# Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

> **NCT03887533** · PHASE1,PHASE2 · TERMINATED · sponsor: **Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)** · enrollment: 2 (actual)

## Conditions studied

- Niemann-Pick Disease, Type C1

## Interventions

- **DRUG:** VTS-270

## Key facts

- **NCT ID:** NCT03887533
- **Lead sponsor:** Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- **Sponsor class:** NIH
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2020-01-06
- **Primary completion:** 2021-10-25
- **Final completion:** 2021-10-25
- **Target enrollment:** 2 (ACTUAL)
- **Why stopped:** Enrollment was poor due to COVID-19 pandemic
- **Last updated:** 2022-08-30


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03887533

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03887533, "Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1". Retrieved via AI Analytics 2026-06-24 from https://api.ai-analytics.org/clinical/NCT03887533. Licensed CC0.

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