Gene Therapy for Huntington's Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests AMT-130, a one-time gene therapy, in patients with early-stage Huntington's Disease. The treatment aims to lower a harmful brain protein to slow down the disease's progression. AMT-130 has shown promise in early research.
Will I have to stop taking my current medications?
The trial requires that all medications for Huntington's disease symptoms must be stable for 3 months before joining the study, meaning you should not change your current medications during this time.
Will I have to stop taking my current medications?
The trial requires that all medications for Huntington's disease symptoms must be stable for 3 months before joining the study, meaning you should not change your current medications during this time.
Is gene therapy for Huntington's disease safe for humans?
Research on gene therapy for Huntington's disease, specifically using AAV5-miHTT, shows it is generally safe and well tolerated in animal studies, including non-human primates and rodents. Additionally, a related therapy, RG6042, was found to be safe in a small human trial, suggesting a positive safety profile for these types of treatments.12345
Is gene therapy for Huntington's Disease generally safe in humans?
Research on gene therapy for Huntington's Disease, specifically using AAV5-miHTT, shows it is generally safe and well-tolerated in animal studies, with no significant safety concerns observed. Additionally, a related therapy, RG6042, was found to be generally safe in a small human study, suggesting a positive safety profile for these types of treatments.12345
How is the treatment AMT-130 for Huntington's Disease different from other treatments?
How is the treatment AMT-130 for Huntington's Disease different from other treatments?
What data supports the effectiveness of the treatment AMT-130 for Huntington's Disease?
What data supports the effectiveness of the treatment AMT-130 for Huntington's Disease?
The research highlights that new therapies for Huntington's Disease are being developed to target the disease at its origin, such as lowering the levels of the mutant huntingtin protein. While specific data on AMT-130 is not provided, similar approaches like RNA interference therapies are being explored in clinical trials, suggesting a promising direction for treatments like AMT-130.1112131415
Who Is on the Research Team?
David Margolin, MD, PhD
Principal Investigator
UniQure Biopharma B.V.
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either high or low dose AMT-130 with pre and post-operative immunosuppressant therapies
Follow-up
Participants are monitored for safety and effectiveness after treatment
Optional Extended Follow-Up
Cohort 2 Sham participants who do not cross over to receive AMT-130 treatment will be followed for an additional 2 years
What Are the Treatments Tested in This Trial?
Interventions
- AMT-130
- Imitation (sham) surgery
AMT-130 is already approved in United States, European Union for the following indications:
- None approved; under investigation for Huntington's disease
- None approved; under investigation for Huntington's disease
Find a Clinic Near You
Who Is Running the Clinical Trial?
UniQure Biopharma B.V.
Lead Sponsor