Currently Recruiting Clinical Trials/Studies For Propionic Acidemia
NIH Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
This study will evaluate patients with propionic acidemia to learn more about the genetic and biochemical causes and the medical complications associated with it. They also plan to study how bacteria living in our gut (microbiome) can affect the course of propionic acidemia.
Insight Study – HemoShear is now recruiting participants for the MMA and PA Insight Study. The study will evaluate how different assessments can measure verbal communication, processing speed, life skills, quality of life and sleep. The study is conducted virtually – no clinic visits are required.
HERO Study – HST5040 – Hemoshear is now recruiting participants
- HemoShear Receives IND clearance for HST5040 for metabolic diseases June 2020
- Introducing HST5040 Being Developed for Methylmalonic and Propionic Acidemias (for clinicians) – presented by HemoShear Therapeutics-April13,2021
- PAF/ OAA invite you to Caregiver Webinar on New Horizons for MMA/PA: Current Treatments, Clnical Trials and New Approaches Saturday, April 17, 2021 – Video from Webinar
- HERO Study Information
- Letter to community about HERO study and webinar
- VIDEO On PA and HST5040
As of 6/9/21 – the HERO study is currently enrolling patients at these sites:
- Boston Children’s Hospital
- Children’s Hospital of Pittsburgh
- Children’s National Health System
- Children’s Mercy in Kansas City
- University of Minnesota
They will soon be recruiting at:
- Rady Children’s Hospital (UC San Diego)
- University of Florida
- University of Utah
- Vanderbilt University
- Yale (in the fall)
Open Label Study of mRNA-3927 in Patients With Propionic Acidemia
The Paramount Study is a Phase 1/2 study. It is designed to evaluate if an investigational treatment called mRNA-3927 is safe and effective in reducing the symptoms of PA in individuals one year of age and older. mRNA 3927 is an investigational intravenous (IV) infusion treatment that instructs a persons’ body to make a PCC enzyme that works.
- PAF/OAA invite you to Advancing mRNA to Treat Organic Acidemias – Informational Webinar on Moderna’s upcoming treatment and clinical trial. – April 10, 2021
- Study Information
- Moderna Flier Spring 2021
Study on BBP-671
CoA Therapeutics has completed the necessary preclinical studies in animals with their small molecule drug candidate, BBP-671, to support beginning human trials. Additionally, the U.S. Federal Drug Association (FDA) has approved their Investigational New Drug (IND) application to study BBP-671 in humans. In April 2021, they started a Phase 1 study to assess the safety of the drug in healthy volunteers before it is studied in PA and MMA patients. They plan to begin PA and MMA patient trials once we know that the drug candidate is safe, and have identified a dose appropriate for PA and MMA patients.
Propionic Acidemia Foundation website is designed for educational purposes only and is not intended to serve as medical advice. The information provided on this site should not be used for diagnosing or treating a health problem or disease. It is not a substitute for professional care. If you suspect that you or your children may have Propionic Acidemia, you should consult your health care provider. Any potential therapy should be thoroughly discussed with your medical provider. The Propionic Acidemia Foundation does not recommend nor endorse any particular products, therapeutics, companies, or manufacturers.