# A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

> **NCT04080050** · — · UNKNOWN · sponsor: **REGENXBIO Inc.** · enrollment: 8 (actual)

## Conditions studied

- Homozygous Familial Hypercholesterolemia (HoFH)

## Interventions

- **DRUG:** Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy

## Key facts

- **NCT ID:** NCT04080050
- **Lead sponsor:** REGENXBIO Inc.
- **Sponsor class:** INDUSTRY
- **Phase:** —
- **Study type:** OBSERVATIONAL
- **Status:** UNKNOWN
- **Start date:** 2019-09-30
- **Primary completion:** 2024-09-29
- **Final completion:** 2025-09-29
- **Target enrollment:** 8 (ACTUAL)
- **Last updated:** 2023-03-09


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04080050

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04080050, "A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501". Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/clinical/NCT04080050. Licensed CC0.

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