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Gene Therapy

Gene Therapy for Huntington's Disease

Phase 1 & 2

Recruiting

Led By David Cooper, MD, MBA

Research Sponsored by UniQure Biopharma B.V.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

HTT gene expansion testing with the presence of ≥40 CAG repeats

Cohort 3: Early manifest HD as defined by a UHDRS TFC score of ≥ 11 and EITHER a DCL of 4 or a DCL of 3 with either a positive 'Yes' response to UHDRS Question 80 (multidimensional manifest diagnosis on motor, cognitive, behavioral, functional) or DSM5 criteria for cognitive disorder (Movement Disorder Society Task Force criteria)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months (cohorts 1 & 2) and 12 months (cohort 3)

Awards & highlights

Study Summary

This trial is testing a new drug for Huntington's disease. It is designed to establish safety and proof-of-concept.

Who is the study for?

Adults aged 25-65 with early manifest Huntington's Disease (HD), stable on medications for at least 3 months, and not involved in other trials or brain surgeries. They must have specific gene markers, be able to follow the study plan, and use effective birth control if applicable. Exclusions include certain medical conditions, recent major surgery, abnormal lab values, implanted devices in the brain, MRI contraindications, cancer within 5 years (except some skin cancers), or recent COVID-19 infection.Check my eligibility

What is being tested?

The trial is testing AMT-130 in patients with early HD to check its safety and effectiveness. It involves a randomized comparison between two doses of intra-striatal rAAV5-miHTT gene therapy versus imitation (sham) surgery. Participants are assigned randomly to receive either a high or low dose of the treatment.See study design

What are the potential side effects?

While specific side effects aren't listed here as it's an initial human trial for AMT-130, potential risks may include reactions related to surgical procedures like infection or bleeding and any unforeseen effects from the gene therapy itself.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My genetic test shows I have more than 40 CAG repeats in the HTT gene.

Select...

I have early-stage Huntington's disease with specific functional and cognitive scores.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months (cohorts 1 & 2) and 12 months (cohort 3)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months (cohorts 1 & 2) and 12 months (cohort 3)

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months (cohorts 1 & 2) and 12 months (cohort 3) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number and type of Adverse Events (AE)

Secondary outcome measures

Duration of persistence of AMT-130 in the brain

Other outcome measures

CSF Mutant Protein (fM)

CSF/Serum Neurofilament Light Chain (pg/mL)

HDQLIFE Measures

+7 more

Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: Cohort 3Experimental Treatment1 Intervention

Low dose rAAV5-miHTT (6x10^12 gc/subject). High dose rAAV5-miHTT (6x10^13 gc/subject).

Group II: Cohort 2Experimental Treatment1 Intervention

High dose rAAV5-miHTT (6x10^13 gc/subject).

Group III: Cohort 1Experimental Treatment1 Intervention

Low dose rAAV5-miHTT (6x10^12 gc/subject).

Group IV: Cohorts 1, 2Placebo Group1 Intervention

Imitation (sham) surgery

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

UniQure Biopharma B.V.Lead Sponsor

10 Previous Clinical Trials

238 Total Patients Enrolled

David Cooper, MD, MBAPrincipal InvestigatorUniQure Biopharma B.V.

1 Previous Clinical Trials

15 Total Patients Enrolled

Kenechi Ejebe, MDPrincipal InvestigatorUniQure Biopharma B.V.

Media Library

AMT-130 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04120493 — Phase 1 & 2

Huntington's Disease Research Study Groups: Cohort 3, Cohorts 1, 2, Cohort 1, Cohort 2

Huntington's Disease Clinical Trial 2023: AMT-130 Highlights & Side Effects. Trial Name: NCT04120493 — Phase 1 & 2

AMT-130 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04120493 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this experiment open to new participants?

"Affirmative. Clinicaltrials.gov details this medical trial is seeking participants, having been initially made public on 6th September 2019 and most recently edited on 17th October 2022. 44 patients need to be recruited from 13 different research centres."

Answered by AI

Has an age restriction been placed on the participants of this experiment?

"The minimum age of participants for this trial is 25 and the upper limit is 65, as stated in the entry requirements."

Answered by AI

Are there openings for me to join this research endeavor?

"This scientific experiment is accepting enrolments from individuals between 25 and 65 years old that have been diagnosed with Huntington's disease. Currently, 44 persons are needed to complete the trial."

Answered by AI

What is the current enrollment capacity for this research endeavor?

"The sponsor, UniQure Biopharma B.V., necessitates 44 individuals who meet the criteria for participation in order to conduct this trial. The study will be conducted at Johns Hopkins University in Birmingham and UAB in Gainesville both of which are situated within the United States."

Answered by AI

Are there multiple healthcare facilities conducting this clinical experiment in the same state?

"At this time, 13 different sites are participating in the trial. These include Birmingham, Gainesville and Chicago as well as 10 other locations. To reduce travel demands for potential enrollees, it is recommended that they choose a site closest to them."

Answered by AI