Clinical Trial for Individuals with Pyruvate Dehydrogenase Complex Deficiency (PDCD)

Saol Therapeutics has partnered with InformedDNA to assist in locating individuals who may be eligible for a clinical trial aimed at treating Pyruvate Dehydrogenase Complex Deficiency (PDCD).

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InformedDNA’s 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.

Speaking with a genetic counselor at InformedDNA about this study is provided at no cost to the individual. After speaking with a genetic counselor, individuals interested in learning more about the clinical research study will be connected with a trial site.

Information about the Clinical Trial

Phase 3 Trial of Dichloroacetate (DCA) for Pyruvate Dehydrogenase Complex Deficiency (PDCD)

( link: Trial of DCA in Pyruvate Dehydrogenase Complex Deficiency)

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The goal of this research study is to conduct a pivotal phase-3 trial of treatment with the investigational drug dichloroacetate (DCA) in young children with deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency (PDCD) is the most common cause of congenital (disease present from birth) lactic acidosis (condition in which a person overproduces or under uses lactic acid and the body is not able to adjust to these changes) and is a metabolic disease of childhood for which no proven treatment exists.

The purpose of the research study is to determine if DCA improves the health and function of patients with PDCD. A novel Observer reported outcome (ObsRO) survey – that is completed by study participant’s parent/caregiver – is the efficacy outcome measure.

  • Compensation will be provided for completion of the daily survey tool.
  • Participation only requires up to six visits to a study site over 10 months and a daily five-minute survey about how the patient is feeling that day. The study allows the option of attending two of the visits at a laboratory located near the patient’s home.
  • The study medicine is a strawberry flavored syrup that is mailed to the patient’s home each month. The medicine is administered by mouth or gastrostomy tube twice per day.
  • The study is being conducted at nine academic health centers throughout the U.S.  Visit this webpage to find a study site near you. Travel assistance is provided (if needed).

How to refer a patient or to request an appointment with a Genetic Counselor at InformedDNA to further discuss this trial opportunity:

Healthcare providers

Patients and Caregivers

Speak with a member of our patient care team to schedule an appointment with a board-certified genetic counselor.

Call 888-315-5133

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