Trial · NCT06523517
Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3
NCT06523517, Phase 2, Unknown, Peking Union Medical College Hospital
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3. Trials, record NCT06523517. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT06523517
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3
- NCT number
- NCT06523517
- Status
- Unknown
- Phases
- Phase 2
- Enrollment
- 5 participants, estimated
- Lead sponsor
- Peking Union Medical College Hospital
- Conditions
- Gaucher Disease
- Interventions
- Eliglustat Tartrate Capsules
- First posted
- 2024-07-26
- Last updated
- 2024-07-26
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Changes in hemoglobin level | Baseline, Weeks 13, 26, 39 and 52 | Absolute change from baseline for hemoglobin (g/dL) |
| Primary | Changes in platelet count | Baseline, Weeks 13, 26, 39 and 52 | Percent change from baseline for platelet count |
| Primary | Changes in spleen volume | Baseline, Weeks 26 and 52 | Percent change from baseline for spleen volume |
| Primary | Changes in liver volume | Baseline, Weeks 26 and 52 | Percent change from baseline for liver volume |
| Primary | Changes in Lyso-GL1 level | Baseline, Weeks 13, 26, 39 and 52 | Percent change from baseline for Lyso-GL1 level |
| Primary | Skeletal improvement | Baseline, Weeks 26 and 52 | Proportion of patients with improvement in skeletal disease |
| Primary | Assessment of pharmacokinetic (PK) parameter of eliglustat: Cmax | Baseline, Weeks 2, 13, 26 and 52 | Peak concentration (Cmax) of eliglustat in plasma (ng/mL) |
| Primary | Assessment of pharmacokinetic (PK) parameter of eliglustat: Ctrough | Baseline, Weeks 2, 13, 26 and 52 | Trough concentration (Ctrough) of eliglustat in plasma (ng/mL) |
| Primary | Adverse events | Up to Week 52 | Number of adverse events in pediatric patients |
| Secondary | Changes in 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.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT06523517
GET https://conductscience.org/api/trials/v1/NCT06523517/registered