Not recruitingRare disease

Safety and Tolerability Study of AB-1001 Brain Injection in Adults with Early Huntington's Disease

Registered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on patients with Huntington's Disease (HD), a genetic brain disorder that causes progressive breakdown of nerve cells. The study will test a new investigational treatment called AB-1001, which is administered directly into specific areas of the brain (the caudate nucleus and putamen). This treatment uses a modified virus to deliver a therapeutic gene and is given as a one-time injection into both sides of the brain.

The purpose of this study is to evaluate the safety and tolerability of AB-1001 in adults with early manifest Huntington's Disease and to determine the appropriate dose for future research. During the study, participants will receive several types of brain scans including MRI scans with gadoteric acid (a contrast agent), PET scans using fludeoxyglucose, and other medical assessments. Some participants may receive methylprednisolone, a corticosteroid medication, as part of their treatment.

The study will monitor participants for 52 weeks after receiving the treatment. Throughout this period, various tests will be performed to check brain structure and function, measure disease markers in blood and spinal fluid, and assess changes in disease symptoms and daily functioning. These assessments will help determine how well the treatment is working and identify any potential side effects.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial brain imaging
You will undergo a magnetic resonance imaging (MRI) scan to measure your brain structure.
The scan will focus on specific brain regions: the caudate and putamen, which are important areas affected by Huntington's disease.
Step 2
Pre-treatment assessments
You will complete several tests to measure your movement abilities and thinking skills.
Blood and spinal fluid samples will be collected to measure specific markers related to Huntington's disease.
Additional brain scans will be performed: MR Spectroscopy to measure brain chemistry and PET scan using a special tracer called fludeoxyglucose to measure brain activity.
Step 3
Treatment procedure
The treatment involves a one-time surgical procedure to deliver the medication AB-1001 into both sides of your brain.
The medication will be injected into two specific areas: the caudate nucleus and putamen.
You will receive gadoteric acid during the procedure for imaging purposes.
You will also receive methylprednisolone by injection to help manage potential inflammation.
Step 4
Follow-up period
Regular check-ups will occur over 52 weeks to monitor your health and any side effects.
At week 52, you will repeat the brain scans (MRI, MR Spectroscopy, and PET scan).
Additional blood and spinal fluid samples will be collected.
Your movement abilities and thinking skills will be reassessed using the same tests as before treatment.

Who can join the trial?

8 criteria

Age between 18 and 65 years old, able to give informed consent and follow study procedures
Must have genetic test confirmation showing CAG expansion of 40 or more in the huntingtin gene (this is a specific genetic marker associated with the condition)
Must be in early stages of the condition with:
- A Total Functional Capacity (TFC) score between 9 and 13 (this measures independence in daily activities)
- A Diagnostic Classification Level (DCL) of 4, or DCL of 3 with cognitive problems and clear disease progression
- Brain scan (MRI) must show minimum required size of specific brain regions:
  - Putamen: at least 2.3 cubic centimeters on each side
  - Caudate: at least 1.7 cubic centimeters on each side
  - Any current medications for the condition must be stable for at least 30 days before screening
  - Both men and women can participate

Who cannot join the trial?

14 criteria

History of any brain surgery, including deep brain stimulation or prior gene therapy
Active psychiatric conditions that could interfere with the study procedures
Pregnant or breastfeeding women
Presence of other neurological conditions besides Huntington's disease
Current participation in other clinical trials or use of investigational drugs within 30 days
Severe medical conditions affecting the heart, liver, or kidneys
History of substance abuse within the past year
Unable to undergo MRI scanning (magnetic resonance imaging - a medical imaging technique)
Blood clotting disorders or use of blood thinning medications
Known allergies to components of the study treatment
Significant changes in medications within the past 3 months
Inability to comply with study procedures or follow-up visits
Active infections or compromised immune system
Previous adverse reactions to similar treatments or procedures

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Investigated drugs

AB-1001 is an investigational therapy that is administered directly into specific regions of the brain (the caudate nucleus and putamen). This is a one-time treatment being studied for people with early-stage Huntington's Disease. The therapy is delivered through a surgical procedure that targets these specific areas of the brain. The purpose is to evaluate how safe and well-tolerated this treatment is in patients with Huntington's Disease.

What is already known about the treatment

AB-1001 - A novel investigational therapeutic agent being studied for the treatment of Huntington's disease through direct administration into the brain (specifically the caudate nucleus and putamen). This experimental medication is currently undergoing Phase I/II clinical trials to evaluate its safety, tolerability, and preliminary effectiveness in patients with early manifest Huntington's disease. The treatment involves a one-time bilateral administration through a specialized surgical procedure targeting specific regions of the brain, representing a potential therapeutic approach for this genetic neurodegenerative disorder. While the exact molecular mechanism is still under investigation in clinical trials, this treatment is being developed as a targeted therapy for addressing the underlying pathology of Huntington's disease.

Investigated diseases

Huntington's disease - A genetic disorder that causes progressive degeneration of nerve cells in the brain. The disease is caused by an inherited defect in a single gene that causes damage to both brain and body. Early signs include personality changes, mood swings, and problems with coordination. As the disease progresses, it leads to difficulty with movement (chorea), cognitive decline, and psychiatric symptoms. The condition typically appears in adults between ages 30 and 50, though it can develop earlier or later in life. The disease affects various regions of the brain, particularly the striatum (which includes the caudate nucleus and putamen).