Trial · NCT00418847
Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
NCT00418847, Phase 2, Completed, The Hospital for Sick Children
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis. Trials, record NCT00418847. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00418847
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
- NCT number
- NCT00418847
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 5 participants, actual
- Lead sponsor
- The Hospital for Sick Children
- Conditions
- Gangliosidoses GM2
- Interventions
- miglustat
- First posted
- 2007-01-05
- Last updated
- 2016-05-19
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Concentration of miglustat in plasma | Periodic intervals up to 24 hours | |
| Secondary | Changes in volume loss and signal intensity from baseline MRI | 12 months | |
| Secondary | Change in single-voxel N acetylaspartate (NAA) from baseline MRS | 1 month, 3 months, 6 months, 9 months, and 12 months | |
| Secondary | Change in neuropsychological testing from baseline | 6 months and 12 months | |
| Secondary | Change in nerve conduction | 6 months and 12 months | |
| Secondary | Change in neurological examination from baseline | 1 month, 3 months, 6 months, 9 months, and 12 months |
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.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 19595619 | RESULT |
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/NCT00418847
GET https://conductscience.org/api/trials/v1/NCT00418847/registered