Pompe Disease Clinical Trials 2024

Pompe Disease Clinical Trials 2024

Pompe Disease research studies recruiting patients in 2024 need your help. Receive premium care & cutting edge treatments by enrolling in pompe disease clinical trials today.

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Pompe Disease Clinical Trials With No Placebo

View 12 pompe disease medical studies that do not have a placebo group.

Frequently Asked Questions

Do I need insurance to participate in a trial?
Almost all clinical trials will cover the cost of the 'trial drug' — so no insurance is required for this. For trials where this trial drug is given alongside an already-approved medication, there may be a cost (which your insurance would normally cover).
Is there any support for travel costs?
Many of the teams running clinical trials will cover the cost of transportation to-and-from their care center.
Will I know what medication I am taking?
This depends on the specific study. If you're worried about receiving a placebo, you can actively filter out these trials using our search.
How long do clinical trials last?
Some trials will only require a single visit, while others will continue until your disease returns. It's fairly common for a trial to last somewhere between 1 and 6 months.
Do you verify all the trials on your website?
All of the trials listed on Power have been formally registered with the US Food and Drug Administration. Beyond this, some trials on Power have been formally 'verified' if the team behind the trial has completed an additional level of verification with our team.
How quickly will I hear back from a clinical trial?
Sadly, this response time can take anywhere from 6 hours to 2 weeks. We're working hard to speed up how quickly you hear back — in general, verified trials respond to patients within a few days.

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.

Note: The previous response went through several iterations but did not provide an improved or more concise answer.

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.