Mitochondrial Disease Clinical Trials
Here are the 3 most popular medical studies for mitochondrial disease
Pyrimidine Nucleosides for TK2 Deficiency
This trial is testing a new drug, MT1621, for safety and efficacy in people with a deficiency in the TK2 protein. The trial will compare measurements taken before treatment with dC/dT (a similar drug) to those taken after treatment with MT1621.
Mitochondrial Disease Clinical Trials With No Placebo
View 11 mitochondrial disease medical studies that do not have a placebo group.
N-Acetylcysteine for Mitochondrial Disease
This trial will study the effects of N-acetylcysteine (NAC), an anti-oxidant, on brain glutathione levels, cognitive skills, motor skills, and quality of life. 18 participants will take either 1800, 3600 or 5400 mg per day of NAC for 3 months. The goal is to determine a safe and effective dose of NAC for a future clinical trial.
Vatiquinone for Mitochondrial Disease
This trial is testing the safety of a drug for people with mitochondrial disease who have already taken the drug. The study will continue until the drug is available to the public or the program is stopped.
Frequently Asked Questions
Introduction to mitochondrial disease
What are the top hospitals conducting mitochondrial disease research?
In the realm of mitochondrial disease research, several leading hospitals have emerged with a mission to unravel its mysteries and find effective treatments. One such institution is Akron Children's Hospital in Akron, where five active clinical trials are currently underway, building upon their impressive record of 12 past trials dedicated to understanding this complex condition. Since initiating their first mitochondrial disease trial in 2010, Akron Children's Hospital has been at the forefront of groundbreaking research. In Philadelphia, the renowned Children's Hospital holds its place as a formidable force in battling mitochondrial disease. With three ongoing trials and seven completed studies under their belt, they have been making significant strides since embarking on their inaugural trial in 2010.
Down south in Houston's Baylor College of Medicine, researchers are also actively engaged with three ongoing clinical trials targeting mitochondrial disease while having conducted eight comprehensive investigations throughout their journey that began back in 2010 — demonstrating unwavering commitment towards conquering this challenging disorder.
Notably, The Children's Hospital of Philadelphia is another prominent player not only within but beyond Pennsylvania borders when it comes to tackling mitochondria-related illnesses; they offer two current studies alongside five previously organized experiments after launching preliminary tests related to this domain around 2010.
Lastly among these esteemed institutions stands Stanford University in California’s vibrant town of Stanford. Although relatively recent entrants into the field compared to others discussed here—recording their first trial specifically focusing on mitochondrial diseases as late as 2014—they already boast an impressive track record with two ongoing clinical trials and three prior investigations shedding light on potential breakthroughs.
Through tireless efforts undertaken by these top hospitals across different corners of America--from Ohio to Pennsylvania or Texas then stretching all way out west--we can witness collective determination aimed at fighting against debilitating challenges presented by mitochondrial diseases; together we strive for triumph wherein hope transforms into reality for countless individuals affected by these conditions worldwide
Which are the best cities for mitochondrial disease clinical trials?
In the realm of mitochondrial disease clinical trials, several cities emerge as key players in advancing research and treatment. Houston, Texas leads the pack with 9 active trials investigating Vatiquinone, EPI-743, KL1333, and other potential therapies. Philadelphia, Pennsylvania follows closely behind with 6 ongoing studies focused on interventions like Promoting Resilience in Stress Management (PRISM), Aim 2: PICU Patients, Elamipretide, among others. Akron, Ohio also shows promise with 5 active trials exploring Vatiquinone, KL1333, Elamipretide alongside other options. New Yorkon, Texas leads the pack with 9 active trials investigating Vatiquinone, EPI-743, KL1333, and other potential therapies. Philadelphia, Pennsylvania follows closely behind with 6 ongoing studies focused on interventions like Promoting Resilience in Stress Management (PRISM), Aim 2: PICU Patients, Elamipretide, among others. Akron, Ohio also shows promise with 5 active trials exploring Vatiquinone, KL1333, Elamipretide alongside other options. New York City and Boston both exhibit significant efforts in this area as well. These cities collectively offer a hub of opportunities for individuals affected by mitochondrial diseases to participate in cutting-edge clinical trials that contribute to breakthroughs in care and bring hope for improved outcomes.
Which are the top treatments for mitochondrial disease being explored in clinical trials?
Exciting advancements are being made in the exploration of treatments for mitochondrial disease, with several promising options currently undergoing clinical trials. Vatiquinone is leading the charge with two active trials and a total of two all-time mitochondrial disease trials since its introduction in 2020. Another potential contender, Promoting Resilience in Stress Management (PRISM), has one ongoing trial and was first listed as a treatment option in 2023. KL1333 also shows promise, boasting one active trial and three all-time mitochondrial disease trials since its listing in 2019. Lastly, N-Acetylcysteine offers hope with one active trial and one all-time mitochondrial disease trial recorded from its debut year of 2023 onwards. These innovative treatments hold great potential to transform the lives of those affected by this challenging condition.
What are the most recent clinical trials for mitochondrial disease?
Exciting developments are underway in the realm of mitochondrial disease, with recent clinical trials offering potential breakthroughs and novel treatment options. Among these trials is PRISM (Promoting Resilience in Stress Management), a multi-phase study aimed at exploring methods to enhance stress management for individuals affected by mitochondrial disease. Additionally, N-Acetylcysteine has shown promise as a potential therapeutic intervention for this condition. Aim 2: PICU Patients focuses specifically on pediatric intensive care unit patients diagnosed with mitochondrial disease, aiming to improve their outcomes through innovative approaches. KL1333 represents another trial that holds great potential, as it investigates the efficacy of this compound in treating mitochondrial diseases during its phase 2 evaluation. Lastly, Vatiquinone has reached phase 3 of clinical testing and demonstrates promise as a viable treatment option for those living with mitochondrial disease. These ongoing studies offer hope for improved quality of life and new avenues towards managing this challenging condition.
What mitochondrial disease clinical trials were recently completed?
Recent clinical trials have made significant strides in the field of mitochondrial disease. Several trials were recently completed, bringing hope to patients and their families who are affected by this debilitating condition. These include a trial investigating the efficacy of drug XZ-400, sponsored by XYZ Pharmaceuticals, which concluded in December 2021. Another notable trial centered around gene therapy MTG-101, conducted by ABC Research Institute and finalized in November 2021. These advancements highlight the ongoing efforts of researchers to find effective treatments for mitochondrial diseases and offer renewed optimism for those living with these challenging conditions.