Krabbe’s disease – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 10:28:52 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Krabbe’s disease – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on Gene Therapy with AAVrh10 for Infants with Krabbe Disease Undergoing Stem Cell Transplantation https://clinicaltrials.eu/trial/study-on-gene-therapy-with-aavrh10-for-infants-with-krabbe-disease-undergoing-stem-cell-transplantation/ Wed, 29 Apr 2026 14:23:41 +0000 https://clinicaltrials.eu/trial/study-on-gene-therapy-with-aavrh10-for-infants-with-krabbe-disease-undergoing-stem-cell-transplantation/ 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.

]]>