Program Discusses Access to a Pivotal Phase 3 Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency (DCA/PDCD trial; NCT02616484)
Saol Therapeutics, a company researching new treatments for rare diseases, is pleased to announce a partnership with InformedDNA®, the nation’s leading applied genomics solutions company, to offer genetic counseling for the families of individuals with a rare mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD). The counseling will also review potential participation in a Phase 3 clinical trial. PDCD affects less than 300 children in the United States annually and lacks any FDA-approved treatment.
InformedDNA’s board-certified genetic counselors are highly knowledgeable about inherited metabolic disorders and can answer questions and review eligibility to enable individuals to make more informed decisions about clinical trial participation. After speaking with a genetic counselor, families interested in learning more about the clinical research study will be connected with a trial site.
This pivotal phase 3 trial administers the investigational drug dichloroacetate (DCA) to children who have a deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency is the most common cause of congenital lactic acidosis and is frequently fatal. DCA has Orphan Product designation from the FDA for congenital lactic acidosis (CLA), including patients with PDCD. (ClinicalTrials.gov link: Trial of DCA in Pyruvate Dehydrogenase Complex Deficiency)
Some of the questions and topics addressed during the genetic counseling appointment include:
- Discussion of the clinical features, progression, and inheritance of Pyruvate Dehydrogenase Complex Deficiency (PDCD)
- High-level overview of the clinical trial and participation
Dr. Peter Stacpoole, Principal Investigator of the DCA/PDCD trial and Prof. of Medicine at the University of Florida, notes that “There are currently no FDA-approved treatments for patients with PDCD. This service allows them to make an informed decision about clinical trial participation, which is critical to advancing treatment options for rare diseases.”
Dave Penake, CEO of Saol Therapeutics, is pleased to partner with InformedDNA to provide this valuable service. “Individualized genetic counseling offers families the insights needed to better understand PDCD and to determine if participation in this clinical trial makes sense. Completing the trial and having a potential FDA-approved treatment for PDCD will be a huge milestone for Saol and the families that have participated in the study.”
“Like many rare genetic diseases, PDCD is a severe disease with no proven therapies. Many affected patients do not survive childhood. We are thrilled that Saol Therapeutics is investing in this rare disease, and we are passionate about leveraging InformedDNA’s highly effective virtual screening process to engage and connect appropriate patients to the clinical trial and advance therapeutic progress in this devastating disease,” said Karmen Trzupek, Director of Clinical Trial Services at InformedDNA. Healthcare providers and caregivers for children with Pyruvate Dehydrogenase Complex Deficiency (PDCD) may request a genetic counseling appointment by visiting www.InformedDNA/Saol.
About Saol Therapeutics
Saol Therapeutics (pronounced “Sail”) is a privately held, biopharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda. Saol is focused on clinical development activity in rare diseases, with a focus on mitochondrial disorders, as well as central nervous system disorders such as spasticity and pain management. Saol is one of the collaborators on a Phase 3 trial studying the first potential treatment for pyruvate dehydrogenase complex deficiency (PDCD). More information on the clinical trial can be found at Phase 3 PDCD Trial. More information about Saol can be found at https://saolrx.com/.
InformedDNA is the country’s leading applied genomics solutions company, helping harness the full power of precision medicine. With the largest independent staff of board-certified genetics specialists in the U.S., InformedDNA ensures that health organizations have access to the highest quality, most current genomics insights to optimize clinical decisions. Our solutions, which cover evidence-based guideline development, patient experience management, and value management, have helped optimize the health benefits of more than 135 million covered lives and have navigated hundreds of thousands of people to the right treatments or clinical trials. For more information, visit www.InformedDNA.com.
Clinical Trial Contact:
Kathy Dorsey, Director of Clinical Operations, Saol Therapeutics, KDorsey@Saolrx.com
Brian Nappi, Senior Vice President Strategy, Saol Therapeutics, firstname.lastname@example.org
Megan Smith, MERGE for InformedDNA, email@example.com, 404-408-3379
SOURCE Saol Therapeutics