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Cyclodextrin

IV VTS-270 for Niemann-Pick Disease

Phase 1 & 2

Recruiting

Led By Patricia I Dickson, MD

Research Sponsored by Washington University School of Medicine

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subjects with evidence of NPC-related liver disease as defined by direct bilirubin (DB) >2mg/dL or DB/total bilirubin ratio >0.2.

Diagnosis of NPC (either NPC1 or NPC2) based upon meeting any of the two following conditions: A. Two NPC1/NPC2 pathological variants, or B. One NPC1/NPC2 pathological variant and a positive NPC biochemical marker (oxysterol or bile acid biomarker or PPCS/Lyso) test Variants will be interpreted using the American College of Medical Genetics guidelines for the interpretation of sequence variants (2015) and testing must be performed by a CLIA-certified laboratory.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up phase 1: 6 weeks; phase 2: 6 months

Awards & highlights

Study Summary

This trial is testing a drug to see if it's effective in treating acute liver disease in infants with Niemann-Pick disease, type C (NPC), which is a rare, fatal disease that causes progressive degeneration of the nervous system.

Who is the study for?

This trial is for infants aged 0-6 months with Niemann-Pick disease type C (NPC) and evidence of NPC-related liver disease. They must be able to travel for the study, undergo blood collections, and have parental consent. Infants with severe immune suppression, kidney injury, low neutrophil or platelet counts, or severe neonatal encephalopathy cannot participate.Check my eligibility

What is being tested?

The trial tests adrabetadex (VTS-270), given intravenously to treat acute liver disease in infants with NPC1. It's an open-label Phase 1/2a study where all participants receive the drug in increasing doses to evaluate its effectiveness and safety.See study design

What are the potential side effects?

While specific side effects are not listed here, potential risks may include reactions at the infusion site, impact on blood cell counts or organ function due to adrabetadex's mechanism of action as observed in animal models and earlier phases.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My liver is affected by my condition, shown by high bilirubin levels.

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I have been diagnosed with NPC based on genetic tests or a combination of genetic and biochemical tests.

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I am a baby aged 0 to 6 months.

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My parent or guardian has agreed in writing for me to join the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ phase 1: 6 weeks; phase 2: 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~phase 1: 6 weeks; phase 2: 6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and phase 1: 6 weeks; phase 2: 6 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Efficacy of adrabetadex (VTS-270) to reduce plasma levels of glycine-conjugated trihydroxycholanic acid ("bile acid biomarker"), an NPC-specific pharmacodynamic biomarker.

Secondary outcome measures

Effect of drug on serum transaminases

Reduction of liver and/or spleen volumes

Trial Design

1Treatment groups

Experimental Treatment

Group I: IV adrabetadex (VTS-270) for NPC1 infantsExperimental Treatment1 Intervention

Phase 1: Dosing frequency will be twice a week administered via a peripherally inserted central catheter (PICC) for six weeks for a total of 12 administrations. Doses 3-12 will occur as an outpatient. Doses to be studied are 500, and 1000 mg/kg. Six subjects will be studied at each dose level. Cohort 1: Subjects 1-6 will receive 500 mg/kg Cohort 2: Subjects 7-12 will receive 1000 mg/kg Subjects who demonstrate significant reduction either in the glycine-conjugated trihydroxycholanic acid biomarker or serum bilirubin (direct bilirubin or direct bilirubin:total bilirubin ratio) will be allowed to crossover into the second phase of the study, an open-label phase of six months duration. In the this phase of the study, dosing frequency will be monthly with IV VTS-270 administered via peripheral IV access for six months for a total of six administrations.

0 / 4Eligibility criteria met

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Who is running the clinical trial?

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NIH

1,965 Previous Clinical Trials

2,672,785 Total Patients Enrolled

Washington University School of MedicineLead Sponsor

1,937 Previous Clinical Trials

2,299,690 Total Patients Enrolled

Patricia I Dickson, MDPrincipal InvestigatorWashington University School of Medicine

2 Previous Clinical Trials

12 Total Patients Enrolled

Media Library

2-Hydroxypropyl-Beta-Cyclodextrin (Cyclodextrin) Clinical Trial Eligibility Overview. Trial Name: NCT03471143 — Phase 1 & 2

Niemann-Pick Disease Research Study Groups: IV adrabetadex (VTS-270) for NPC1 infants

Niemann-Pick Disease Clinical Trial 2023: 2-Hydroxypropyl-Beta-Cyclodextrin Highlights & Side Effects. Trial Name: NCT03471143 — Phase 1 & 2

2-Hydroxypropyl-Beta-Cyclodextrin (Cyclodextrin) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03471143 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many participants are there in the current clinical trial?

"This research has concluded its recruitment efforts. It was initially posted on February 22nd 2019 and last updated on April 11th 2022. Alternative studies exist for Niemann-Pick Disease Type C, with 320 trials currently accepting patients, as well as 1 study recruiting for 2-Hydroxypropyl-Beta-Cyclodextrin."

Answered by AI

What prior research has been conducted utilizing 2-Hydroxypropyl-Beta-Cyclodextrin?

"At present, there is a single clinical trial analyzing 2-Hydroxypropyl-Beta-Cyclodextrin. This research has not progressed to Phase 3 and the majority of its testing sites are located in Saint Louis, Missouri; however, other locations have been identified as suitable for this study."

Answered by AI

Are enrollment slots available for this experimental research?

"Unfortunately, this research is no longer accepting participants. The trial was initially posted on February 22nd 2019 and the most recent update took place on April 11th 2022. For those seeking alternative studies, there are presently 320 clinical trials recruiting for Niemann-Pick Disease Type C and one study actively looking for patients to participate in a 2-Hydroxypropyl-Beta-Cyclodextrin experiment."

Answered by AI

Recent research and studies

The LancetJournal

Intrathecal 2-hydroxypropyl-β-cyclodextrin Decreases Neurological Disease Progression in Niemann-pick Disease, Type C1: A Non-randomised, Open-label, Phase 1–2 Trial

Ory, Daniel S, Elizabeth A Ottinger, Nicole Yanjanin Farhat, Kelly A King, Xuntian Jiang, Lisa Weissfeld, Elizabeth Berry-Kravis, et al.. 2017. “Intrathecal 2-hydroxypropyl-β-cyclodextrin Decreases Neurological Disease Progression in Niemann-pick Disease, Type C1: A Non-randomised, Open-label, Phase 1–2 Trial”. The Lancet. Elsevier BV. doi:10.1016/s0140-6736(17)31465-4.

clinicaltrials.govStudy

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

2019. "Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03471143.

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