Trial · NCT04860960
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 16 of 16
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change from Baseline in 4-Domain NPC Severity Score (US only) | Interim Analysis at Week 48 | Ambulation, Fine Motor, Speech, Swallow |
| Primary | Change from Baseline in 4-Domain NPC Severity Score (US only) | End of Study at Week 96 | Ambulation, Fine Motor, Speech, Swallow |
| Primary | Change from Baseline in 5-Domain NPC Severity Score (ex-US) | Interim Analysis at Week 48 | Ambulation, Fine Motor, Speech, Swallow, Cognition |
| Primary | Change from Baseline in 5-Domain NPC Severity Score (ex-US) | End of Study at Week 96 | Ambulation, Fine Motor, Speech, Swallow, Cognition |
| Secondary | Change in ataxia as measured by Spinocerebellar ataxia functional index | Change from Baseline as measured every 12 weeks through week 96 and end of OLE week 192 | SCAFI |
| Secondary | Change in adaptive behavior as measured by Vineland Adaptive Behavior Scale II | Change from Baseline as measured every 12 weeks through week 96 and end of OLE week 192 | Vineland Adaptive Behavior Scale II |
| Secondary | Change in Swallow function evaluated by videofluoroscopy or fiberoptic endoscopy and measured by Penetration Aspiration Scale | Change from Baseline measured at Interim Analysis Week 48 | PAS |
| Secondary | Change in Swallow function evaluated by videofluoroscopy or fiberoptic endoscopy and measured by Penetration Aspiration Scale | Change from Baseline measured at End of Study Week 96 | PAS |
| Other | Change in speaking ability compared to Baseline as measured by voice recordings collected in SpeechVitals mobile device application | Baseline and every two weeks through week 192 | Measurement of speech features including articulatory precision, speaking and pause rates |
| Other | Change in speaking ability compared to Pre-Infusion as measured by voice recordings collected in SpeechVitals mobile device application | Every two weeks through week 192 | Measurement of speech features including articulatory precision, speaking and pause rates within 24 hours post-infusion |
| Other | Change in Scores of Clinical Global Impression of Severity and of Change compared to Baseline | Baseline, weeks 2, 4, 12, 24, 36, 48, 60, 72, 84, 96, 100, 120, 144, 168, 192 | Clinical Global Impression of Severity and of Change |
| Other | Change in Scores of Caregiver Global Impression of Severity and of Change scales | Baseline, weeks 2, 4, 12, 24, 36, 48, 60, 72, 84, 96, 100, 120, 144, 168, 192 | Caregiver Global Impression of Severity and of Change |
| Other | Caregiver Global Impression of Change at 24 hours post infusion | Baseline and every 2 weeks through week 192 | Caregiver Global Impression of Change at 24 hours post infusion |
| Other | Change from Baseline in Respiratory function measured by Forced Expiratory Volume in 1 second | Baseline, weeks 2, 4, 12, 24, 36, 48, 60, 72, 84, 96, 100, 192 | FEV1 |
| Other | Change from Baseline in Liver function as measured by liver enzyme assessments | Baseline, weeks 2, 4, 12, 24, 36, 48, 60, 72, 84, 96, 100, 120, 144, 168, 192 | Liver function measured by liver enzyme assessments including alanine and aspartate aminotransferases |
| Other | Safety assessments to include incidence of Adverse Events and Serious Adverse Events | Regular assessments per protocol through week 192 | Incidence of AEs, SAEs, incidence of abnormal laboratory test results, abnormal ECGs, abnormal physical exams, abnormal vital signs and abnormal hearing assessments assessments |
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
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GET https://conductscience.org/api/trials/v1/NCT04860960/registered