Not yet recruitingRare disease

Study on Gene Therapy with AAVrh10 for Infants with Krabbe Disease Undergoing Stem Cell Transplantation

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What is this trial about?

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

This clinical trial is focused on studying Krabbe Disease, a rare genetic disorder that affects the nervous system. The study is testing a new treatment called FBX-101, which is a type of gene therapy. This treatment involves using a special virus, known as an adeno-associated virus serotype rh10, to deliver a healthy version of the GALC gene into the body. The GALC gene is important because it helps break down certain fats in the body, and its malfunction is what leads to Krabbe Disease.

The purpose of this study is to determine if FBX-101 is safe for infants with Krabbe Disease. Participants in the study will receive the treatment through an injection into their veins, which is known as an intravenous injection. This will occur 21 to 60 days after they have undergone a procedure called a hematopoietic stem cell transplantation, which is a standard treatment for Krabbe Disease. The study will monitor the participants for any side effects and assess their development over time.

Throughout the study, researchers will observe the participants to see if they experience any adverse effects from the treatment. They will also evaluate whether the treatment helps improve the participants' ability to sit independently and their overall motor skills. The study aims to compare these outcomes with those of patients who have not received the gene therapy or have only undergone the stem cell transplantation. The study is expected to continue until 2026.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, you will be informed about the trial's purpose, procedures, and potential risks and benefits. Your consent will be required to participate, which means you agree to follow the study's guidelines and procedures.
Step 2
Initial assessment
An initial assessment will be conducted to confirm your eligibility. This includes checking your diagnosis of Krabbe Disease and ensuring you meet the necessary health criteria, such as adequate organ function and age requirements.
Step 3
Stem cell transplantation
You will undergo a procedure called hematopoietic stem cell transplantation (HSCT). This is a standard treatment for Krabbe Disease and involves receiving healthy stem cells to replace damaged ones.
Step 4
Gene therapy administration
Between 21 to 60 days after the stem cell transplant, you will receive the investigational product, FBX-101. This is a gene therapy delivered through an intravenous injection, which means it is administered directly into your bloodstream.
Step 5
Monitoring and follow-up
After receiving the gene therapy, you will be closely monitored for any side effects or adverse reactions. Regular follow-up visits will be scheduled to assess your health and the effectiveness of the treatment. These visits will include various tests and evaluations to track your progress.
Step 6
Long-term evaluation
Your development and motor skills will be evaluated over time, with specific assessments at one and two years after the gene therapy. This will help determine the therapy's impact on your ability to achieve milestones such as independent sitting and overall motor function.

Who can join the trial?

13 criteria

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Diagnosis of infantile Krabbe disease, which means:
- Low levels of an enzyme called Galactocerebrosidase (GALC) in white blood cells.
- check
  At least one of the following:
  - High levels of a substance called psychosine in a blood test, indicating early onset.
  - Imaging or tests like MRI or nerve tests showing signs of Krabbe disease.
  - Two specific changes (mutations) in the GALC gene that suggest early onset.
  - Age between 1 day and 12 months at the time of screening.
  - Eligible for treatment with Hematopoietic Stem Cell Transplant (HSCT)
  - Parents or legal guardian must agree to the child's participation and provide written consent.
  - Parents or legal guardian must be able to follow the study requirements.
  - The child must have proper organ function, which means:
    
    Kidney function: Creatinine levels should be within 1.5 times the normal range for age, and creatinine clearance should be at least 60 mL/min/1.73 m².
    Liver function: Liver enzymes (ALT/AST) should be no more than twice the normal range for age.
    Heart function: Heart's ejection fraction should be more than 50% without signs of high blood pressure in the lungs.
    Lung function: Oxygen levels should be above 95% when breathing room air.
    Blood clotting tests should be within 110% of normal ranges for age.

Who cannot join the trial?
7 criteria

Patients who have not received a stem cell transplant.
Patients who are younger than 21 days or older than 60 days after receiving a stem cell transplant.
Patients with any other serious medical condition that might interfere with the study.
Patients who are unable to follow the study procedures.
Patients who have participated in another clinical trial recently.
Patients who have allergies to any of the ingredients in the investigational product.
Patients who are not able to provide consent or do not have a legal guardian to provide consent on their behalf.

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Verified sites
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Hospital Sant Joan De Deu Barcelona
Esplugues De Llobregat, Spain
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Investigated drugs

AAVrh.10-hGALC is a special type of treatment that uses a virus to deliver a healthy gene into the body. This therapy is designed for patients with a condition called Krabbe disease, which affects the nervous system. The goal of this treatment is to provide the body with the correct gene it needs to function better. In this study, the treatment is given through an intravenous (IV) infusion, which means it is delivered directly into the bloodstream. This is done after the patient has received a stem cell transplant, which is another type of treatment that helps to replace damaged cells in the body. The main aim of using this therapy is to see if it is safe for young patients with Krabbe disease.

Adeno-Associated Virus Serotype Rh10 Containing The Human Galc Gene

What is already known about the treatment

AAVrh.10-hGALC – This medication is administered intravenously, meaning it is delivered directly into the bloodstream through a vein. It is currently being studied in clinical trials for its potential use in treating Krabbe Disease, a rare and serious genetic disorder. The main goal of this treatment is to provide a safe and effective way to deliver a gene therapy that can help manage the disease in infants who have undergone a stem cell transplant. At the molecular level, the medication works by using a viral vector to introduce a healthy version of the GALC gene into the patient's cells, which is crucial for breaking down certain fats in the body. This medication falls under the pharmacological classification of gene therapy.

Investigated diseases

Krabbe Disease – Krabbe Disease is a rare genetic disorder that affects the nervous system. It is caused by a deficiency of the enzyme galactocerebrosidase, which leads to the buildup of certain fats in the brain and other areas of the nervous system. This accumulation damages the protective covering of nerve cells, known as myelin, leading to severe neurological symptoms. The disease typically begins in infancy, with symptoms such as irritability, muscle weakness, feeding difficulties, and developmental delays. As the disease progresses, it can lead to more severe neurological impairments, including loss of vision, hearing, and motor skills. The progression of Krabbe Disease varies, but it generally leads to significant neurological decline.