# Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects

> **NCT00891306** · PHASE2,PHASE3 · COMPLETED · sponsor: **Amsterdam Molecular Therapeutics** · enrollment: 5 (actual)

## Conditions studied

- Familial Lipoprotein Lipase Deficiency

## Interventions

- **GENETIC:** Alipogene Tiparvovec (AMT-011), Human LPL [S447X]
- **DRUG:** mycophenolate mofetil
- **DRUG:** cyclosporine
- **DRUG:** methylprednisolone

## Key facts

- **NCT ID:** NCT00891306
- **Lead sponsor:** Amsterdam Molecular Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2,PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2009-02
- **Primary completion:** 2010-08
- **Final completion:** 2011-04
- **Target enrollment:** 5 (ACTUAL)
- **Last updated:** 2011-09-29

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT00891306

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT00891306, "Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects". Retrieved via AI Analytics 2026-05-29 from https://api.ai-analytics.org/clinical/NCT00891306. Licensed CC0.

---

*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*