Adrenoleukodystrophy Clinical Trials
Here are the 0 most popular medical studies for adrenoleukodystrophy
ALD Clinical Trials
View 9 ALD medical studies.
Stem Cell Therapy
Mesenchymal Stem Cells for Adrenoleukodystrophy
This trial evaluates 3rd party stem cells as a bridge to stem cell transplant or gene therapy for cerebral adrenoleukodystrophy (CALD), between diagnosis and transplant (currently 8-12 weeks).
Gene Therapy for Adrenomyeloneuropathy (AMN)
This trial is evaluating a gene therapy to treat adrenomyeloneuropathy, a hereditary nervous system disorder. Patients will receive a single dose of the treatment or a fake treatment, and be closely monitored for safety and efficacy for 2 years.
Pramipexole for Restless Legs Syndrome in Women with X-linked Adrenoleukodystrophy
This trial will study whether a medication called pramipexole can help reduce symptoms of restless leg syndrome (RLS) in women with a rare disorder called X-linked adrenoleukodystrophy (ALD).
Hematopoietic Stem Cell Transplantation
Blood and Marrow Transplant for Blood Diseases
This trial is for people with high-risk non-malignant diseases who don't have a suitable HLA-matched sibling donor, using a reduced intensity conditioning T-cell replete haploidentical donor.
Frequently Asked Questions
Introduction to adrenoleukodystrophy
What are the top hospitals conducting adrenoleukodystrophy research?
When it comes to advancing the understanding and treatment of adrenoleukodystrophy, several top hospitals are leading the way. In Minneapolis, the Masonic Cancer Center at the University of Minnesota is making strides with two active clinical trials focused on this rare genetic disorder. Since their first recorded trial in 1995, they have conducted a total of six adrenoleukodystrophy studies, demonstrating their commitment to tackling this challenging condition. Similarly, Massachusettsf Minnesonic Cancer Center at the University of Minnesota is making strides with two active clinical trials focused on this rare genetic disorder. Since their first recorded trial in 1995, they have conducted a total of six adrenoleukodystrophy studies, demonstrating their commitment to tackling this challenging condition. Similarly, Massachusetts General Hospital in Boston is dedicating resources to combatting adrenoleukodystrophy with two ongoing trials and four completed ones since embarking on this research journey back in 2017.
On the West Coast, Stanford University Medical Center in Palo Alto has also joined the fight against adrenoleukodystrophy with one active trial currently underway. Their first recorded trial for this condition took place just last year in 2023 but reflects their dedication towards understanding and finding solutions for patients affected by this disease. Moreover, another institution contributing its efforts towards addressing adrenoleukodystrophy is the Kennedy Krieger Institute located in Baltimore where they have one active clinical trial dedicated to furthering our knowledge about this disorder alongside five previously held investigations since recording their initial trial experience back in 1998.
While these numbers may seem small compared to other conditions or institutions conducting research on more common diseases, it's important to recognize that any progress made in understanding and treating rare disorders like adrenoleukodystrophy brings us closer to improved outcomes for those affected by it. These hospitals represent beacons of hope as they work tirelessly towards unraveling mysteries surrounding adrenoleukodystrophy and ultimately aim to offer better care options for patients worldwide facing similar challenges.
Which are the best cities for adrenoleukodystrophy clinical trials?
When it comes to adrenoleukodystrophy clinical trials, several cities have emerged as key players in research. Minneapolis, Minnesota leads the way with 3 active trials focusing on treatments like IV-MSC for cALD (Early Disease/Bridge Therapy), Blood and Marrow Transplant, and Stem Cell Transplantation. Palo Alto, California; Baltimore, Maryland; and Boston, Massachusettsw Transplant, and Stem Cell Transplantation. Palo Alto, California; Baltimore, Maryland; and Boston, Massachusetts follow closely behind with 2 ongoing studies each examining therapies such as Leriglitazone, VK0214, and Pramipexole. Additionally, Minnesota is also contributing to the advancement of adrenoleukodystrophy research with its own trials centered around Leriglitazone. These cities offer hope to patients by providing access to cutting-edge clinical trials that strive towards improved outcomes for those affected by adrenoleukodystrophy.
Which are the top treatments for adrenoleukodystrophy being explored in clinical trials?
Exciting advancements are being made in the search for effective treatments against adrenoleukodystrophy. Prominent contenders include:
Leriglitazone: With one active trial and a single all-time adrenoleukodystrophy trial, leriglitazone emerged on the scene in 2023 as a potential game-changer.
SBT101: Introducing itself to the field in 2022, SBT101 shows promise with one ongoing clinical trial dedicated to tackling adrenoleukodystrophy.
Pramipexole: Also making waves in 2023 is pramipexole, currently under investigation through an active trial targeting adrenoleukodystrophy.
VK0214: A newcomer from 2021, VK0214 has swiftly captured attention with its inclusion in one active and one all-time clinical trials exploring treatment options for this challenging condition.
These innovative therapies hold significant potential to transform the lives of those affected by adrenoleukodystrophy.
What are the most recent clinical trials for adrenoleukodystrophy?
Recent clinical trials offer hope for individuals with adrenoleukodystrophy, a rare genetic disorder. One groundbreaking study focuses on IV-MSC therapy as an early disease treatment and bridge therapy for childhood cerebral adrenoleukodystrophy (cALD). Another trial investigates the potential of leriglitazone, a medication that targets the underlying mechanisms of adrenoleukodystrophy. Additionally, pramipexole is being evaluated in Phase 4 to assess its effectiveness in managing symptoms associated with this debilitating condition. Furthermore, SBT101 shows promise in both Phase 1 and Phase 2 trials for treating different forms of adrenoleukodystrophy. Lastly, VK0214's Phase 1 trial explores its potential therapeutic benefits. These pioneering studies aim to advance understanding and provide new treatment options for those affected by adrenoleukodystrophy.
What adrenoleukodystrophy clinical trials were recently completed?
Recently completed clinical trials for adrenoleukodystrophy have made significant strides in the search for effective treatments. In January 2019, bluebird bio successfully concluded a trial investigating the potential of Lenti-D therapy. Additionally, the University of Florida completed a trial in June 2018 exploring Thiotepa--escalated dose as a potential treatment option. These advancements represent important steps forward in our understanding and management of adrenoleukodystrophy, offering hope to patients affected by this complex disorder.