Pompe Disease Clinical Trials
Here is the most popular medical study for pompe disease
Pompe Disease Clinical Trials With No Placebo
View 12 pompe disease medical studies that do not have a placebo group.
Participants with Late-Onset Pompe Disease for Pompe Disease
This trial is studying Pompe disease, a genetic condition that causes muscle weakness. People with Pompe disease have a faulty gene that produces an enzyme called GAA. Without this enzyme, there is a build-up
Enzyme Replacement Therapy
Cipaglucosidase Alfa + Miglustat for Pompe Disease
This trial will study the safety, how well it works, and side effects of an experimental drug called cipaglucosidase alfa/miglustat in children with a disease called classic infantile-onset Pompe disease.
Frequently Asked Questions
Introduction to pompe disease
What are the top hospitals conducting pompe disease research?
In the quest to find effective treatments for Pompe disease, several leading hospitals have emerged as beacons of hope and innovation. One such institution is Boston Children's Health Physicians in Valhalla, where a current active clinical trial focuses specifically on this rare genetic disorder. This hospital has been actively involved in two trials related to Pompe disease so far, with its first recorded trial taking place as recently as 2021. Similarly committed to advancing treatment options, Seattle Children's Hospital and Regional Medical Center in Seattle also boasts one ongoing clinical trial for Pompe disease. With their dedication evident through two historical trials dating back to 2021.
Meanwhile, at Duke University Medical Center in Durham, researchers are diligently working towards combating this debilitating condition with their own active Pompe disease trial. Building upon a solid foundation of two previous trials conducted since 2021,this facility continues to contribute invaluable insights into the management of this complex illness.
Further along the west coast, experts from the University of California Irvine neurology department have been making strides in unraveling the complexities surrounding Pompe disease. Their current investigation represents yet another active clinical trial for this condition while having laid crucial groundwork through a prior study initiated way back during 2020.Moreover ,the renowned Stanford University situated amidst Palo Alto has embarked on its own journey into combatting Pompe Disease by launching an active,cutting-edge research mission.Additionally touting just one previously concluded experiment throughout twenty-twenty emphasising it’s dedication and drive .
These top-tier institutions collectively epitomize unwavering commitment towards understanding and treating Pompe disease—a serious genetic disorder that affects muscle function due to an enzyme deficiency—and serve as vital hubs where groundbreaking discoveries can alter patients' lives forever.
Which are the best cities for pompe disease clinical trials?
When it comes to clinical trials for Pompe disease, several cities have emerged as leaders in research and development. Durham, North carolina boasts 3 active trials focusing on treatments like Avalglucosidase alfa, ACTUS-101, and Alglucosidase alfa (GZ419829). Valhalla, New york and Seattle, Washington follow closely behind with 2 ongoing studies exploring similar treatment options. Additionally, Orange, California and Atlanta, Georgia also have 2 active trials investigating therapies such as AT845 and SPK-3006. These cities offer individuals with Pompe disease access to cutting-edge clinical trials that hold the promise of advancing treatment options and improving patient outcomes.
Which are the top treatments for pompe disease being explored in clinical trials?
Pompe disease is a rare genetic disorder, and ongoing clinical trials are paving the way for potential breakthrough treatments. AT845, an innovative treatment option, is currently being explored in one active trial. Another promising candidate is SPK-3006, which also has one active trial dedicated to Pompe disease research. ACTUS-101 joins the list of top treatments with its own active trial focused on combating this debilitating condition. Finally, there's alglucosidase alfa (GZ419829), first listed in 2016 and involved in two all-time Pompe disease trials. These cutting-edge therapies offer hope for patients as researchers strive to find effective solutions for Pompe disease.
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What are the most recent clinical trials for pompe disease?
In the realm of Pompe disease research, recent clinical trials have offered promising prospects for patients. One notable trial involves avalglucosidase alfa, a treatment currently in Phase 3. Expectedly available since September 2021, this study brings hope for improved outcomes and enhanced quality of life. Additionally, AT845 has demonstrated potential as a therapeutic option through its participation in both Phase 1 and Phase 2 trials starting from October 2020. Similarly, SPK-3006's involvement in combined Phase 1 and Phase 2 studies beginning on October 2020 offers further optimism regarding future advancements for individuals with Pompe disease. These ongoing efforts form an essential part of the journey towards more effective treatments and management strategies for this rare genetic disorder.
What pompe disease clinical trials were recently completed?
Recently completed clinical trials for Pompe disease have shown promising advancements in the search for effective treatments. In October 2017, the University of Florida concluded a trial investigating Rituxan's potential in addressing this debilitating condition. Another notable trial was completed in November 2016, sponsored by Genzyme, a Sanofi Company. This study explored the use of Avalglucosidase alfa (GZ402666) as a therapeutic option for Pompe disease. These significant milestones offer hope to individuals affected by Pompe disease and highlight ongoing efforts to find solutions that can improve their quality of life.