Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria
SynPheny-1
An Open-label Study of the Efficacy and Safety of SYNB1618 and SYNB1934 in Patients With Phenylketonuria (SynPheny-1)
1 other identifier
interventional
20
1 country
11
Brief Summary
This Phase 2 study in patients with phenylketonuria (PKU) will be an open-label, dual-arm study of either a SYNB1618 or SYNB1934 dose-ramp regimen. All evaluations and assessments throughout this study may be conducted either at the clinical site or by a home healthcare professional at an alternative location (e.g., patient's home, hotel).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Aug 2020
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 24, 2020
CompletedStudy Start
First participant enrolled
August 25, 2020
CompletedFirst Posted
Study publicly available on registry
September 1, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 7, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
October 7, 2022
CompletedOctober 17, 2022
October 1, 2022
2.1 years
August 24, 2020
October 14, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
Changes from baseline in labeled Phe (D5-Phe) in plasma
The primary efficacy outcome will be assessed by measuring change from baseline in plasma D5-Phe AUC over 24 hours after D5-Phe administration
Day 14
Secondary Outcomes (2)
Changes from baseline in fasting levels of plasma Phe
Day 14
Incidence of Treatment-Emergent Adverse Events
Day 43
Study Arms (2)
SYNB1618
EXPERIMENTALDose ramp of SYNB1618
SYNB1934
EXPERIMENTALDose ramp of SYNB1934
Interventions
Eligibility Criteria
You may qualify if:
- Age ≥ 18 years.
- Able and willing to voluntarily complete the informed consent process.
- Diagnosis of classic PKU based on medical history as assessed by the investigator (e.g., Phe concentration of \>1200 µmol/L at any time, low dietary Phe tolerance, or genetic diagnosis).
- Blood Phe ≥ 600 µmol/L at Screening.
- Stable diet including stable medical formula regimen (if used) for at least 1 month prior to Screening.
- Available for and agree to all study procedures, including urine and blood collection, adherence to diet control, follow-up visits, and IMP ingestion compliance.
- Female patients who meet one of the following criteria:
- Premenopausal woman with one of the following:
- i. Documented hysterectomy, ii. Documented bilateral salpingectomy, iii. Documented bilateral oophorectomy, iv. Documented tubal ligation/occlusion, v. Sexual abstinence is preferred or usual lifestyle of the patient c. Postmenopausal women (12 months or more amenorrhea verified by follicle stimulating hormone \[FSH\] assessment and over 45 years of age, in the absence of other biological or physiological causes).
- Screening laboratory evaluations (e.g., chemistry panel, complete blood count \[CBC\] with differential, urinalysis, creatinine clearance, CRP) within normal limits or judged to be not clinically significant by the investigator.
You may not qualify if:
- Currently taking Palynziq® (pegvaliase-pqpz) (within 1 month of Screening).
- Inflammatory or irritable bowel disorder of any grade.
- History of or current immunodeficiency disorder.
- Intolerance of or allergic reaction to E. coli Nissle or any of the ingredients in SYNB1618 formulation.
- Any condition (e.g., celiac disease, gastrectomy, bypass surgery, ileostomy) or receiving prescription medication or an over-the-counter product that may possibly affect absorption of medications or nutrients.
- 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, including planned surgery, hospitalizations, dental procedures, or interventional studies that are expected to require antibiotics. Exception: topical antibiotics are allowed.
- Within the 3 months prior to anticipated first dose, major surgery (an operation upon an organ within the cranium, chest, abdomen, or pelvic cavity) or inpatient hospital stay.
- Dependence on alcohol or drugs of abuse.
- Administration or ingestion of an investigational drug within the 30 days or 5 half-lives before Screening, whichever is longer, or cell/gene therapy prior to the Screening visit, or current enrollment in an investigational drug study.
- Acute or chronic medical, surgical, psychiatric, or social condition or laboratory abnormality that may increase patient risk associated with study participation, compromise adherence to study procedures and requirements, or may confound interpretation of study safety or PD results and, in the judgment of the investigator, would make the patient inappropriate for enrollment.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Synlogiclead
Study Sites (11)
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Excel Medical Clinical Trials, LLC
Boca Raton, Florida, 33434, United States
University of Florida College of Medicine
Gainesville, Florida, 32608, United States
Washington University in St Louis
St Louis, Missouri, 63110, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
Oregon Health and Science University
Portland, Oregon, 97201, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
University of Texas Health Science Center at Houston
Houston, Texas, 77030, United States
PRA Health Sciences
Salt Lake City, Utah, 84124, United States
University Hospital Adult Services
Salt Lake City, Utah, 84132, United States
Related Publications (1)
Vockley J, Sondheimer N, Puurunen M, Diaz GA, Ginevic I, Grange DK, Harding C, Northrup H, Phillips JA 3rd, Searle S, Thomas JA, Zori R, Denney WS, Ernst SL, Humphreys K, McWhorter N, Kurtz C, Brennan AM. Efficacy and safety of a synthetic biotic for treatment of phenylketonuria: a phase 2 clinical trial. Nat Metab. 2023 Oct;5(10):1685-1690. doi: 10.1038/s42255-023-00897-6. Epub 2023 Sep 28.
PMID: 37770764DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 24, 2020
First Posted
September 1, 2020
Study Start
August 25, 2020
Primary Completion
October 7, 2022
Study Completion
October 7, 2022
Last Updated
October 17, 2022
Record last verified: 2022-10