Trial · NCT03887533
Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
NCT03887533, Phase 1, Phase 2, Terminated, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); results first posted 2022-08-30
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1. Trials, record NCT03887533. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03887533
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
- NCT number
- NCT03887533
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 2 participants, actual
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Conditions
- Niemann-Pick Disease, Type C1
- Interventions
- VTS-270
- First posted
- 2019-03-25
- Last updated
- 2022-08-30
- Results first posted
- 2022-08-30
- Limitations and caveats
- Enrollment was poor due to COVID-19 pandemic
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Participants With Adverse Events by Grade | 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 life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL\*\*. Grade 4 Life-threatening consequences; urgent intervention indicated. Grade 5 Death related to AE |
| Primary | Participants With Reduction in Plasma Cholestane-3β | 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 |
| Primary | Participants With Reduction in Plasma Bile Acid B (5α) | 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 |
| Primary | Participants With Reduction in C-Triol (6β-triol) | 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 |
| Secondary | Participants With Reduction in Liver Stiffness (kPa) | 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) |
| Secondary | Participants With Reduction in Alanine Aminotransferase Level | 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) |
| Secondary | Participants With Reduction in Aspartate Aminotransferase | 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 |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Participants With Adverse Events by Grade | 18 months | All participants who completed the study | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Participants With Reduction in Plasma Cholestane-3β | Assessed at baseline and at 52 weeks | All participants who completed the study | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Participants With Reduction in Plasma Bile Acid B (5α) | Assessed at baseline and at 52 weeks | All participants who completed the study | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Participants With Reduction in C-Triol (6β-triol) | Assessed at baseline and at 52 weeks | All participants who completed the study | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Participants With Reduction in Liver Stiffness (kPa) | Assessed at baseline and at 52 weeks | All participants who completed the study | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Participants With Reduction in Alanine Aminotransferase Level | Assessed at baseline and at 52 weeks | All participants who completed the study | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Participants With Reduction in Aspartate Aminotransferase | Assessed at baseline and at 52 weeks | All participants who completed the study | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group22
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Participants With Adverse Events by Grade | VTS-270 at 500 mg/kg | Grade 1 | 1 | Participants | |
| Participants With Adverse Events by Grade | VTS-270 at 1000 mg/kg | Grade 1 | 1 | Participants | |
| Participants With Adverse Events by Grade | VTS-270 at 500 mg/kg | Grade 2 | 1 | Participants | |
| Participants With Adverse Events by Grade | VTS-270 at 1000 mg/kg | Grade 2 | 1 | Participants | |
| Participants With Adverse Events by Grade | VTS-270 at 500 mg/kg | Grade 3 | 0 | Participants | |
| Participants With Adverse Events by Grade | VTS-270 at 1000 mg/kg | Grade 3 | 0 | Participants | |
| Participants With Adverse Events by Grade | VTS-270 at 500 mg/kg | Grade 4 | 0 | Participants | |
| Participants With Adverse Events by Grade | VTS-270 at 1000 mg/kg | Grade 4 | 0 | Participants | |
| Participants With Adverse Events by Grade | VTS-270 at 500 mg/kg | Grade 5 | 0 | Participants | |
| Participants With Adverse Events by Grade | VTS-270 at 1000 mg/kg | Grade 5 | 0 | Participants |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | VTS-270 at 500 mg/kg | 1 | |
| Overall Study | STARTED | VTS-270 at 1000 mg/kg | 1 | |
| Overall Study | COMPLETED | VTS-270 at 500 mg/kg | 1 | |
| Overall Study | COMPLETED | VTS-270 at 1000 mg/kg | 1 | |
| Overall Study | NOT COMPLETED | VTS-270 at 500 mg/kg | 0 | |
| Overall Study | NOT COMPLETED | VTS-270 at 1000 mg/kg | 0 |
Source ClinicalTrials.gov
Adverse events58
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 | Eosinophilia | Blood and lymphatic system disorders | VTS-270 at 500 mg/kg | 0 | 1 | 0 |
| other | Eosinophilia | Blood and lymphatic system disorders | VTS-270 at 1000 mg/kg | 1 | 1 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | VTS-270 at 500 mg/kg | 1 | 1 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | VTS-270 at 1000 mg/kg | 0 | 1 | 0 |
| other | Dizziness | Cardiac disorders | VTS-270 at 500 mg/kg | 1 | 1 | 1 |
| other | Dizziness | Cardiac disorders | VTS-270 at 1000 mg/kg | 0 | 1 | 0 |
| other | Flatulence | Gastrointestinal disorders | VTS-270 at 500 mg/kg | 0 | 1 | 0 |
| other | Flatulence | Gastrointestinal disorders | VTS-270 at 1000 mg/kg | 1 | 1 | 1 |
| other | Constipation | Gastrointestinal disorders | VTS-270 at 500 mg/kg | 0 | 1 | 0 |
| other | Constipation | Gastrointestinal disorders | VTS-270 at 1000 mg/kg | 1 | 1 | 1 |
1 to 10 of 58Source ClinicalTrials.gov
See all 58 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | VTS-270 at 500 mg/kg | 0 | 1 |
| Total, all-cause mortality | VTS-270 at 1000 mg/kg | 0 | 1 |
| Total, other adverse events | VTS-270 at 500 mg/kg | 1 | 1 |
| Total, other adverse events | VTS-270 at 1000 mg/kg | 1 | 1 |
| Total, serious adverse events | VTS-270 at 500 mg/kg | 1 | 1 |
| Total, serious adverse events | VTS-270 at 1000 mg/kg | 0 | 1 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
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.
None in this release.
Trial comparisons8
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in Liver Stiffness (kPa) | Trial comparisons |
| VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in Plasma Bile Acid B (5α) | Trial comparisons |
| VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in Aspartate Aminotransferase | Trial comparisons |
| VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in Plasma Cholestane-3β | Trial comparisons |
| VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in C-Triol (6β-triol) | Trial comparisons |
| VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: Participants With Reduction in Alanine Aminotransferase Level | Trial comparisons |
| VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: deaths from any cause | Trial comparisons |
| VTS-270 at 500 mg/kg vs VTS-270 at 1000 mg/kg: serious adverse events | Trial comparisons |
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/NCT03887533
GET https://conductscience.org/api/trials/v1/NCT03887533/results?page=2