and Week 52 | Percent change from baseline for spleen volume (Cohort 1 patients) |
| Secondary | Pulmonary disease improvement | Baseline and Week 52 | Proportion of patients with improvement in pulmonary disease (Cohort 2 patients) |
| Secondary | Bone disease improvement | Baseline and Week 52 | Proportion of patients with improvement in bone disease (Cohort 2 patients) |
| Secondary | Thrombocytopenia | Baseline and Week 52 | Proportion of patients with improvement in thrombocytopenia (Cohort 2 patients) |
| Secondary | Quality of Life | Baseline and Week 52 | Health-related quality of life will be measured by the Pediatric Quality of Life Inventory™ (PedsQL™) questionnaires |
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.
Twenty rows a page, as JSON. No key.
GET https://conductscience.org/api/trials/v1/NCT03485677/registered