Trial · NCT03471143
Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
NCT03471143, Phase 1, Phase 2, Completed, Washington University School of Medicine; results first posted 2025-12-12
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C. Trials, record NCT03471143. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03471143
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
- NCT number
- NCT03471143
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 4 participants, actual
- Lead sponsor
- Washington University School of Medicine
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- VTS-270
- First posted
- 2018-03-20
- Last updated
- 2026-01-05
- Results first posted
- 2025-12-12
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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 | Phase 1: 6 weeks; Phase 2: 6 months | 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. |
| Secondary | Effect of Drug on Serum Transaminases | Phase 1: 6 weeks; Phase 2: 6 months | 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. |
| Secondary | Reduction of Liver and/or Spleen Volumes | Phase 1: 6 weeks; Phase 2: 6 months | 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. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | 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 | Phase 1: 6 weeks; Phase 2: 6 months | One of the participants only had baseline measurement. | ng/ml | MEDIAN |
| SECONDARY | Effect of Drug on Serum Transaminases | Phase 1: 6 weeks; Phase 2: 6 months | One of the participants only had baseline measurement. | U/L | MEDIAN |
| SECONDARY | Reduction of Liver and/or Spleen Volumes | Phase 1: 6 weeks; Phase 2: 6 months | 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 | MEDIAN |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| 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 | IV Adrabetadex (VTS-270) for NPC1 Infants | 6 weeks | 32.6 | Inter-Quartile Range 10.5 to 129 | ng/ml |
| 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 | IV Adrabetadex (VTS-270) for NPC1 Infants | 6 months | 87.9 | Inter-Quartile Range 46.6 to 101 | ng/ml |
| Effect of Drug on Serum Transaminases | IV Adrabetadex (VTS-270) for NPC1 Infants | ALT 6 weeks | 87 | Inter-Quartile Range 80 to 203 | U/L |
| Effect of Drug on Serum Transaminases | IV Adrabetadex (VTS-270) for NPC1 Infants | AST 6 weeks | 114 | Inter-Quartile Range 86 to 293 | U/L |
| Effect of Drug on Serum Transaminases | IV Adrabetadex (VTS-270) for NPC1 Infants | ALT 6 months | 65 | Inter-Quartile Range 48 to 71 | U/L |
| Effect of Drug on Serum Transaminases | IV Adrabetadex (VTS-270) for NPC1 Infants | AST 6 months | 77 | Inter-Quartile Range 75 to 89 | U/L |
| Reduction of Liver and/or Spleen Volumes | Treatment Group | 6 weeks | 71.8 | Inter-Quartile Range 58.6 to 98.1 | cc |
| Reduction of Liver and/or Spleen Volumes | Treatment Group | 6 months | 127 | Inter-Quartile Range 69.2 to 177.2 | cc |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | IV Adrabetadex (VTS-270) for NPC1 Infants | 4 | |
| Overall Study | COMPLETED | IV Adrabetadex (VTS-270) for NPC1 Infants | 3 | |
| Overall Study | NOT COMPLETED | IV Adrabetadex (VTS-270) for NPC1 Infants | 1 |
Source ClinicalTrials.gov
Adverse events38
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | upper respiratory infection | Respiratory, thoracic and mediastinal disorders | IV Adrabetadex (VTS-270) for NPC1 Infants | 1 | 4 | 1 |
| other | limited vertical gaze | Eye disorders | IV Adrabetadex (VTS-270) for NPC1 Infants | 1 | 4 | 1 |
| other | small umbilical hernia | General disorders | IV Adrabetadex (VTS-270) for NPC1 Infants | 2 | 4 | 2 |
| other | 1/6 systolic murmur | Cardiac disorders | IV Adrabetadex (VTS-270) for NPC1 Infants | 1 | 4 | 1 |
| other | dry skin and maculopapular rash on both cheeks | Skin and subcutaneous tissue disorders | IV Adrabetadex (VTS-270) for NPC1 Infants | 1 | 4 | 1 |
| other | elevated prothrombin time | Hepatobiliary disorders | IV Adrabetadex (VTS-270) for NPC1 Infants | 2 | 4 | 2 |
| other | diaper rash | Skin and subcutaneous tissue disorders | IV Adrabetadex (VTS-270) for NPC1 Infants | 1 | 4 | 1 |
| other | right ventricular hypertrophy on ECG | Cardiac disorders | IV Adrabetadex (VTS-270) for NPC1 Infants | 1 | 4 | 1 |
| other | mild truncal hypotonia | Nervous system disorders | IV Adrabetadex (VTS-270) for NPC1 Infants | 1 | 4 | 1 |
| other | oozing around PICC line-blood | Vascular disorders | IV Adrabetadex (VTS-270) for NPC1 Infants | 1 | 4 | 1 |
1 to 10 of 38Source ClinicalTrials.gov
See all 38 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | IV Adrabetadex (VTS-270) for NPC1 Infants | 1 | 4 |
| Total, other adverse events | IV Adrabetadex (VTS-270) for NPC1 Infants | 2 | 4 |
| Total, serious adverse events | IV Adrabetadex (VTS-270) for NPC1 Infants | 3 | 4 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 28803710 | BACKGROUND |
Source PubMed
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| U01HD090845 | Intravenous delivery of 2-hydroxypropyl-Beta-cyclodextrin for treatment of Niemann-Pick C disease | DICKSON, PATRICIA I | WASHINGTON UNIVERSITY | EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT | 2017 to 2022 |
Source NIH RePORTER
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03471143
GET https://conductscience.org/api/trials/v1/NCT03471143/events?page=2