Trial · NCT02534844
Outcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
1 to 14 of 14
Download this page as JSON| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Parts A/B: Change From Baseline to Week 52 in 4-Item Composite Score of Niemann Pick Type C Severity Scale (NPC-SS) Score | Baseline, Week 52 | Modified intent-to-treat (mITT) population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, number analyzed = participants evaluable at specified time-point. | score on a scale | MEAN |
| PRIMARY | Parts A/B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) at Week 52 | Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Parts A/B: Change From Baseline to Week 52 in NPC-SS Total Score (Excluding Hearing and Auditory Brainstem Response [ABR]) | Baseline, Week 52 | mITT population included all randomized participants who received at least one treatment. As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, overall number of participants analyzed = participants evaluable for this outcome measure. Number analyzed = participants evaluable at specified time-point. | score on a scale | MEAN |
| SECONDARY | Parts A/ B: Number of Participants Classified as CGIC Responders at Week 52 | Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Parts A/B: Number of Participants Classified as Responders on NPC-SS Total Score (Excluding Hearing and ABR) at Week 52 | Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Parts A/B: EQ-5D-3L Questionnaire Visual Analog Scale (VAS) Score (for Health Status) at Baseline and at Week 52 | Baseline, Week 52 | mITT population included all randomized participants who received at least one treatment. As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, overall number of participants analyzed = participants evaluable for this outcome measure. Number analyzed = participants evaluable at specified time-point. | score on a scale | MEAN |
| SECONDARY | Parts A/B: Number of Participants Treated for at Least 6 Months Who Qualified for the Rescue Option | Baseline up to Week 26 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Parts A/B: Change From Baseline to Week 52 in Each of the 9 Clinical Domains of the NPC-SS | Baseline, Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, Number analyzed = participants evaluable at specified time-point. | score on a scale | MEAN |
| SECONDARY | Parts A/B: Change From Baseline to Week 52 in the Total NPC-SS (With Hearing Domain and ABR Modifier Included) | Baseline, Week 52 | mITT population included all randomized participants who received at least one treatment). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, overall number of participants analyzed = participants evaluable for this outcome measure, and number analyzed = participants evaluable at specified time-point. | score on a scale | MEAN |
| SECONDARY | Parts A/B: Time to One Point Increase (Worsening) in NPC-SS Composite Score | Baseline up to Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. | Days | MEDIAN |
| SECONDARY | Parts A/B: Change From Baseline in the Timed Up and Go (TUG) Test at Week 52 | Baseline, Week 52 | mITT population included all randomized participants who received at least one treatment. As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, overall number of participants analyzed = participants evaluable for this outcome measure, and number analyzed = participants evaluable at specified time-point. | seconds | MEAN |
| SECONDARY | Parts A/B: Change From Baseline in the 9-Hole Peg Test at Week 52 | Baseline, Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | seconds | MEAN |
| SECONDARY | Parts A/B: Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Baseline up to Week 52 | Safety population was defined as all randomized participants who received at least 1 procedure (adrabetadex infusion via LP or sham). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Parts A/B: Change From Baseline to Week 52 in Mean Annualized Rate of Change (Slope) of NPC-SS Composite Score | Baseline, Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | score on NPC-SS scale/year | MEAN |
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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