Trial · NCT02843035
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 18 of 18
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of participants with Treatment Emergent Adverse Events (TEAEs) | From screening up to end of study, up to approximately 10 years | |
| Primary | Assessment of pharmacodynamic (PD) parameter: Lyso-glucosylceramide (lyso-GL1) and glucosylceramide (GL-1) in cerebrospinal fluid (CSF) | From screening through Week 52 | |
| Secondary | Assessment of pharmacodynamic (PD) parameter: Lyso-glucosylceramide (lyso-GL1) and glucosylceramide (GL-1) in plasma | From screening up to end of study, up to approximately 10 years | Change from baseline in plasma lyso-GL1 and GL1 |
| Secondary | Assessment of plasma pharmacokinetic parameter: Cmax (Part 2) | Day 1 | Plasma maximum concentration (Cmax) |
| Secondary | Assessment of plasma pharmacokinetic parameter: Tmax (Part 2) | Day 1 | Plasma time at Cmax (Tmax) |
| Secondary | Assessment of plasma pharmacokinetic parameter: AUC0-24h (Part 2) | Day 1 | Plasma area under the curve (AUC0-24h) |
| Secondary | Assessment of plasma pharmacokinetic parameter: Ctrough | Weeks 12 and 39 (Part 2), and on Weeks 78, 104, and 156 (for Part 3) | Plasma trough concentration (Ctrough) |
| Secondary | Assessment of CSF pharmacokinetic parameter: Cmax | Week 4, Week 26, and Week 52 | CSF maximum concentration (Cmax) |
| Secondary | Assessment of spleen volume | From screening up to end of study, up to approximately 10 years | Percent change from baseline in spleen volume assessed by Magnetic Resonance Imaging (MRI) |
| Secondary | Assessment of spleen volume (Part 4) | From Week 260 up to end of study, up to approximately 10 years | Percent change from Part 4 baseline in spleen volume assessed by MRI |
| Secondary | Assessment of liver volume | From screening up to end of study, up to approximately 10 years | Percent change from baseline in liver volume assessed by MRI |
| Secondary | Assessment of liver volume (Part 4) | From Week 260 up to end of study, up to approximately 10 years | Percent change from Part 4 baseline in liver volume assessed by MRI |
| Secondary | Assessment of hemoglobin level | From screening up to end of study, up to approximately 10 years | Change from baseline in hemoglobin level |
| Secondary | Assessment of hemoglobin level (Part 4) | From Week 260 up to end of study, up to approximately 10 years | Change from Part 4 baseline in hemoglobin level |
| Secondary | Assessment of platelet level | From screening up to end of study, up to approximately 10 years | Percent change from baseline in platelet count |
| Secondary | Assessment of platelet level (Part 4) | From Week 260 up to end of study, up to approximately 10 years | Percent change from Part 4 baseline in platelet count |
| Secondary | Assessment of Ataxia | From screening up to end of study, up to approximately 10 years | Ataxia is assessed by using the Scale for the Assessment and Rating of Ataxia (SARA), which consists of 8 items that are related to gait, stance, sitting, speech disturbance, finger-chase test, nose-finger test, fast alternating hand movements and heel-shin slide test. A modified scoring algorithm is used to determine the SARA modified total score from 0 (no ataxia) to 32 (very severe ataxia). Change from baseline in SARA modified total score. |
| Secondary | Assessment of Ataxia (Part 4) | From Week 260 up to end of study, up to approximately 10 years | Ataxia is assessed by using the Scale for the Assessment and Rating of Ataxia (SARA), which consists of 8 items that are related to gait, stance, sitting, speech disturbance, finger-chase test, nose-finger test, fast alternating hand movements and heel-shin slide test. A modified scoring algorithm is used to determine the SARA modified total score from 0 (no ataxia) to 32 (very severe ataxia). Change from Part 4 baseline in SARA modified total score. |
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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GET https://conductscience.org/api/trials/v1/NCT02843035/registered