Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria

NCT05948020 | Completed Early Phase 1

• 1 other identifier: CBT-102
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Phenylketonuria

Outcome Measures

Primary Outcomes (1)

• Changes from Baseline in Blood Phe Concentration

  This is a repeatedly measures outcome; Blood Phe concentration will be detected at baseline, administration(Day 1\~Day 20), observation(Day 21\~Day 23) and follow-up periods(Day 51); During the administraion time, Phe concentrations will be measured at 4 hours after each day on Day 4,Day 8, Day 12, Day 15, Day 18, Day 20; A drop of blood will be collected from the end of the finger on the filter paper and will be detected by tandem mass spectrometry.

  From baseline to Day 51

Secondary Outcomes (5)

• Occurrence of Treatment-Emergent Adverse Events（TEAE）≥2 Grade

  From baseline to Day 23

• Occurrence of TEAE

  From baseline to Day 51

• Changing Value of Subjects Urinary Metabolites

  From baseline to Day 23

• Changing Value of Subjects Fecal Metabolites

  From baseline to Day 23

• Clearance of CBT102-A from Fecal

  From baseline to Day 23

Study Arms (2)

CBT102-A group

EXPERIMENTAL

10 subjects receive oral CBT102-A with three meals per day for a total of 20 days

Biological: CBT102-A capsule

Placebo group

PLACEBO COMPARATOR

5 subjects receive oral placebo with three meals per day for a total of 20 days

Other: Placebo capsule

Interventions

CBT102-A capsule BIOLOGICAL

Orally CBT102-A will be supplied by CommBio Therapeutics. It is an enteric-coated capsule with 1.25×10\^11 live cell. The shelf life is 6 months. Subjects receive oral dose of 1 capsule CBT102-A (1.25 x 10\^11 live cell) before three meals per day on Day 1 to Day 4; Subjects receive oral dose of 2 capsule CBT102-A (2.5 x 10\^11 live cell) before three meals per day on Day 5 to Day 8; Subjects receive oral dose of 4 capsule CBT102-A (5 x 10\^11 live cell) before three meals per day on Day 9 to Day 12; Subjects receive oral dose of 8 capsule CBT102-A (1 x 10\^12 live cell) before three meals per day on Day 13 to Day 20; All subjects will be observed for 3 days (Day 21\~Day 23) without intervene in hospital and will be followed up weekly for 4 consecutive weeks after discharge（Day 51）.

CBT102-A group

Placebo capsule OTHER

Orally placebo will be supplied by CommBio Therapeutics. It is an enteric-coated capsule with Lactose powder filler. The shelf life is 6 months. The color, condition, smell and other appearances are exactly the same as CBT102-A. Subjects receive oral dose of 1 capsule placebo before three meals per day on Day 1 to Day 4; Subjects receive oral dose of 2 capsule placebo before three meals per day on Day 5 to Day 8; Subjects receive oral dose of 4 capsule placebo before three meals per day on Day 9 to Day 12; Subjects receive oral dose of 8 capsule placebo before three meals per day on Day 13 to Day 20; All subjects will be observed for 3 days (Day 21\~Day 23) without intervene in hospital and will be followed up weekly for 4 consecutive weeks after discharge（Day 51）.

Placebo group

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead
• CommBio Therapeutics limited company: collaborator

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, China

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Wenhao Zhou

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

• Huijun Wang

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

• Haitao Zhu

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

• Yajie Su

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Masking to the investigation sites and subjects (including subjects' guardians)
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 9, 2023
• First Posted: July 17, 2023
• Study Start: September 2, 2023
• Primary Completion: November 25, 2023
• Study Completion: March 7, 2024
• Last Updated: February 27, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)