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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.

Must not have

Age > 6 months at time of enrollment in the trial.

History of renal disease or evidence of acute kidney injury defined as serum creatinine greater than 1.5 mg/dL or an increase of at least 0.2-0.3 mg/dL per day.

Timeline

Screening 3 weeks

Treatment Varies

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

Awards & highlights

No Placebo-Only Group

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am older than 6 months.

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I have kidney problems or my creatinine levels are high.

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I had a severe brain injury as a newborn with specific symptoms.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Secondary study objectives

Effect of drug on serum transaminases

Reduction of liver and/or spleen volumes

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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 / 7Eligibility criteria met