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NCT04860960 · ClinicalTrials.gov registry record · Phase 3
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
A Phase 3 study of Niemann-Pick Disease, Type C1, sponsored by Cyclo Therapeutics.
- Active
- Registry status
- Phase 3
- Development phase
- 94
- Enrollment target
- 20
- Study locations
NCT04860960: Active Phase 3 study of Niemann-Pick Disease, Type C1, sponsored by Cyclo Therapeutics.
NCT04860960 is a Phase 3 study of Niemann-Pick Disease, Type C1 that is active but no longer recruiting, run by Cyclo Therapeutics. The registered enrollment target is 94 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (88% lower). The trial reports 20 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04860960, a Phase 3 study of Niemann-Pick Disease, Type C1, is active but no longer recruiting, sponsored by Cyclo Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 94 participants
- Enrollment target
- 20
- Study locations
Study Summary
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1). The objective of this study is to evaluate the safety, tolerability and efficacy of 2000 mg/kg dose of Trappsol Cyclo (hydroxypropyl betacyclodextrin) administered intravenously compared to standard of care. An open-label sub-study in countries following European Medicines Agency (EMA) guidance will enroll asymptomatic or symptomatic patients from infancy up to age 3 to evaluate safety in that population.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Hydroxypropyl-beta-cyclodextrin
Study Locations (20)
Other
- Hospital de Alta Complejidad en Red "El Cruce" - Buenos Aires
- Hospital de Niños de la Santísima Trinidad - Córdoba
- Metabolic Clinical Trials Unit - Adelaide
- Hospital de Clínicas de Porto Alegre - Porto Alegre
- Universidade de São Paulo - São Paulo
- University of Campinas - São Paulo
- SphinCS GmbH - Höchheim
- University Munster - Münster
- Emek Medical Center-Department of Pediatrics - Afula
- Soroka Medical Center - Beersheba
- University of Catania - Catania
Victoria
- Melbourne Children's Trials Centre Murdoch Children's Research Institute - Parkville
- Royal Melbourne Hospital - Parkville
California
- UCSF Benioff Children's Hospital Oakland - Oakland
Florida
- University of Florida - Jacksonville
Georgia
- Emory - Atlanta
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Pennsylvania
- UPMC Children's Hospital - Pittsburgh
Utah
- University Utah - Salt Lake City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 94 participants |
| Start Date | 2021-07-20 |
| Est. Completion | 2026-06 |
| Phase | Phase 3 |
What the registry record for NCT04860960 still lists
NCT04860960 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 94 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (88% lower).
The record links to 1 condition, with Niemann-Pick Disease, Type C1 appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT04860960 names 20 study sites across 9 states, led by Other, Victoria, California.
Frequently Asked Questions
What is clinical trial NCT04860960 about?
NCT04860960 is a clinical study titled "Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1". A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1). The objective of this study is to evaluate the safety, tolerability and efficacy of 2000 mg/kg dose of Trappsol Cy...
What is the current status of trial NCT04860960?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 94 participants. The study started on 2021-07-20. Estimated completion is 2026-06.
What conditions does trial NCT04860960 study?
This clinical trial studies the following conditions: Niemann-Pick Disease, Type C1.
What interventions are being tested in trial NCT04860960?
The interventions under investigation include: Placebo (DRUG), Hydroxypropyl-beta-cyclodextrin (DRUG).
Who is sponsoring clinical trial NCT04860960?
This trial is sponsored by Cyclo Therapeutics, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04860960 being conducted?
This trial has 20 study locations across California, Florida, Georgia, Ohio, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Niemann-Pick Disease, Type C1
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04860960's enrollment target sits among peer trials
94 1720th of 2000 higher than 279 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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