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NCT04120493 · ClinicalTrials.gov registry record · Phase 1

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

A Phase 1 study of Huntington's Disease, sponsored by UniQure Biopharma B.V..

Active
Registry status
Phase 1
Development phase
43
Enrollment target
12
Study locations

NCT04120493 is a Phase 1 study of Huntington's Disease that is active but no longer recruiting, run by UniQure Biopharma B.V.. The registered enrollment target is 43 participants, below the 4,788-participant average among 8 other Huntington's Disease trials with a reported enrollment target (99% lower). The trial reports 12 study locations across 12 states.

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The verdict

NCT04120493, a Phase 1 study of Huntington's Disease, is active but no longer recruiting, sponsored by UniQure Biopharma B.V..

ACTIVE NOT RECRUITING
Registry status
Phase 1
Development phase
43 participants
Enrollment target
12
Study locations

Study Summary

This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase 1/2, multicenter, first-in-human (FIH) study. The first three cohorts of the study have completed enrollment, including the randomized, double-blind, sham-controlled cohorts. Cohort 4 is open-label. Cohort 4 participants will receive high dose AMT-130.

Conditions Studied

Huntington's Disease

Interventions

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

Study Locations (12)

Alabama

  • University of Alabama at Birmingham - Birmingham

Arizona

  • University of Arizona (Surgical Site Only) - Tucson

California

  • University of California, San Francisco - San Francisco

Colorado

  • CenExel Rocky Mountain Clinical Research - Englewood

Illinois

  • Rush University Medical Center - Chicago

Maryland

  • Johns Hopkins University - Baltimore

Michigan

  • University of Michigan Department of Neurology - Ann Arbor

Ohio

  • Ohio State University - Columbus

Trial Details

FieldValue
Enrollment Target 43 participants
Start Date 2019-09-06
Est. Completion 2029-12
Phase Phase 1

Sponsor

UniQure Biopharma B.V.

4 total trials

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What the Registry Record Tells You About NCT04120493

The ClinicalTrials.gov registry entry for NCT04120493 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 43 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 4,788-participant average among 8 other Huntington's Disease trials with a reported enrollment target (99% lower). The listed sponsor is UniQure Biopharma B.V., which has 4 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Huntington's Disease appearing as the primary indexed condition, and to 2 interventions - of which intra-striatal rAAV5-miHTT is the first listed.

NCT04120493 reports 12 study locations spanning 12 distinct geographic areas - top geographies include Alabama, Arizona, California.

Frequently Asked Questions

What is clinical trial NCT04120493 about?

NCT04120493 is a clinical study titled "Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease". This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase 1/2, multicenter, first-in-human (FIH) study. The first three cohorts of the study have completed enrollment, including the randomized, doubl...

What is the current status of trial NCT04120493?

This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 43 participants. The study started on 2019-09-06. Estimated completion is 2029-12.

What conditions does trial NCT04120493 study?

This clinical trial studies the following conditions: Huntington's Disease.

What interventions are being tested in trial NCT04120493?

The interventions under investigation include: intra-striatal rAAV5-miHTT (GENETIC), Imitation (sham) surgery (OTHER).

Who is sponsoring clinical trial NCT04120493?

This trial is sponsored by UniQure Biopharma B.V., which has 4 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT04120493 being conducted?

This trial has 12 study locations across Alabama, Arizona, California, Colorado, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.