Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Who can join
Adults 3 to 60, any sex, with Niemann-Pick Disease, Type C1. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Participants With Adverse Events by GradePrimary· 18 months
Adverse events (AEs) were used to determine safety and assess safety of intravenous VTS-270 in subjects with Niemann-Pick Disease, type C1. Assessment of safety was made by evaluation of summary statistics of adverse events and unanticipated problems. AEs were assessed using CTCAE version 5.0 grades 1-5.
Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated.
Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental ADL\*.
Grade 3 Severe or medically significant but not immediately
Grade 1
Group
Value
95% CI
VTS-270 at 500 mg/kg
1
VTS-270 at 1000 mg/kg
1
Grade 2
Group
Value
95% CI
VTS-270 at 500 mg/kg
1
VTS-270 at 1000 mg/kg
1
Grade 3
Group
Value
95% CI
VTS-270 at 500 mg/kg
0
VTS-270 at 1000 mg/kg
0
Grade 4
Group
Value
95% CI
VTS-270 at 500 mg/kg
0
VTS-270 at 1000 mg/kg
0
Grade 5
Group
Value
95% CI
VTS-270 at 500 mg/kg
0
VTS-270 at 1000 mg/kg
0
Participants With Reduction in Plasma Cholestane-3βPrimary· Assessed at baseline and at 52 weeks
Efficacy of IV VTS-270 in treating chronic liver disease associated with Niemann-Pick Disease, type C1 as measured by reduction in plasma cholestane-3β, an NPC1-specific pharmacodynamic biomarker
Group
Value
95% CI
VTS-270 at 500 mg/kg
1
VTS-270 at 1000 mg/kg
0
Participants With Reduction in Plasma Bile Acid B (5α)Primary· Assessed at baseline and at 52 weeks
Efficacy of IV VTS-270 in treating chronic liver disease associated with Niemann-Pick Disease, type C1 as measured by reduction in bile acid B (5α), an NPC1-specific pharmacodynamic biomarker
Group
Value
95% CI
VTS-270 at 500 mg/kg
1
VTS-270 at 1000 mg/kg
0
Participants With Reduction in C-Triol (6β-triol)Primary· Assessed at baseline and at 52 weeks
Efficacy of IV VTS-270 in treating chronic liver disease associated with Niemann-Pick Disease, type C1 as measured by reduction in C-Triol (6β-triol), an NPC1-specific pharmacodynamic biomarker
Group
Value
95% CI
VTS-270 at 500 mg/kg
1
VTS-270 at 1000 mg/kg
0
Participants With Reduction in Liver Stiffness (kPa)Secondary· Assessed at baseline and at 52 weeks
Efficacy of IV VTS-270 in treating chronic liver disease associated with Niemann-Pick Disease, type C1 as measured by reduction in liver stiffness (kPa)
Group
Value
95% CI
VTS-270 at 500 mg/kg
0
VTS-270 at 1000 mg/kg
0
Participants With Reduction in Alanine Aminotransferase LevelSecondary· Assessed at baseline and at 52 weeks
Efficacy of IV VTS-270 in treating chronic liver disease associated with Niemann-Pick Disease, type C1 as measured by reduction of serum alanine aminotransferase level (ALT)
Group
Value
95% CI
VTS-270 at 500 mg/kg
0
VTS-270 at 1000 mg/kg
0
Participants With Reduction in Aspartate AminotransferaseSecondary· Assessed at baseline and at 52 weeks
Efficacy of IV VTS-270 in treating chronic liver disease associated with Niemann-Pick Disease, type C1 as measured by reduction of serum aspartate aminotransferase (AST) level
Group
Value
95% CI
VTS-270 at 500 mg/kg
1
VTS-270 at 1000 mg/kg
1
Adverse events — posted to ClinicalTrials.gov
Time frame: 18 months.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Background:
For people who have Niemann-Pick disease, type C1 (NPC1), cholesterol and other fats have trouble moving out of liver and other tissue cells. This makes the cells sick. Researchers want to find out if a drug called VTS-270 can help.
Objective:
To test if VTS-270 is safe and effective in treating chronic liver disease associated with NPC1.
Eligibility:
People ages 3-60 with NPC1
Design:
Participants may be screened by phone or under another protocol.
Participants will have visits once a month for 12 months. If they have intrathecal injections, the study may last 15 months or more. The first visit will last about 5 days. Others will last 2-3 days.
Participants will get VTS-270 injected into a vein at each visit. They can also choose to have intrathecal injections. These are like spinal taps.
Some visits will also include:
Physical exam
Urine tests
Blood tests. A small tube or needle will be inserted into the participants vein to collect blood. The small tube will also be used to give the VTS-270.
Hearing tests: For one test, participants will have electrodes taped to their head. These will record brain waves.
Breathing tests
Ultrasound of abdomen: Sounds waves will take pictures of the participant s body.
Chest x-ray: This is a picture of the lungs.
Publications & conference data
6 peer-reviewed publications reference this trial (live from Europe PMC):
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Last refreshed: 30 August 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03887533.