Phenylketonuria
Conditions
Interventions
placebo group:Receive an oral intervention with an inactivated placebo that is identical in appearance, formulation, and route of administration to the study drug
low dose group:Receive oral administration of the low dose engineered probiotic CPK .
High dose group:Receive oral administration of the high dose engineered probiotic CPK .
Sponsors
Peking University First Hospital
Eligibility
Sex/Gender
All
Age
3 Years to 17 Years
Inclusion criteria
Inclusion criteria: 1. Clinically diagnosed PKU, with a blood Phe level of >=360 µmol/L in the newborn screening, and at least three instances of a blood Phe level of >=360 µmol/L within the year prior to screening, with the closest test result still being >=360 µmol/L; 2. Applicants must be between the ages of 3 and 17; there are no gender restrictions; 3. Maintain a stable low-Phe diet for at least 60 days prior to screening, with no significant dietary changes; 4. Average of at least two bowel movements per week, no persistent diarrhea, and no use of laxatives; 5. Body weight falls between the 3rd and 97th percentiles of the standard growth curve for children and adolescents of the same age and gender; 6. Laboratory tests conducted during the screening period—including complete blood count, liver and kidney function tests, urinalysis, creatinine clearance, and CRP—are essentially normal, or any abnormalities are clinically insignificant; 7. No immunodeficiency, autoimmune diseases, active infections, or severe chronic diseases; 8. The guardian fully understands the study protocol, voluntarily signs the informed consent form, and is able to cooperate fully with follow-up visits, sample collection, and medication administration throughout the study.
Exclusion criteria
Exclusion criteria: 1. A history of or current occurrence of loose stools >=3 times daily, chronic diarrhea, or severe gastrointestinal dysfunction; 2. Presence of gastrointestinal diseases that affect intestinal absorption, active bleeding, or a history of surgery (major surgery within 90 days prior to screening); 3. Participants who have used antibiotics or probiotic supplements within the past 28 days, or who will need to use them during the study; 4. Exclude participants who have used gastrointestinal medications or medications that affect gastrointestinal function within the past 30 days, or who have active infections such as fever or bacteremia; 5. Patients who have taken sapropterin (KUVAN) within the 60 days prior to screening, polyethylene glycol-conjugated phenylalanine ammonia-lyase (PALYNZIQ) within the 30 days prior to screening, or who have experienced a serious immune-mediated adverse reaction to either of these drugs; 6. Individuals who test positive for hepatitis B surface antigen, hepatitis C antibodies, or syphilis antibodies; 7. History of drug or alcohol dependence; 8. Has previously received gene therapy for phenylketonuria; 9. Individuals who are allergic to Escherichia coli Nissle 1917 (EcN) or any component of the study drug; 10. Exclude participants who have participated in other interventional clinical trials and received study medication within the past 60 days; 11. The researcher determines that there are any circumstances that make participation in this study unsuitable.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Serum Phe levels;Adverse Event Monitoring; | — |
Secondary
| Measure | Time frame |
|---|---|
| Electrocardiogram ;16S rRNA sequencing;Physical Examination and Vital Signs;Complete Blood Count;Merge Medication Records;Thyroid-Stimulating Hormone, TSH;CPK nucleic acid copy number;Clinical Biochemistry;Urinalysis; | — |
Countries
China
Contacts
Public ContactHou Xinlin
Peking University First Hospital
Outcome results
None listed