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

Status unconfirmed · Phase 3 · Has a placebo group

Conditions studied: Niemann-Pick Disease, Type C1

In brief

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.

Key facts

Study ID: NCT04860960
Run by: Cyclo Therapeutics, Inc.
People needed: 94
Starts: 2021-07-20
Expected to finish: 2026-06-01
Last updated by the study team: 2024-06-04

Who can join

Age: 3 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Confirmed diagnosis of NPC1
Annual Severity Increment Score between 0.5 and 2.0 using the 17-domain NPC Severity Scale
Treated or Not Treated with Miglustat (patients must be on a stable dose for at least 3 months prior to the Screening Visit, or have discontinued Miglustat for at least 3 months prior to Screening Visit).
Body weight greater than 4.5 kg and less than or equal to 125 kg
Presenting at least 1 neurological symptom of the disease
Written informed consent
Willing and capable to participate in all aspects of trial design
Ability to travel to the trial site at scheduled times
Contraception requirements per protocol
Caregiver consent as appropriate to participate in all protocol-specified assessments for duration of trial
Inclusion criteria for Open Label Extension are 1) Received double-blind treatment for at least 48 weeks with CGI-S deterioration by at least 2 levels for 2 consecutive assessment visits 12 weeks apart, or 2) completion of double-blind treatment and completed all assessments through week 96, or 3) Discontinued early from double-blind treatment but completed all assessments through week 96
Inclusion criteria for patients age 0 to 3 years in open-label sub-study in countries following EMA guidance only: Confirmed diagnosis of NPC1; treated or not with Miglustat per main study; body weight greater than 4.5kg; patient may be asymptomatic; written assent for child to participate in safety assessments; caregiver consent to participate in caregiver assessments; ability to travel to the trial site for all scheduled visits.

You may not qualify if…

Recipient of a liver transplant within <12 months or planned liver transplantation
Patients with active liver disease from any cause other than NPC1
Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or international normalized ratio > 1.8
Stage 3 chronic kidney disease or worse as indicated by an estimated glomerular filtration rate <60ml/min/1.73m2.
Use of curcumin or fish oil within 12 weeks prior to enrollment
Known or suspected allergy or intolerance to the study treatment
In the opinion of the Investigator, the patient's clinical condition does not allow for the blood collection required as per protocol specific procedures.
Treatment with any investigational drug during the 3 months prior to entering the study. If the investigational drug has a short half-life (<8 hours) and would be expected to be cleared from the body within 1 month, then the wash-out period is 1 month. Treatment with any form of leucine, whether as an investigational drug or other formulation is not allowed
Treatment with any other investigational drug during the study
Pregnancy or breastfeeding
Current participation in another trial is not permitted unless it is a noninterventional study and the sole purpose of the trial is for long-term follow up describing clinical features or survival data (registry)
Patients with uncontrolled, severe epileptic seizure periods (at least 3 consecutive severe epileptic seizures that required medication) within 2 months prior to completion of informed consent or assent, as applicable.
Neurologically asymptomatic patients
Inability to participate in the primary study assessment (4D-NPC-SS or 5D-NPC-SS) as determined by the Investigator
Exclusion criteria for patients age 0 to 3 years in open-label sub-study in countries following EMA guidance only are similar to the main study with the addition of exclusion criterion of history of fetal hydrops or fetal ascites

Where it is running

UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
University of Florida — Jacksonville, Florida, United States
Emory — Atlanta, Georgia, United States
Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
UPMC Children's Hospital — Pittsburgh, Pennsylvania, United States
University Utah — Salt Lake City, Utah, United States
Lysosomal and Rare Disorders Research & Treatment Center, Inc. — Fairfax, Virginia, United States
Hospital de Alta Complejidad en Red "El Cruce" — Buenos Aires, Argentina
Hospital de Niños de la Santísima Trinidad — Córdoba, Argentina
Melbourne Children's Trials Centre Murdoch Children's Research Institute — Parkville, Victoria, Australia
Royal Melbourne Hospital — Parkville, Victoria, Australia
Metabolic Clinical Trials Unit — Adelaide, Australia
Hospital de Clínicas de Porto Alegre — Porto Alegre, Brazil
Universidade de São Paulo — São Paulo, Brazil
University of Campinas — São Paulo, Brazil
SphinCS GmbH — Höchheim, Germany
University Munster — Münster, Germany
Emek Medical Center-Department of Pediatrics — Afula, Israel
Soroka Medical Center — Beersheba, Israel
University of Catania — Catania, Italy
Istituto Neurologico Carlo Besta — Milan, Italy
University Hospital of Padova — Padova, Italy
Centro di Coordinamento Regionale Malattie Rare — Udine, Italy
Szpital Uniwersytecki w Krakowie — Krakow, Poland
MediPark — Warsaw, Poland

Full record on ClinicalTrials.gov

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