Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria
Phenylketonuria
About this trial
This is an interventional treatment trial for Phenylketonuria
Eligibility Criteria
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.
Sites / Locations
- Children's Hospital of Fudan UniversityRecruiting
Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
CBT102-A group
Placebo group
10 subjects receive oral CBT102-A with three meals per day for a total of 20 days
5 subjects receive oral placebo with three meals per day for a total of 20 days