Fabry Disease Clinical Trials 2024

Fabry Disease Clinical Trials 2024

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

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Phase 3 Fabry Disease Clinical Trials

View 14 phase 3 fabry disease medical studies.

Fabry Disease Clinical Trials With No Placebo

View 14 fabry 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 fabry disease

What are the top hospitals conducting fabry disease research?

Fabry disease, a rare genetic disorder, has caught the attention of top hospitals across the United States. In Atlanta, the Emory University School of Medicine leads the way with three active clinical trials focused on Fabry disease and a total of nine trials conducted over time. This institution has been committed to investigating this condition since they recorded their pioneering trial in 2001. Meanwhile, in Grand Rapids at Infusion Associates, researchers are actively working on three Fabry disease trials and have completed eight studies thus far since embarking on their first trial in 2016.

Emory University itself also contributes to the advancement of knowledge surrounding Fabry disease with two ongoing clinical trials and a history of three previous investigations since their initial study began in 2007. Notably, Salt Lake City's University of Utah is making strides as well, currently engaging in two active clinical trials for Fabry disease after recording its first trial only recently in 2018 while completing four studies overall within this field.

Adding another important perspective is the University of South Florida located in Tampa which presently conducts two active fabry-related examinations contributing towards treatment options formation; similarly joining research efforts just couple yearsback from now like that esteemed institute before them also accomplished conducting exactly same number (3)of past work specific to said condition.

These dedicated hospitals showcase how medical professionals across different locations are united by a shared commitment to unraveling the complexities surrounding Fabry disease. Their collaborative efforts offer hope for individuals affected by this rare disorder and illuminate potential breakthroughs that can improve lives worldwide

Which are the best cities for fabry disease clinical trials?

When it comes to fabry disease clinical trials, several cities are leading the way in research and development. Fairfax, Virginia has 11 active trials exploring treatments like Venglustat (GZ402671), migalastat HCl 150 mg, and pegunigalsidase alfa. Dallas, Texas closely follows with 10 ongoing studies focusing on pegunigalsidase alfa, Venglustat (GZ402671), and Lucerastat. Atlanta, Georgia also plays a significant role with 8 active trials examining migalastat HCl 150 mg, pegunigalsidase alfa, and Venglustat. Additionally, Birmingham in Alabama and Salt Lake City in Utah each have 5 active trials investigating various treatment options for fabry disease such as pegunigalsidase alfa and Venglustat (GZ402671). These cities offer individuals affected by fabry disease access to cutting-edge clinical trials that contribute to advancements in care for this condition.

Which are the top treatments for fabry disease being explored in clinical trials?

Fabry disease research is advancing rapidly, with several promising treatments being explored in clinical trials. Leading the way is migalastat HCl 150 mg, currently involved in two active trials for fabry disease. Since its introduction in 2015, it has been a part of three all-time fabry disease trials. Another contender making waves is pegunigalsidase alfa, also engaged in two ongoing trials and first listed in 2018. Additionally, Venglustat (GZ402671) has captured attention with one active trial and two all-time fabry disease trials since its recent debut in 2022. Rounding out this list is the intriguing prospect of 4D-310—a newcomer to the field—with one active trial and two all-time fabry disease trials since entering the scene in 2020. These innovative treatments hold promise for improving outcomes for patients living with fabry disease worldwide.

What are the most recent clinical trials for fabry disease?

Exciting advancements have been made in recent clinical trials for individuals with Fabry disease, offering hope for improved treatment options. One noteworthy trial involves the investigation of migalastat HCl 150 mg, a potential therapeutic intervention in Phase 3 development. Additionally, Venglustat shows promise as another Phase 3 candidate that could potentially address the needs of those affected by this rare genetic disorder. Notably, both these treatments hold significant potential to improve patient outcomes and enhance their quality of life. With ongoing research and developments like these, there is optimism within the medical community about finding effective solutions for Fabry disease in the near future.

What fabry disease clinical trials were recently completed?

Recently, several clinical trials focusing on Fabry disease have concluded, demonstrating progress in the field of treatment. Notably, a trial sponsored by Avrobio was completed in October 2021 and showed promising results. Another significant milestone was reached with the completion of a study for QR-010 conducted by ProQR Therapeutics in September 2021. These important advancements highlight the ongoing efforts to develop effective therapies for Fabry disease and provide hope for individuals affected by this rare genetic disorder.