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Cyclodextrin
Trappsol Cyclo for Niemann-Pick Disease (TransportNPC Trial)
Phase 3
Recruiting
Research Sponsored by Cyclo Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Body weight greater than 4.5 kg and less than or equal to 125 kg
Ability to travel to the trial site at scheduled times
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, weeks 2, 4, 12, 24, 36, 48, 60, 72, 84, 96, 100, 120, 144, 168, 192
Awards & highlights
TransportNPC Trial Summary
This trial is testing a new treatment for Niemann Pick disease type C1, a rare and fatal disorder. The new treatment is being compared to the standard of care to see if it is safe, effective, and well tolerated. The trial will also enroll infants and young children to evaluate safety in that population.
Who is the study for?
This trial is for pediatric and adult patients with Niemann-Pick Disease Type C1. Participants must have a confirmed diagnosis, be able to follow the study's procedures, and weigh between 4.5 kg and 125 kg. They should not have severe kidney disease, recent liver transplant or active liver disease unrelated to NPC1, nor be pregnant or breastfeeding.Check my eligibility
What is being tested?
The trial tests Trappsol Cyclo (hydroxypropyl betacyclodextrin) administered intravenously at a dose of 2000 mg/kg against a placebo in patients with NPC1. It aims to assess safety, tolerability, and effectiveness compared to standard care.See study design
What are the potential side effects?
While specific side effects are not listed here, participants will be monitored for any adverse reactions due to Trappsol Cyclo treatment which could include issues related to infusion such as discomfort or allergic reactions.
TransportNPC Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My weight is between 4.5 kg and 125 kg.
Select...
I can travel to the trial location when needed.
Select...
I am experiencing at least one symptom related to my nervous system.
Select...
I have been diagnosed with NPC1.
TransportNPC Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline, weeks 2, 4, 12, 24, 36, 48, 60, 72, 84, 96, 100, 120, 144, 168, 192
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, weeks 2, 4, 12, 24, 36, 48, 60, 72, 84, 96, 100, 120, 144, 168, 192
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change from Baseline in 4-Domain NPC Severity Score (US only)
Change from Baseline in 5-Domain NPC Severity Score (ex-US)
Secondary outcome measures
Change in Swallow function evaluated by videofluoroscopy or fiberoptic endoscopy and measured by Penetration Aspiration Scale
Change in adaptive behavior as measured by Vineland Adaptive Behavior Scale II
Change in ataxia as measured by Spinocerebellar ataxia functional index
Other outcome measures
Caregiver Global Impression of Change at 24 hours post infusion
Change from Baseline in Liver function as measured by liver enzyme assessments
Change from Baseline in Respiratory function measured by Forced Expiratory Volume in 1 second
+5 moreTransportNPC Trial Design
3Treatment groups
Experimental Treatment
Placebo Group
Group I: Open Label sub-study for Infants up to age 3Experimental Treatment1 Intervention
Up to 12 patients age 0 - 3 yrs in countries following EMA guidance may be enrolled in this open label sub-study. All patients will receive 2000 mg/kg hydroxypropyl betacyclodextrin (Trappsol Cyclo) diluted with 0.5N saline at the clinician's discretion over 6.5 hours every 2 weeks. Outcome measures are safety, clinician and caregiver impressions.
Group II: ExperimentalExperimental Treatment1 Intervention
Intravenous administration of 2000 mg/kg hydroxypropyl betacyclodextrin (Trappsol Cyclo) (based on body weight) diluted with 0.5N saline over at least 6.5 hours every 2 weeks
Group III: Placebo comparatorPlacebo Group1 Intervention
Intravenous administration of 0.5N saline over at least 6.5 hours every 2 weeks
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Hydroxypropyl-beta-cyclodextrin
2017
Completed Phase 2
~30
Find a Location
Who is running the clinical trial?
Cyclo Therapeutics, Inc.Lead Sponsor
5 Previous Clinical Trials
127 Total Patients Enrolled
Lise Kjems, MDStudy DirectorCyclo Therapeutics, Inc.
Sharon Hrynkow, PhDStudy DirectorCyclo Therapeutics, Inc.
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Eligibility Criteria:
This trial includes the following eligibility criteria:- My weight is between 4.5 kg and 125 kg.I have been on a stable dose of Miglustat for 3 months or stopped it 3 months ago.Your condition score falls between 0.5 and 2.0 on a scale that measures the severity of your symptoms.My child does not have a history of fetal hydrops or ascites.I've had severe, uncontrolled seizures needing medication in the last 2 months.I can travel to the trial location when needed.I am willing and able to follow all the study requirements.My kidney function is reduced, with a filtration rate below 60ml/min.My child is 0-3 years old, diagnosed with NPC1, weighs more than 4.5kg, and can travel for the trial.I completed or participated significantly in a previous phase of the trial and met all required assessments.My liver disease is not caused by NPC1.I haven't taken any experimental drugs or leucine in the last 3 months.I am experiencing at least one symptom related to my nervous system.I had a liver transplant less than a year ago or am planning to have one.I have been diagnosed with NPC1.I have signs of acute liver disease, like jaundice or pain in my upper right abdomen.I do not have any symptoms related to my nervous system.I have not taken curcumin or fish oil in the last 12 weeks.
Research Study Groups:
This trial has the following groups:- Group 1: Open Label sub-study for Infants up to age 3
- Group 2: Experimental
- Group 3: Placebo comparator
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any current vacancies for participants in this research?
"Yes, this information is accurate. The last update to this posting on clinicaltrials.gov was on 9/15/2022 and recruitment for patients is still active."
Answered by AI
Has the FDA acknowledged Hydroxypropyl-beta-cyclodextrin as a safe substance?
"Hydroxypropyl-beta-cyclodextrin's safety is estimated to be a 3. This is based off it being in Phase 3 clinical trials, which suggests that not only is there some data supporting efficacy, but also multiple rounds of data affirming its safety."
Answered by AI
This clinical trial is being run at how many different sites?
"Currently, there are 4 sites where patients can enroll in this trial. The locations of these centres are Pittsburgh, Cincinnati, Fairfax and other cities. If you do decide to participate in the study, try to select a location nearest you to limit travel."
Answered by AI
How many people total will be included in this scientific research?
"The sponsor, Cyclo Therapeutics, Inc., needs to recruit 93 patients that meet the inclusion criteria in order to start the trial. The research will be conducted at multiple hospitals, with UPMC Children's Hospital in Pittsburgh and Cincinnati Children's Hospital Medical Center being two of them."
Answered by AI
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