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Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

NCT04860960 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 94

Last updated 2024-06-04

No results posted yet for this 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

  • Niemann-Pick Disease, Type C1

Interventions

DRUG

Hydroxypropyl-beta-cyclodextrin

Dose is 2000 mg/kg body weight provided every 2 weeks intravenously

DRUG

Placebo

0.5N saline provided every 2 weeks intravenously

Sponsors & Collaborators

  • Cyclo Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Karen Mullen, MD · Cyclo Therapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-07-20
Primary Completion
2025-06-30
Completion
2026-06-30
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • Brazil
  • Germany
  • Israel
  • Italy
  • Poland
  • Saudi Arabia
  • Spain
  • Taiwan
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04860960 on ClinicalTrials.gov