Trial · NCT04860960
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
NCT04860960, Phase 3, Unknown, Cyclo Therapeutics, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1. Trials, record NCT04860960. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04860960
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
- NCT number
- NCT04860960
- Status
- Unknown
- Phases
- Phase 3
- Enrollment
- 94 participants, actual
- Lead sponsor
- Cyclo Therapeutics, Inc.
- Conditions
- Niemann-Pick Disease, Type C1
- Interventions
- Hydroxypropyl-beta-cyclodextrin, Placebo
- First posted
- 2021-04-27
- Last updated
- 2024-06-04
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| 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 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 36279795 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
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 trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04860960
GET https://conductscience.org/api/trials/v1/NCT04860960/registered