In short
Currently, there is 1 ongoing clinical trial for Krabbe's disease, a rare genetic disorder affecting the nervous system. This trial is investigating gene therapy combined with stem cell transplantation for infants with this condition. (Also known as: Krabbe Disease, Globoid Cell Leukodystrophy)
Clinical trial locations
Study on Gene Therapy with AAVrh10 for Infants with Krabbe Disease Undergoing Stem Cell Transplantation
This clinical trial is testing a new gene therapy treatment called FBX-101 for infants diagnosed with Krabbe's disease. The condition occurs when the body cannot produce enough of an enzyme called galactocerebrosidase, which normally helps break down certain fats in the body. When these fats build up, they damage the protective covering of nerve cells, leading to serious neurological problems.
Main inclusion criteria:
- Confirmed diagnosis of infantile Krabbe's disease with low levels of the GALC enzyme in white blood cells
- Evidence of the condition through elevated psychosine levels in blood tests, imaging results showing signs of the disease, or genetic testing showing two mutations in the GALC gene
- Age between 1 day and 12 months at screening
- Eligible to receive hematopoietic stem cell transplantation
Main exclusion criteria:
- Patients who have not received a stem cell transplant or who are receiving the gene therapy outside the 21 to 60 day window after transplantation
- Presence of other serious medical conditions that could interfere with the study
- Recent participation in another clinical trial
- Allergies to any components of the investigational product
- Inability to follow study procedures or lack of legal guardian consent
Focus and goal:
The primary focus of this trial is to determine whether FBX-101 is safe for infants with Krabbe's disease. The treatment uses a special virus called adeno-associated virus serotype rh10 to deliver a healthy version of the GALC gene into the body. Participants receive this gene therapy through an intravenous injection between 21 and 60 days after undergoing a standard stem cell transplantation procedure.
Researchers will carefully monitor participants for any side effects or adverse reactions. They will also evaluate whether the treatment helps improve the children's ability to sit independently and their overall motor skills development. These outcomes will be compared with those of patients who received only stem cell transplantation without gene therapy. Long-term evaluations will continue at one and two years after treatment to assess the therapy's impact on developmental milestones and motor function.
Investigational drug:
The study uses AAVrh.10-hGALC, a gene therapy that employs a viral vector to introduce a healthy copy of the GALC gene into the patient's cells. This therapy is administered intravenously, meaning it is delivered directly into the bloodstream. The treatment aims to provide the body with the correct genetic information needed to produce the missing enzyme, potentially helping to manage the progression of the disease. The study is expected to continue until 2026.
Summary
There is currently one clinical trial available for Krabbe's disease, taking place in Spain. This trial represents an innovative approach to treating this rare and serious genetic disorder by combining gene therapy with the existing standard treatment of stem cell transplantation. The focus on infants reflects the early-onset nature of this condition and the critical importance of early intervention.
The trial is investigating AAVrh.10-hGALC, a gene therapy designed to address the underlying genetic cause of the disease by delivering a functional copy of the GALC gene. This approach differs from traditional treatments by attempting to provide a long-term solution rather than just managing symptoms. Families affected by Krabbe's disease who meet the eligibility criteria may wish to discuss this trial with their healthcare providers to determine if participation might be appropriate for their child.
