A Phase 1/2 interventional study of VTS-270 in Niemann-Pick Disease, Type C, sponsored by Washington University School of Medicine. Completed at 1 site in United States. Open to participants aged Up to 6 Months. Per ClinicalTrials.gov, last updated 2026-01-05.
Sponsored by Washington University School of Medicine · Phase 1/2, Interventional, and Treatment
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Among 23 studies in Niemann-Pick Disease, Type C with a site in United States — 6 open to participants now, 8 under way, 4 of them phase 3. Niemann-Pick Disease, Type C · United States.
Among the 23 studies in Niemann-Pick Disease, Type C with a site in United States, this one is the 16th largest by enrollment of the 17 interventional ones that give a count, is one of 3 that started in 2019, is one of 4 in phase 1/2, and was the 10th registered.
37 studies on the registry are indexed under Niemann-Pick Disease, Type C; 8 are open to participants now.
Browse Niemann-Pick Disease, Type C studies →6 of the 23 studies in Niemann-Pick Disease, Type C with a site in United States are open now. Recruiting first, the newest first:
Azafaros B.V. · Sites in United States, Argentina, Australia and 14 more countries
Ages 4+
Azafaros B.V. · Sites in United States, Argentina, Australia and 12 more countries
Ages 4+
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · Sites in United States
Ages 3 mo+
IntraBio Inc · Sites in United States, Australia, Czechia and 5 more countries
Ages 4+
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · Sites in United States
Ages 1 days–120 yrs · Healthy volunteers
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 5 Niemann-Pick Disease, Type C studies recruiting now, 4 set no upper age limit and none stop at 75 or younger; 5 take children under 18; 1 takes healthy volunteers.
This study: Up to 6 Months · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
23 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 7 · Phase 2 9 · Phase 3 8
First in 2006; this study’s year is marked. 3 started in 2019.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 4 is below the median of 21 across 27 interventional studies indexed under Niemann-Pick Disease, Type C.
Median enrollment 33 across the 17 interventional studies, 900 across the 5 observational. Of the 13 that completed or were terminated, 10 (77%) have results posted.
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13 of 23 have an industry lead sponsor. The 10 most frequent:
Washington University School of Medicine is the lead sponsor of 1,804 studies on the registry; 270 are open to participants now.
Of its 329 completed or terminated interventional studies of FDA-regulated products, 216 (66%) have results posted.
Washington University School of Medicine has 1 study filed under Niemann-Pick Disease, Type C — fewer than ten, so there is no comparison to draw.
2 of the studies in Niemann-Pick Disease, Type C with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
| NCT05588167Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C | 2027-09-30 |
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Among the 23 studies in Niemann-Pick Disease, Type C with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
Niemann-Pick disease, type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence. NPC results from mutation of either the Niemann-Pick C1 disease (NPC1) (\~95% of cases) or NPC2 genes. NPC is characterized by the endolysosomal storage of unesterified cholesterol and lipids in both the central nervous system and peripheral tissues such as the liver. Individuals with NPC demonstrate progressive central nervous system decline including inability to coordinate balance, gait, extremity and eye movements. Acute liver disease in the newborn/infant period is frequently observed, but subsequently resolves. However, chronic, sub-clinical liver disease persists. Intrathecal 2-Hydroxypropyl-β-Cyclodextrin (HP-β-CD, VTS-270), also known as adrabetadex, has proven effective in reducing the signs and prolonging life in animal models and Phase 1/2a data support efficacy in NPC1 patients. Adrabetadex (VTS-270) also has been shown to be effective in treating liver disease in the NPC1 cat.
This Phase 1/2a, open-label, multiple ascending dose trial will evaluate whether adrabetadex (VTS-270) administered intravenously is effective in treating acute liver disease in NPC1 infants.
In the first phase of the study, infants will be treated for a total of 6 weeks, treated twice weekly. Infants will be admitted to the Neonatal Intensive Care Unit (NICU) for the first week of treatment. Procedures during the first week of the study will include blood draws for genetic testing, clinical and research blood draws, urine collection, abdominal ultrasound, peripheral inserted central catheter (PICC) placement, hearing screening, and the first two IV adrabetadex (VTS-270) infusions through the PICC line. Weeks 2-6 will occur on an outpatient basis. During week 2-6, the infant will receive 2 doses per week of adrabetadex (VTS-270) with blood draws and urine collection during weeks 2, 4, and 6. PICC line will be removed after final infusion.
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 which IV adrabetadex (VTS-270) will be administered monthly for a total of six doses. Month 1-6 procedures will occur on an outpatient basis. Procedures during the second phase include a monthly intravenous line placement. After each monthly visit, the intravenous line will be removed.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Diagnosis of NPC (either NPC1 or NPC2) based upon meeting any of the two following conditions:
A. Two variants classified as pathogenic or likely pathogenic in NPC1/NPC2 on clinical laboratory testing, or B. One variant classified as pathogenic or likely pathogenic on clinical laboratory testing and a positive NPC biochemical marker (oxysterol or bile acid biomarker or PPCS/Lyso509) test, if acid sphingomyelinase deficiency and cholesterol ester storage disease have been excluded either by clinical molecular testing of the SMPD1 and LIPA genes or by clinical biochemical assay for acid sphingomyelinase and lysosomal acid lipase enzymes (or a combination of enzymatic and molecular testing).
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.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Drug: VTS-270
VTS-270 (2-Hydroxypropyl-Beta-Cyclodextrin) will be administered intravenously to specifically target liver disease. In the first phase of the study, dosing frequency will be twice a week with IV adrabetadex (VTS-270) for six weeks for a total of 12 administrations. Subjects will be evaluated at each study visit for evidence of adverse effects. Doses to be studied are 500, and 1000 mg/kg. Six subjects will be studied at each dose level. Cohort 1: Subjects 1-6, 500 mg/kg Cohort 2: Subjects 7-12, 1000 mg/kg
Also known as: adrabetadex
Efficacy of Adrabetadex (VTS-270) to Reduce Plasma Levels of a Conjugated Bile Acid, Known as 5α-cholanic Acid-3β, 5α, 6β-triol N-(Carboxymethyl)-Amide
This bile acid, 5α-cholanic acid-3β, 5α, 6β-triol N-(carboxymethyl)-amide, is elevated \>99% of NPC1 subjects, is largely generated in the liver and therefore provides a biochemical measure of oxidizable lysosomal unesterified cholesterol in liver tissue. The primary outcome measure in phase 1 of the study is the change in plasma levels of 5α-cholanic acid-3β, 5α, 6β-triol N-(carboxymethyl)-amide measured in nanograms/ml from baseline to 6 weeks. The primary outcome measure in phase 2 of the study is the change in plasma levels of 5α-cholanic acid-3β, 5α, 6β-triol N-(carboxymethyl)-amide measured in nanograms/ml from 6 weeks to the end of the 6 month treatment period.
Time frame: Phase 1: 6 weeks; Phase 2: 6 months
Effect of Drug on Serum Transaminases
Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) are elevated with liver dysfunction. The outcome measure in phase 1 of the study is the change in ALT and AST levels from baseline to 6 weeks. The outcome measure in phase 2 of the study is the change in ALT and AST levels from 6 weeks to the end of the 6 month treatment period.
Time frame: Phase 1: 6 weeks; Phase 2: 6 months
Reduction of Liver and/or Spleen Volumes
Abdominal ultrasound. The outcome measure in phase 1 of the study is the change in liver and/or spleen volumes from baseline to 6 weeks. The outcome measure in phase 2 of the study is the change in liver and/or spleen volumes from 6 weeks to the end of the 6 month treatment period. We were only able to collect spleen volume data due to the radiologist reporting they could not accurately obtain liver volume data. The contact radiologist for this protocol has left the institution, stopping any additional work on liver ultrasound volumes. None of the remaining radiology colleagues had time to bring forward. Liver volumes will never be obtained for this measure in the future.
Time frame: Phase 1: 6 weeks; Phase 2: 6 months
The original enrollment goal was 12 subjects up to maximum of 15 subjects to account for potential study withdrawals, discontinuations or unconfirmed diagnosis of NPC. Six subjects would be studied at each dose level. Cohort 1: Subjects 1-6 500mg/kg Cohort 2: Subjects 7-12 1000mg/kg The four subjects enrolled received 500mg/kg study drug.
| Milestone | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| Started | 4 |
| Completed | 3 |
| Not completed | 1 |
This bile acid, 5α-cholanic acid-3β, 5α, 6β-triol N-(carboxymethyl)-amide, is elevated \>99% of NPC1 subjects, is largely generated in the liver and therefore provides a biochemical measure of oxidizable lysosomal unesterified cholesterol in liver tissue. The primary outcome measure in phase 1 of the study is the change in plasma levels of 5α-cholanic acid-3β, 5α, 6β-triol N-(carboxymethyl)-amide measured in nanograms/ml from baseline to 6 weeks. The primary outcome measure in phase 2 of the study is the change in plasma levels of 5α-cholanic acid-3β, 5α, 6β-triol N-(carboxymethyl)-amide measured in nanograms/ml from 6 weeks to the end of the 6 month treatment period.
| ng/ml | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| 6 weeks | 32.6 (10.5 to 129) |
| 6 months | 87.9 (46.6 to 101) |
Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) are elevated with liver dysfunction. The outcome measure in phase 1 of the study is the change in ALT and AST levels from baseline to 6 weeks. The outcome measure in phase 2 of the study is the change in ALT and AST levels from 6 weeks to the end of the 6 month treatment period.
| U/L | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| ALT 6 weeks | 87 (80 to 203) |
| AST 6 weeks | 114 (86 to 293) |
| ALT 6 months | 65 (48 to 71) |
| AST 6 months | 77 (75 to 89) |
Abdominal ultrasound. The outcome measure in phase 1 of the study is the change in liver and/or spleen volumes from baseline to 6 weeks. The outcome measure in phase 2 of the study is the change in liver and/or spleen volumes from 6 weeks to the end of the 6 month treatment period. We were only able to collect spleen volume data due to the radiologist reporting they could not accurately obtain liver volume data. The contact radiologist for this protocol has left the institution, stopping any additional work on liver ultrasound volumes. None of the remaining radiology colleagues had time to bring forward. Liver volumes will never be obtained for this measure in the future.
| cc | Treatment Group |
|---|---|
| 6 weeks | 71.8 (58.6 to 98.1) |
| 6 months | 127 (69.2 to 177.2) |
Collected over 7 months and 2 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| IV Adrabetadex (VTS-270) for NPC1 Infants | 1/4 (25%) | 3/4 (75%) | 2/4 (50%) |
| Event | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| HospitalizationInfections and infestations | 3/4 |
| DeathHepatobiliary disorders | 1/4 |
| Event | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| small umbilical herniaGeneral disorders | 2/4 |
| elevated prothrombin timeHepatobiliary disorders | 2/4 |
| occasional cough and nasal congestionRespiratory, thoracic and mediastinal disorders | 2/4 |
| ankle clonusNervous system disorders | 2/4 |
| increased patellar reflexesNervous system disorders | 2/4 |
| upper respiratory infectionRespiratory, thoracic and mediastinal disorders | 1/4 |
| limited vertical gazeEye disorders | 1/4 |
| 1/6 systolic murmurCardiac disorders | 1/4 |
| dry skin and maculopapular rash on both cheeksSkin and subcutaneous tissue disorders | 1/4 |
| diaper rashSkin and subcutaneous tissue disorders | 1/4 |
| Age, Categorical(Participants) | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| <=18 years | 4 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| Female | 1 |
| Male | 3 |
| Ethnicity (NIH/OMB)(Participants) | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| Hispanic or Latino | 3 |
| Not Hispanic or Latino | 1 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 4 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Plasma level of 5α-cholanic acid-3β, 5α, 6β-triol N-(carboxymethyl)-amide(ng/ml) | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| Median | 120.6 (96.2 to 145) |
| Aspartate aminotransferase (AST)(U/L) | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| Median | 168 (167 to 169) |
| Alanine transaminase (ALT)(U/L) | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| Median | 158.5 (124 to 193) |
| Spleen volume(cc) | IV Adrabetadex (VTS-270) for NPC1 Infants |
|---|---|
| Median | 86.3 (84.2 to 88.4) |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Feb 2019 to primary completion in Oct 2024, 67 months.
Results were first posted in Dec 2025, 1 year 2 months after primary completion.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: Adrabetadex
Niemann-Pick Disease, Type C →
Washington University School of Medicine →