Trial · NCT03879655
Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
NCT03879655, Phase 2, Phase 3, Terminated, Mandos LLC; results first posted 2023-12-26
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1. Trials, record NCT03879655. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03879655
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
- NCT number
- NCT03879655
- Status
- Terminated
- Phases
- Phase 2, Phase 3
- Enrollment
- 2 participants, actual
- Lead sponsor
- Mandos LLC
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- VTS-270
- First posted
- 2019-03-19
- Last updated
- 2023-12-26
- Results first posted
- 2023-12-26
- Limitations and caveats
- Study was terminated by the Sponsor and only 2 participants were enrolled in the study.
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Baseline up to Week 156 | An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as an AE with onset on or after the start of adrabetadex treatment. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. |
Source ClinicalTrials.gov
Outcomes1
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Baseline up to Week 156 | All enrolled participants | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group2
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Adrabetadex | All TEAEs | 2 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Adrabetadex | SAEs | 0 | Participants |
Source ClinicalTrials.gov
Participant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Adrabetadex | 2 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Adrabetadex | 2 | |
| Overall Study | COMPLETED | Adrabetadex | 2 | |
| Overall Study | NOT COMPLETED | Adrabetadex | 0 |
Source ClinicalTrials.gov
Adverse events16
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 | Joint injury | Injury, poisoning and procedural complications | Adrabetadex | 1 | 2 | |
| other | Influenza | Infections and infestations | Adrabetadex | 1 | 2 | |
| other | Hypoacusis | Ear and labyrinth disorders | Adrabetadex | 2 | 2 | |
| other | Asthenia | General disorders | Adrabetadex | 1 | 2 | |
| other | Gelastic seizure | Nervous system disorders | Adrabetadex | 1 | 2 | |
| other | Respiratory tract infection viral | Infections and infestations | Adrabetadex | 1 | 2 | |
| other | Dysphagia | Gastrointestinal disorders | Adrabetadex | 1 | 2 | |
| other | Anal incontinence | Gastrointestinal disorders | Adrabetadex | 1 | 2 | |
| other | Ataxia | Nervous system disorders | Adrabetadex | 1 | 2 | |
| other | Disturbance in attention | Nervous system disorders | Adrabetadex | 1 | 2 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Adrabetadex | 0 | 2 |
| Total, other adverse events | Adrabetadex | 2 | 2 |
| Total, serious adverse events | Adrabetadex | 0 | 2 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 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/NCT03879655
GET https://conductscience.org/api/trials/v1/NCT03879655/registered