Clinical Trial 41379

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Portland, OR 97239


Study Summary:

WHAT’S NEW IN PKU?

Synlogic is conducting a Phase 1 clinical research study to evaluate the safety of a potential new treatment option for PKU. We’re seeking adult patients with classic PKU to participate in the study.

STUDY OVERVIEW

Phenylketonuria (PKU) is a rare genetic disorder affecting about 16,500 people in the U.S. PKU is caused by a defect in the gene that helps create the enzyme responsible for breaking down an amino acid called phenylalanine. Phenylalanine builds up in the body, causing health problems like:

  • Trouble focusing or concentrating
  • Neurological problems, including seizures
  • Behavioral, emotional, or social problems
  • Irreversible brain damage
With strict diet management you can manage your symptoms and prevent health problems. However, if you’re looking for more options to help you better manage your PKU, you may be interested in this study investigating a new oral treatment option.

STUDY DURATION: 19 weeks, including:
  • 60-day screening
  • 10-day inpatient treatment period
  • 8-week follow-up period
INVESTIGATIONAL DRUG
  • SYNB1618, a modified probiotic
  • Designed to break down phenylalanine in the diet
  • Given orally as a liquid
KEY ELIGIBILITY CRITERIA
  • Between ages 18 and 64 (inclusive)
  • Have been diagnosed with classic PKU
  • Not currently taking sapropterin (KUVAN®) (within the past week) or pegylated recombinant phenylalanine ammonia lyase (PALYNZIQTM) (within the past 6 months)
ALL ELIGIBLE PARTICIPANTS WILL RECEIVE AT NO COST
  • Close care and monitoring throughout the study
  • Investigational drug or placebo
  • All study-related visits
  • Compensation for participation, including travel and study-related activities

See if you may be eligible.


Study is Available At:

See if you may be eligible.

To view an updated list of participating research sites click here.

 


Clinical trials are medical research studies designed to test the safety and/or effectiveness of new investigational drugs, devices, or treatments in humans. These studies are conducted worldwide for a range of conditions and illnesses. Learn more about clinical research and participating in a study at About Clinical Trials.