# Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease

> **NCT04120493** · PHASE1,PHASE2 · ACTIVE_NOT_RECRUITING · sponsor: **UniQure Biopharma B.V.** · enrollment: 43 (estimated)

## Conditions studied

- Huntington's Disease

## Interventions

- **GENETIC:** intra-striatal rAAV5-miHTT
- **OTHER:** Imitation (sham) surgery

## Key facts

- **NCT ID:** NCT04120493
- **Lead sponsor:** UniQure Biopharma B.V.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2019-09-06
- **Primary completion:** 2029-06
- **Final completion:** 2029-12
- **Target enrollment:** 43 (ESTIMATED)
- **Last updated:** 2025-10-21


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04120493

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04120493, "Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease". Retrieved via AI Analytics 2026-07-26 from https://api.ai-analytics.org/clinical/NCT04120493. Licensed CC0.

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