37 Participants Needed

Gene Therapy for Huntington's Disease

Recruiting at 13 trial locations
DL
LR
EE
uu
Overseen ByuniQure uniQure
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: UniQure Biopharma B.V.
Must be taking: Immunosuppressants
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 2 JurisdictionsThis treatment is already approved in other countries

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?

AMT-130 is a gene therapy that targets the root cause of Huntington's Disease by delivering genetic material directly to the brain to reduce the production of the harmful huntingtin protein, which is unique compared to traditional treatments that mainly address symptoms.678910

How is the treatment AMT-130 for Huntington's Disease different from other treatments?

AMT-130 is a gene therapy that targets the root cause of Huntington's Disease by delivering genetic material to modify the expression of the huntingtin gene, which is not addressed by traditional treatments that mainly focus on managing symptoms.678910

What data supports the effectiveness of the treatment AMT-130 for Huntington's Disease?

The research highlights that new therapies for Huntington's Disease, including those targeting the disease at its genetic origin, are in clinical trials. These therapies aim to lower levels of the mutant huntingtin protein, which is a promising approach for treating the disease.1112131415

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?

DM

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

I am between 25 and 65 years old.
Your brain MRI must show that certain areas called the putamen and caudate are a certain size.
My medications for HD symptoms have been stable for 3 months.
See 9 more

Exclusion Criteria

Your blood tests show: a. High levels of alanine aminotransferase (ALT) or aspartate aminotransferase (AST) b. High levels of total bilirubin or alkaline phosphatase (ALP) c. High levels of creatinine d. Low platelet count e. Abnormal prothrombin time (PT) or partial thromboplastin time (PTT)
I am not allowed to have a lumbar puncture due to medical reasons.
I haven't had cancer in the last 5 years, except for certain skin cancers or cervical cancer that was treated.
See 11 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either high or low dose AMT-130 with pre and post-operative immunosuppressant therapies

12 months
Monthly visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

48 months
Periodic follow-up visits

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

24 months

What Are the Treatments Tested in This Trial?

Interventions

  • AMT-130
  • Imitation (sham) surgery
Trial Overview 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.
How Is the Trial Designed?
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

AMT-130 is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as AMT-130 for:
  • None approved; under investigation for Huntington's disease
🇪🇺
Approved in European Union as AMT-130 for:
  • 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

Trials
12
Recruited
260+

Published Research Related to This Trial

There are several promising disease-modifying therapies for Huntington's disease (HD) currently in human clinical trials, including gene therapies and RNA modulation, which aim to address the underlying causes of the disease rather than just its symptoms.
The pathogenesis of HD involves complex toxic pathways stemming from the mutant huntingtin gene, highlighting the need for innovative treatments that target these mechanisms to potentially alter the disease's progression.
Huntington's Disease: New Frontiers in Therapeutics.Pan, L., Feigin, A.[2021]
Huntington's disease (HD) currently has no cure, but various medications and non-drug treatments can help manage symptoms like chorea and cognitive decline, with new therapies targeting the underlying cause of the disease in clinical trials.
Emerging treatments, particularly antisense oligonucleotide (ASO) therapies, aim to lower levels of the harmful mutant huntingtin protein (mHTT), and other innovative approaches like RNA interference and CRISPR are also being explored for their potential to treat HD.
Current and Possible Future Therapeutic Options for Huntington's Disease.Ferguson, MW., Kennedy, CJ., Palpagama, TH., et al.[2022]
New therapeutic approaches for Huntington's disease aim to directly target the HTT gene mutation, with methods like CRISPR-Cas9 and zinc-finger transcription repressors potentially preventing the disease's harmful effects at the DNA level.
Antisense oligonucleotides (ASOs) have been shown to safely reduce levels of the mutant huntingtin protein in patients' brains, and they are currently in late-stage clinical trials, indicating promising progress in modifying the disease's course.
Therapeutic strategies for Huntington's disease.Estevez-Fraga, C., Flower, MD., Tabrizi, SJ.[2021]

Citations

Huntington's Disease: New Frontiers in Therapeutics. [2021]
Current and Possible Future Therapeutic Options for Huntington's Disease. [2022]
Therapeutic strategies for Huntington's disease. [2021]
Therapeutic Strategies in Huntington's Disease. [2021]
Clinical trials in Huntington's disease: Interventions in early clinical development and newer methodological approaches. [2022]
Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology. [2021]
Huntingtin-Lowering Therapies for Huntington Disease: A Review of the Evidence of Potential Benefits and Risks. [2021]
AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease. [2020]
Inducing huntingtin inclusion formation in primary neuronal cell culture and in vivo by high-capacity adenoviral vectors expressing truncated and full-length huntingtin with polyglutamine expansion. [2016]
10.United Statespubmed.ncbi.nlm.nih.gov
Amantadine in the akinetic-rigid variant of Huntington's disease. [2013]
Adoptive cellular gene therapy of autoimmune disease. [2019]
Local delivery of cytokines by retrovirally transduced antigen-specific TCR+ hybridoma cells in experimental autoimmune encephalomyelitis. [2012]
13.United Statespubmed.ncbi.nlm.nih.gov
Adoptive immunotherapy of experimental autoimmune encephalomyelitis via T cell delivery of the IL-12 p40 subunit. [2019]
14.United Statespubmed.ncbi.nlm.nih.gov
Synthetic Cell-Based Immunotherapies for Neurologic Diseases. [2023]
[Construction of TRAIL gene eukaryotic expression vector modulated by hTERT gene core promoter and its effect on apoptosis of ovarian cancer cells]. [2012]
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