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.