Trappsol Cyclo for Niemann-Pick Disease

(TransportNPC Trial)

Not currently recruiting at 41 trial locations

Overseen By Lori M Gorski

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Cyclo Therapeutics, Inc.

Must not be taking: Curcumin, Fish oil, Leucine, others

Disqualifiers: Liver disease, Kidney disease, Epilepsy, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 96 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of Trappsol Cyclo for Niemann-Pick Disease Type C1 (NPC1), a rare genetic disorder that impairs the body's ability to process cholesterol and other fats, leading to serious neurological issues. The study compares Trappsol Cyclo, administered through an IV (intravenous infusion), to standard care to evaluate its efficacy and safety. Individuals diagnosed with NPC1 who experience at least one neurological symptom might be suitable for the trial. Additionally, a special part of the study in some countries focuses on infants up to age 3 to assess the treatment's safety in younger patients. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Do I need to stop my current medications to join the trial?

The trial does not specify if you need to stop all current medications, but you must not use curcumin or fish oil within 12 weeks before enrolling. If you are taking an investigational drug, you need to stop it at least 3 months before the study, unless it has a short half-life, in which case the washout period is 1 month.

Is there any evidence suggesting that Trappsol Cyclo is likely to be safe for humans?

Research has shown that Trappsol Cyclo, a type of hydroxypropyl betacyclodextrin, is generally safe for people with Niemann-Pick Disease Type C1. In earlier studies, most side effects were mild, with 80% being mild and often related to the disease itself, not the treatment. Some moderate side effects occurred, but they were short-lived. This suggests that the treatment is mostly safe for people. Ongoing research continues to support its safety for long-term use. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Niemann-Pick disease?

Trappsol Cyclo is unique because it uses hydroxypropyl betacyclodextrin, a compound that helps transport cholesterol out of cells. Unlike other treatments for Niemann-Pick Disease, which may focus on managing symptoms, Trappsol Cyclo targets the underlying cholesterol accumulation in cells, a key issue in this condition. Researchers are excited about its potential to directly address the cause of Niemann-Pick Disease rather than just alleviating its symptoms. Furthermore, its intravenous delivery every two weeks offers a structured approach that could enhance patient compliance and outcomes.

What evidence suggests that Trappsol Cyclo might be an effective treatment for Niemann-Pick Disease?

Research has shown that Trappsol Cyclo, administered through an IV, offers potential benefits for individuals with Niemann-Pick disease type C1 (NPC1). In this trial, participants will receive Trappsol Cyclo to evaluate its safety and effectiveness using various health measures. Previous studies yielded promising results, particularly for young patients, including newborns, with NPC1. Early treatment with Trappsol Cyclo demonstrated improved safety and suggests it may help manage the disease. These findings support further research into its use for NPC1 patients. ^{1 2 3 6 7}

Who Is on the Research Team?

Karen Mullen, MD

Principal Investigator

Cyclo Therapeutics, Inc.

Are You a Good Fit for This Trial?

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.

Inclusion 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.

Exclusion Criteria

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)

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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 2000 mg/kg of Trappsol Cyclo or placebo intravenously every two weeks

96 weeks

Bi-weekly visits (in-person)

Interim Analysis

Unblinded interim analysis to evaluate efficacy and safety

48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt into continuation of treatment long-term

Up to 96 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Trappsol(R) Cyclo(TM)

Trial Overview 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.

How Is the Trial Designed?

Group I: Open Label sub-study for Infants up to age 3 Experimental Treatment 1 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: Experimental Experimental Treatment 1 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 comparator Placebo Group 1 Intervention

Intravenous administration of 0.5N saline over at least 6.5 hours every 2 weeks

Who Is Running the Clinical Trial?

Cyclo Therapeutics, Inc.

Lead Sponsor

Trials

6

Recruited

220+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/36279795/

Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® ... These data support the initiation of longer-term clinical trials to evaluate the safety and efficacy of intravenous HPβCD in subjects with NPC1. (ClinicalTrials ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S2214426923000344

Long-term administration of intravenous Trappsol® Cyclo ... This 48-week trial is the longest to date to evaluate the safety, tolerability, and efficacy across multiple clinical endpoints of IV administration of Trappsol ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT04860960

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo ... The objective of this study is to evaluate the safety, tolerability and efficacy of 2000 mg/kg dose of Trappsol Cyclo (hydroxypropyl betacyclodextrin) ...

4. cyclotherapeutics.com cyclotherapeutics.com/cyclo-therapeutics-presents-encouraging-preliminary-safety-data-from-ongoing-pivotal-phase-3-study-and-substudy-for-the-treatment-of-niemann-pick-disease-type-c1-npc1/

News A total of 625 Adverse Events (AEs) were reported, 80% were Grade 1 (mild) in severity most related to underlying NPC disease; Grade 2 were 16.7 ...

5. neurologylive.com neurologylive.com/view/trappsol-cyclo-shows-promising-results-newborns-niemann-pick-disease-type-c1

Trappsol Cyclo Shows Promising Results in Newborns ... New findings reveal early treatment with Trappsol Cyclo shows promise for young patients with Niemann-Pick disease type C1, enhancing safety ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT02912793?term=AREA%5BInterventionSearch%5D(%22Betadex%22)&rank=10

Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD ... This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann-Pick disease Type C1 (NPC-1) ...

7. firstwordpharma.com firstwordpharma.com/story/5973706

Rafael Holdings Announces Continuation of its Phase 3 ... Data on the investigational candidate Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin), indicates that it is well-tolerated and has a safety ...