Trappsol Cyclo for Niemann-Pick Disease

(TransportNPC Trial)

Recruiting at 35 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

94 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a medicine called Trappsol Cyclo, given through an IV, for patients with Niemann Pick disease type C1. The medicine helps remove harmful fat buildup in cells. The study will also look at safety in younger children. Trappsol Cyclo has shown promising results by mobilizing cholesterol, extending life, and reducing neurological damage in Niemann-Pick disease type C1.

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.

What data supports the effectiveness of the drug Trappsol Cyclo for Niemann-Pick Disease?

Research shows that Trappsol Cyclo helps manage cholesterol in the body and brain, which is important for people with Niemann-Pick Disease Type C. Studies found that it can slow down disease progression and improve liver function and neurodegeneration in both humans and animal models. ^{1 2 3 4 5}

How is the drug Trappsol Cyclo different from other treatments for Niemann-Pick Disease?

Trappsol Cyclo is unique because it uses 2-hydroxypropyl-β-cyclodextrin to help restore cholesterol balance in the body and brain, which is crucial for treating Niemann-Pick Disease Type C1. Unlike other treatments, it is administered intravenously and has shown potential in improving liver function and reducing neurodegeneration. ^{1 2 3 4 6}

Research Team

Karen Mullen, MD

Principal Investigator

Cyclo Therapeutics, Inc.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

References

1. United States pubmed.ncbi.nlm.nih.gov

Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1-/- mouse. [2021]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Long-Term Neuropsychological Outcomes from an Open-Label Phase I/IIa Trial of 2-Hydroxypropyl-β-Cyclodextrins (VTS-270) in Niemann-Pick Disease, Type C1. [2021]

5. China pubmed.ncbi.nlm.nih.gov

Niemann-Pick disease treatment: a systematic review of clinical trials. [2020]

6. United States pubmed.ncbi.nlm.nih.gov

Efficacy and ototoxicity of different cyclodextrins in Niemann-Pick C disease. [2019]