Trial · NCT07399704
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 13 of 13
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change from baseline in treatment-emergent adverse events (TEAEs) | Through study completion, an average of 4 years | Incidence and severity of all Adverse Events related to study drug treatment, study discontinuation or death |
| Primary | Change from baseline in electrocardiogram (ECG) | Through study completion, an average of 4 years | ECG read out Normal, Abnormal, Not Clinically Significant, Abnormal, Clinically Significant and Not Done. |
| Primary | Change from baseline in seizures | Through study completion, an average of 4 years | Seizure duration (minutes) as per the seizure diary. |
| Primary | Change from baseline in seizures | Through study completion, an average of 4 years | Seizure frequency (number) as per seizure diary. |
| Primary | Maximum observed plasma concentration (Cmax) | Baseline , Month 1 (Cohort 2 only) and Month 6 | |
| Primary | Time to Cmax (Tmax) | Baseline, Month 1 (Cohort 2 only) and Month 6 | |
| Primary | Concentration at trough (Ctrough) | Baseline, Month 1 (Cohort 2 only) and Month 6 | |
| Primary | Area under the plasma concentration-time curve from the time of dosing (zero) to 24 hours post-dose | Baseline, Month 1 (Cohort 2 only) and Month 6 | |
| Secondary | Change from Baseline in the concentrations of Glucosylceramide (GlcCer) C16:0; C18:0 | Baseline, Month 1 (Cohort 2 only) and Month 6 | |
| Secondary | Change from Baseline in the concentrations of Neurofilament light chain (NfL) | Through study completion, an average of 4 years | |
| Secondary | For GM2 gangliosidosis patients: Change from Baseline in the concentrations of Monosialoganglioside GM2 (GM2) | Through study completion, an average of 4 years | |
| Secondary | For GM2 gangliosidosis patients: Change from Baseline in the concentrations of Lyso-monosialoganglioside GM2 | Through study completion, an average of 4 years | |
| Secondary | For NPC disease patients: Change from Baseline in the concentrations of N-palmitoyl-O-phosphocholine-serine (PPCS) | Through study completion, an average of 4 years |
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/NCT07399704/registered