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A Phase 3, Double-blind, Placebo-controlled, Randomized Withdrawal Study to Evaluate the Efficacy and Safety of SYNB1934 in Patients with PKU (SYNPHENY-3)
EU CTIS ID: 2022-502932-37-00
What this study is testing
Dose Escalating, Open-Label Period (DEP; Part 1): To assess the percentage change in blood phenylalanine (Phe) level Randomized Withdrawal Period (RWP; Part 2): To evaluate the efficacy of SYNB1934v1 versus placebo in the responder population Open-Label Extension (OLE; Part 3): To assess the safety and tolerability of SYNB1934v1
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Age ≥ 18 years. Participants 12 to 17 years of age may be enrolled after the first 20 participants have completed the DEP, with approval of the DMC.
- Able and willing to voluntarily complete the informed consent process (adults; parents/guardians) or informed assent process (minors).
- Diagnosis of PKU and failure to maintain recommended blood Phe levels on existing management (sapropterin, sepiapterin and/or Phe-restricted diet), demonstrated by uncontrolled blood Phe level > 360 μmol/L on current therapy any time during screening and uncontrolled blood Phe level > 360 μmol/L on current therapy when taking the average of the 3 most recent Phe levels from the participant’s medical history (inclusive of any screening values). All screening values must be obtained more than 7 days apart, as determined by central or local laboratory.
- Females of childbearing potential must have a negative pregnancy test at screening and the end of DEP (in order to enter Part 2) and RWP (in order to enter Part 3) and be willing to have additional pregnancy tests during the study.
- Sexually active female participants of childbearing potential must be willing to use a highly effective method of contraception while participating in the study and for 2 weeks after the last dose.
- Stable diet including stable medical formula regimen (if used) for at least 1 month prior to screening.
You likely can't join if
- Currently taking Palynziq® (pegvaliase-pqpz) (within 1 month of screening).
- Acute or chronic medical, surgical, psychiatric, or social condition or laboratory abnormality that may increase participant risk associated with study participation, compromise adherence to study procedures and requirements, and, in the judgment of the investigator, would make the participant inappropriate for enrollment.
- A known or suspected diagnosis of DNAJC12 deficiency, biopterin synthesis deficiency, or irritable bowel syndrome.
- Intolerance of or allergic reaction to E. coli Nissle or any of the ingredients in SYNB1934v1 or placebo formulations, or an allergy to cinnamon. Known intolerance to proton pump inhibitors and H2 blockers, since one or the other must be used.
- Currently taking or plans to take any type of systemic (e.g., oral or intravenous) antibiotic within 28 days prior to the first dose of IMP through final safety assessment in RWP, including planned surgery, hospitalizations, dental procedures, or interventional studies that are expected to require antibiotics. Exception: topical antibiotics are allowed.
- Pregnant, planning to become pregnant, or breastfeeding.
See the full eligibility criteria
Who can join
- Age ≥ 18 years. Participants 12 to 17 years of age may be enrolled after the first 20 participants have completed the DEP, with approval of the DMC.
- Able and willing to voluntarily complete the informed consent process (adults; parents/guardians) or informed assent process (minors).
- Diagnosis of PKU and failure to maintain recommended blood Phe levels on existing management (sapropterin, sepiapterin and/or Phe-restricted diet), demonstrated by uncontrolled blood Phe level > 360 μmol/L on current therapy any time during screening and uncontrolled blood Phe level > 360 μmol/L on current therapy when taking the average of the 3 most recent Phe levels from the participant’s medical history (inclusive of any screening values). All screening values must be obtained more than 7 days apart, as determined by central or local laboratory.
- Females of childbearing potential must have a negative pregnancy test at screening and the end of DEP (in order to enter Part 2) and RWP (in order to enter Part 3) and be willing to have additional pregnancy tests during the study.
- Sexually active female participants of childbearing potential must be willing to use a highly effective method of contraception while participating in the study and for 2 weeks after the last dose.
- Stable diet including stable medical formula regimen (if used) for at least 1 month prior to screening.
- If using sapropterin or sepiapterin, must be on a stable dose for at least 3 months.
- Willing and able to continue current diet, sapropterin, sepiapterin and large neutral amino acids unchanged during screening, DEP, and RWP and to engage in all study activities.
What rules you out
- Currently taking Palynziq® (pegvaliase-pqpz) (within 1 month of screening).
- Acute or chronic medical, surgical, psychiatric, or social condition or laboratory abnormality that may increase participant risk associated with study participation, compromise adherence to study procedures and requirements, and, in the judgment of the investigator, would make the participant inappropriate for enrollment.
- A known or suspected diagnosis of DNAJC12 deficiency, biopterin synthesis deficiency, or irritable bowel syndrome.
- Intolerance of or allergic reaction to E. coli Nissle or any of the ingredients in SYNB1934v1 or placebo formulations, or an allergy to cinnamon. Known intolerance to proton pump inhibitors and H2 blockers, since one or the other must be used.
- Currently taking or plans to take any type of systemic (e.g., oral or intravenous) antibiotic within 28 days prior to the first dose of IMP through final safety assessment in RWP, including planned surgery, hospitalizations, dental procedures, or interventional studies that are expected to require antibiotics. Exception: topical antibiotics are allowed.
- Pregnant, planning to become pregnant, or breastfeeding.
- Current participation in any other investigational drug study or use of any investigational agent within 30 days or 5 half-lives (whichever is longer) prior to screening.
- Ever received gene therapy for treatment of PKU.
The study team makes the final eligibility decision.
Where it's taking place
- Turkey
- United States
- Canada
- Georgia
- Israel
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Turkey; United States; Canada; Georgia; Israel. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.