Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria：a Randomized, Double-blind, Placebo-controlled, Parallel-group Clinical Study

CBT-102

This is a randomized, double-blind, placebo-controlled, parallel-group study. A total of 15 children with phenylketonuria(PKU) age 3 to 17 years will be randomized to two groups. Experimental group of 10 children will intervene engineered probiotics (CBT102-A) for 20 days and 5 children will intervene placebo. The goal of this study is to determine whether CBT102-A is an effective and safe treatment for PKU.

2023-09-02

2023-12-01

2024-03-01

Recruiting

15

Inclusion Criteria: * Blood phe ≥ 600μmol/L at newborn screening; * Blood phe ≥ 600μmol/L at least 3 times in the last 1 year before screening, and the blood Phe ≥ 600μmol/L in the last 1 time; * Screening laboratory evaluations (e.g., chemistry panel, complete blood count, urinalysis, creatinine clearance, CRP) within normal limits or judged to be not clinically significant by the investigator; * Stable diet for at least 60 days prior to screening; * Able to produce at least 2 bowel movements per week on average without using any form of laxatives; * Adolescents and children's guardians can voluntarily complete the whole process of informed consent, including stool, urine and blood collection, adherence to diet control, hospital monitoring, follow-up and oral trial drug compliance, and sign informed consent. Exclusion Criteria: * The standard percentile values of height and weight of Chinese children aged 0 to 18 years were evaluated with weight less than P3 or weight greater than P97； * History of active or chronic passage of 3 or more loose stools per day； * Have any medical conditions or medications that may affect the absorption of medications or nutrients； * History of or current immunodeficiency disorder including autoimmune disorders； * Subjects with obvious influenza-like symptoms caused by COVID-19 or other viral infections during screening； * Hepatitis B surface antigen and/or hepatitis C antibodies and/or treponema pallidum antibodies positive； * Subjects who are dependent on drugs and alcohol； * Received gene therapy related to PKU； * Intolerant or allergic to Escherichia coli Nissle 1917 (EcN)； * Active gastrointestinal bleeding or a proven history of gastrointestinal bleeding within 60 days prior to screening； * Antibiotics within 28 days before the planned first dose of investigational product (IP), or anticipated during the study period； * Take probiotic supplements within 28 days before the planned first dose of IP, or anticipated during the study period； * A history of fever, confirmed bacteremia, or other active infection within 30 days prior to the planned first dose of IP； * Drugs that use of the digestive system has been used within 30 days prior to the planned first dose of IP; * Drugs that may affect gastrointestinal function has been used within 30 days prior to the planned first dose of IP; * Major survery performed within 90 days before the anticipated first dose of IP or planned surgery or hospitalization during the study period； * Take sapropterin (KUVAN®) within 1 week before the planned first dose of IP； * Use pegylated recombinant phenylalanine ammonia lyase (PALYNZIQ™) within 30 days before the planned first dose of IP； * History of severe immune adverse reactions to PALYZIQ； * Participated in an interventional clinical trial and used the investigational drug within 60 days or 5 half-lives before the planned first dose of IP； * Subjects who may not be able to complete the study for other reasons.

{'studyType': 'INTERVENTIONAL', 'phases': ['NA'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'TRIPLE', 'maskingDescription': "Masking to the investigation sites and subjects (including subjects' guardians)", 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 15, 'type': 'ESTIMATED'}}

2023-09-08