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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 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. The trial reports 20 study locations across 9 states.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04860960
The ClinicalTrials.gov registry entry for NCT04860960 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 94 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Cyclo Therapeutics, which has 2 total studies on file at ClinicalTrials.gov.
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 reports 20 study locations spanning 9 distinct geographic areas - top geographies include 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.
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