Trial · NCT04221451
Outcomes19
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
1 to 19 of 19
Download this page as JSON| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | PAP: PP: Percent Change From Baseline in CSF GM2 Biomarker to Week 104 | Baseline (Day 1) and Week 104 | PAP: Primary PD population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those participants with data collected at specified timepoints are reported. | percent change | MEAN |
| PRIMARY | PAP: PP: Annualized Rate of Change From Baseline in the 9-HPT to Week 104 | Baseline (Day 1) and Week 104 | PAP: Primary efficacy population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years. Only those participants with data collected at specified timepoints are reported. | percent per year | MEAN |
| PRIMARY | PAP: SP: Percent Change From Baseline in Plasma and CSF Biomarkers (Glucosylceramide [GL-1] and GM2) to Week 104 in Juvenile/Adolescent Late-onset GM2 Gangliosidosis Participants | Baseline (Day 1) and Week 104 | PAP: Analysis was performed on juvenile/adolescent late-onset GM2 gangliosidosis participants from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. | percent change | MEAN |
| PRIMARY | PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1 and GM1 Biomarkers to Week 104 in GM1 Gangliosidosis Participants | Baseline (Day 1) and Week 104 | PAP: Analysis was performed on GM1 gangliosidosis participants from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those participants with data collected at specified timepoints are reported. | percent change | MEAN |
| PRIMARY | PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM2 and Monosialodihexosylganglioside (GM3) Biomarkers to Week 104 in Sialidosis Type 1 Participant | Baseline (Day 1) and Week 104 | Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study. | percent change | MEAN |
| PRIMARY | PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM1 and GM3 Biomarkers to Week 104 in Juvenile/Adult Galactosialidosis Participants | Baseline (Day 1) and Week 104 | Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study. | percent change | MEAN |
| PRIMARY | PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1 Biomarker to Week 104 in Saposin C Deficiency Participants | Baseline (Day 1) to Week 104 | PAP: Analysis was planned to be performed on saposin C deficiency participants from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. As presented in baseline characteristics, there was no participant enrolled with Saposin C deficiency. | ||
| SECONDARY | PAP: PP: Absolute Change From Baseline in CSF GM2 Biomarker to Week 104 | Baseline (Day 1) and Week 104 | PAP: Primary PD population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those participants with data collected at specified timepoints are reported. | ng/mL | MEAN |
| SECONDARY | PAP: PP and SP: Change From Baseline in 25-foot Walk Test (25FWT) to Week 104 | Baseline (Day 1) and Week 104 | PAP PP:primary efficacy population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years.PAP SP: secondary efficacy population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis.Only those participants with ability to walk at baseline and data collected at specified timepoints are reported. | seconds | MEAN |
| SECONDARY | PAP: PP and SP: Change From Baseline in the Neurological Examination of the Friedreich's Ataxia Rating Scale (FARS) (FARS-neuro) to Week 104 | Baseline (Day 1) and Week 104 | Only those participants with data collected at specified timepoints are reported. For sialidosis type 1: due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study. | score on a scale | MEAN |
| SECONDARY | PAP: PP and SP: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) | From first dose of study drug (Day 1) up to end of PAP, 104 weeks | PAP PP: primary safety population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years who received at least 1 dose of study drug.~PAP SP: secondary safety population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis, who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | PAP: PP: Plasma Venglustat Concentration | Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12 | PAP: Primary pharmacokinetic (PK) population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years who received at least 1 dose of study drug and had at least 1 PK assessment. Only those participants with data collected at specified timepoints are reported. | ng/mL | MEAN |
| SECONDARY | PAP: SP: Plasma Venglustat Concentration | Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12 | PAP: Secondary PK population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis, who received at least 1 dose of study drug and had at least 1 PK assessment. 'Overall Number of Participants Analyzed' indicates the total of maximum number of participants ('number analyzed') for each dose. | ng/mL | MEAN |
| SECONDARY | PAP: PP and SP: CSF Venglustat Concentration | Week 104 | PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug \& had at least 1 PK assessment. | ng/mL | MEAN |
| SECONDARY | PAP: PP and SP: Maximum Plasma Concentration Observed (Cmax) of Venglustat | Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12 | PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug \& had at least 1 PK assessment. | ng/mL | MEAN |
| SECONDARY | PAP: PP and SP: Time to Reach Maximum Plasma Concentration (Tmax) of Venglustat | Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12 | PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug \& had at least 1 PK assessment. | hour | MEDIAN |
| SECONDARY | PAP: PP and SP: Area Under the Plasma Concentration Versus Time Curve Calculated Over a Predefined Time Period 0 to 24 Hours (AUC0-24) of Venglustat | Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12 | PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug \& had at least 1 PK assessment. | hour*ng/mL | MEAN |
| SECONDARY | PAP: SP: Percent Change From Baseline in the 9-HPT to Week 104 | Baseline (Day 1) and Week 104 | Only those participants with data collected at specified timepoints are reported. For sialidosis type 1: due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study. | percent change | MEAN |
| SECONDARY | PAP: SP: Number of Participants With >=80% Compliance as Per Method of Intake | Baseline (Day 1) to Week 104 | Secondary safety population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis, who received at least 1 dose of study drug. Only pediatric participants were evaluated for this endpoint. | Participants | COUNT_OF_PARTICIPANTS |
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.
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