Growth Hormone Therapy for Pseudohypoparathyroidism
This trial is observing the effects of growth hormone on patients with Albright hereditary osteodystrophy, specifically those with pseudohypoparathyroidism type 1a. This form of the condition typically leads to patients being short and obese. The trial is also observing the neurocognitive and psychosocial effects of the condition in order to better understand how to manage it.
Device Validation Using Blood Samples for Thrombotic Thrombocytopenic Purpura
This trial is for a new device that needs to be validated with known samples. 3 ml of blood is needed from people diagnosed with hTTP. You will be compensated for your time and resources.
Vasopressin Analog
PB for Nephrogenic Diabetes Insipidus
This trial is testing a drug called PB to see if it can help people who urinate frequently due to certain kidney conditions or treatments. It aims to help the kidneys manage water better, so patients don't have to go to the bathroom as often.
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Trials for HAE Patients
Gene Therapy Vector
Gene Therapy for Hereditary Angioedema
This trial tests BMN 331, a gene therapy given through an IV, for people with hereditary angioedema (HAE) who lack a specific protein. The therapy uses a virus to deliver a healthy gene to liver cells, enabling them to produce the missing protein. The study aims to see if this treatment is safe and effective over several years. BMN 331 is a gene therapy that uses a virus to deliver a healthy gene to liver cells, a method that has shown promise in treating various genetic disorders.
Bradykinin B2 receptor antagonist
Long-Term Safety of KVD900 for Hereditary Angioedema
This trial tests the long-term safety of KVD900 in individuals with HAE. KVD900 aims to reduce symptoms by blocking proteins that cause swelling. Another treatment, Berotralstat, is used to prevent HAE attacks.
Phase 3 Trials
RNAi Therapeutics
Vutrisiran vs Patisiran for Amyloidosis
This trial is testing vutrisiran, an under-the-skin injection, in patients with a genetic condition called hATTR amyloidosis. The medication aims to lower harmful protein levels in their bodies. Vutrisiran is administered as a periodic subcutaneous injection.
Antisense Oligonucleotide
Eplontersen for Amyloid Neuropathy
This trial is testing the safety of Eplontersen, a medication given regularly, in patients with a genetic condition that causes nerve damage. The treatment works by lowering harmful protein levels to reduce nerve damage.
Bradykinin B2 receptor antagonist
Long-Term Safety of KVD900 for Hereditary Angioedema
This trial tests the long-term safety of KVD900 in individuals with HAE. KVD900 aims to reduce symptoms by blocking proteins that cause swelling. Another treatment, Berotralstat, is used to prevent HAE attacks.
Trials With No Placebo
RNAi Therapeutics
Vutrisiran vs Patisiran for Amyloidosis
This trial is testing vutrisiran, an under-the-skin injection, in patients with a genetic condition called hATTR amyloidosis. The medication aims to lower harmful protein levels in their bodies. Vutrisiran is administered as a periodic subcutaneous injection.
Frequently Asked Questions
Introduction to hereditary angioedema
What are the top hospitals conducting hereditary angioedema research?
In the realm of hereditary angioedema research, several hospitals have made significant progress in their clinical trials. AARA Research Center in Dallas leads the way with five ongoing trials and a total of 30 studies dedicated to this condition throughout their history. Since conducting their first hereditary angioedema trial in 2005, they have been at the forefront of understanding and treating this rare genetic disorder. Meanwhile, Washington University School of Medicine in Saint Louis has also emerged as a prominent institution for research on hereditary angioedema. With four active trials and an impressive eleven completed studies thus far, their contributions since starting their inaugural trial in 2010 are noteworthy.
Additionally making valuable contributions is Optimed Research, LTD based in Columbus. This facility currently has three active clinical trials focused on hereditary angioedema and has conducted ten previous investigations since initiating their first trial back in 2009.
Notably joining these leading institutions is Ionis Investigative Site located both in Santa Monica and Dallas; though relatively new to the scene having recorded its initial investigation only last year (2020), it is actively pursuing three concurrent hereditary angioedema trials alongside three past endeavors exploring this field.
These hospitals form a network of expertise that collectively advances our understanding and treatment options for individuals living with hereditary angioedema - an uncommon yet impactful condition characterized by recurrent episodes of swelling beneath the skin's surface. The dedication exhibited by these institutions not only offers hope for affected individuals but reinforces our commitment to unraveling mysteries surrounding genetic disorders while improving lives through innovation within medical science
Which are the best cities for hereditary angioedema clinical trials?
When it comes to hereditary angioedema clinical trials, several cities have emerged as key players in research and development. Dallas, Texas leads the pack with 12 active trials focusing on investigational treatments like Donidalorsen, KVD900 600 mg, and Donidalorsen: Cohort A. Following closely behind are Santa Monica, California and Saint Louis, Missouri with 11 ongoing studies exploring similar interventions. Additionally, Hershey, Pennsylvania and Scottsdale Arizona each offer 10 active trials investigating potential breakthroughs for hereditary angioedema treatment options. These cities provide individuals with access to cutting-edge clinical trials that pave the way for advancements in care for those living with this condition.
Which are the top treatments for hereditary angioedema being explored in clinical trials?
The field of hereditary angioedema (HAE) research is abuzz with exciting developments in clinical trials. Leading the charge is KVD900 600 mg, currently being explored in three active trials for HAE since its listing in 2022. Not far behind is donidalorsen, making waves with two ongoing trials and four all-time HAE studies since its introduction in 2020. Also worth noting is berotralstat, showing promising potential with one active trial and one all-time HAE study after debuting in 2022. These groundbreaking treatments offer hope for individuals affected by HAE and pave the way for improved management strategies moving forward.
What are the most recent clinical trials for hereditary angioedema?
Exciting advancements in clinical trials for hereditary angioedema offer hope and potential treatments for individuals affected by this condition. Among the recent studies, povorcitinib has shown promise as a possible therapy. This Phase 2 trial evaluates the efficacy of povorcitinib at different doses specifically tailored for hereditary angioedema patients. Another study focuses on pazopanib, examining its effectiveness at a dose of 150 mg during both Phase 2 and Phase 3 trials. Additionally, single-dose cohort trials are being conducted to assess various treatment options for hereditary angioedema. These ongoing efforts highlight the commitment to finding effective therapies that can improve the lives of those living with hereditary angioedema
What hereditary angioedema clinical trials were recently completed?
In the realm of hereditary angioedema, several clinical trials have recently concluded, showcasing advancements in potential treatments. Notably, Shire completed a trial for Lanadelumab in both February 2021 and May 2020. Another significant development occurred with CSL Behring's completion of a trial for CSL312 in January 2021. Furthermore, BioCryst Pharmaceuticals conducted two separate trials: one for BCX7353 capsules and another for BCX7353, both concluding in February 2018. These recent endeavors demonstrate the ongoing commitment to finding effective therapies for individuals grappling with hereditary angioedema.