Krabbe Disease Clinical Trials
Here are the 0 most popular medical studies for krabbe disease
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Krabbe Disease Clinical Trials With No Placebo
View 8 krabbe disease medical studies that do not have a placebo group.
Gene Therapy
Gene Therapy with FBX-101 for Krabbe Disease
This trial will test a new gene therapy for Krabbe disease. People with Krabbe disease will receive standard care for their disease, followed by a single infusion of the gene therapy. There will be a control group of people who have Krabbe disease who will not receive the gene therapy.
Stem Cell Therapy for Inherited Metabolic Brain Diseases
This trial is testing a new treatment for inborn errors of metabolism that includes transplanting stem cells from umbilical cords and injecting them into the spinal cord. The goal is to see if this is a safe and effective treatment for early demyelinating disease in the central nervous system.
Frequently Asked Questions
Introduction to krabbe disease
What are the top hospitals conducting krabbe disease research?
While rare diseases often face significant challenges in terms of research and treatment, there are hospitals across the United States that are actively working towards finding solutions for conditions like Krabbe disease. Massachusetts General Hospital in Boston is leading the charge with two ongoing clinical trials dedicated to this devastating disorder. Although they have not conducted any previous Krabbe disease trials, their commitment to exploring potential treatments is commendable. Not far away in Orange, California, the Children's Hospital of Orange County (CHOC) is also making strides with two active Krabbe disease trials. Having already completed two trials since recording their first one in 2021, CHOC demonstrates a promising dedication to unraveling the mysteries of this condition.
On the West Coast at Stanford University Medical Center in Palo Alto, researchers are focusing on one active clinical trial for Krabbe disease. While they don't have a history of conducting past trials for this specific condition yet, their involvement and expertise contribute significantly to advancing knowledge and potential therapies in neurological disorders such as Krabbe disease.
In Florida City, the University of Florida Board joins the efforts by currently running one clinical trial addressing Krabbe disease. Though they have no prior experience conducting similar studies before now; however it worthy knowing more medical minds pooling resources into understanding such complexities offer hope
Lastly but certainly not least Kennedy Krieger Institute located within Baltimore has joined this group by initiating one current krabbespecific clinical test while having accomplished another investigation starting back from 2016.Their contribution signifies how every step taken brings us closer to improving lives through advancement against virtually indomitable health battles
These hospitals serve as beacons of hope for both patients affected by Krabbe disease and their families who tirelessly seek answers and better outcomes. Despite facing unique challenges due to its rarity; these institutions' unwavering dedication showcases that progress can be made when experts come together with compassion perseverance
Which are the best cities for krabbe disease clinical trials?
When it comes to clinical trials for Krabbe disease, several cities stand out as leaders in research and development. Salt Lake City, Utah is at the forefront with 3 active trials focusing on Leriglitazone, PBKR03, SHP611, and other treatment options. Boston, Massachusetts follows closely behind with 2 ongoing studies exploring Leriglitazone and Pramipexole among other potential interventions. Minnesota, Minnesota also offers 2 active trials centered around Leriglitazone and additional treatments. Additionally, Orange, California and Chicago, Illinois both have 2 active trials each investigating various therapeutic approaches like High Dose FBX-101 (aka AAVrh.10-GALC), FBX-101,PBKR03 ,SHP611,and others. These cities provide individuals affected by Krabbe disease access to cutting-edge clinical trials that drive progress in understanding the condition and developing effective treatment strategies.
Which are the top treatments for krabbe disease being explored in clinical trials?
Exciting developments are taking place in the realm of krabbe disease research, with two standout treatments currently being explored in clinical trials. Leading the charge is PBKR03, a promising option under investigation in one active trial for krabbe disease. With its recent debut in 2022, it marks an important milestone on the path towards finding effective therapies. Equally noteworthy is FBX-101, which has garnered attention with one active trial and two all-time krabbe disease trials since its introduction in 2021. As researchers delve deeper into these potential breakthroughs, there is renewed hope that innovative treatments will soon emerge to combat this challenging condition.
What are the most recent clinical trials for krabbe disease?
Exciting advancements are occurring in clinical trials for krabbe disease, offering hope to patients and their families. One such trial involves the use of leriglitazone, which is currently in Phase 3. Another promising study examines the potential benefits of pramipexole, now in Phase 4. Additionally, a trial involving high-dose FBX-101 (aka AAVrh.10-GALC) is progressing through both Phase 1 and Phase 2 stages. Lastly, PBKR03 and FBX-101 have also shown promise during their respective joint Phases 1 and 2 studies conducted earlier this year.
What krabbe disease clinical trials were recently completed?
Recently completed clinical trials have made significant strides in advancing the treatment of Krabbe disease. These groundbreaking efforts include a trial sponsored by The Children's Hospital of Philadelphia, which concluded in September 2021, and focused on exploring potential therapies for this debilitating condition. Additionally, another noteworthy trial led by the National Institute of Neurological Disorders and Stroke reached completion in July 2021, further contributing to our understanding and management of Krabbe disease. These recent achievements highlight the commitment and progress being made within the medical community to improve outcomes for individuals affected by this rare genetic disorder.