NCT05948020

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

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

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Children's Hospital of Fudan University · Academic / Other
• Sex: All
• Age: 3 Years – 17 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Due to an increased blood phenylalanine (Phe) concentration, untreated children with PKU will develop progressively intellectual disability. Engineered probiotics can metabolize Phe into other products in the intestine by expressing related exogenous proteins in the Phe metabolic pathway, thereby reducing Phe concentration in the intestine and blood. Animal experiments have confirmed the efficacy and safety of CBT102-A. This study will enroll children with PKU age 3 to 17 years according to a strict inclusion and exclusion criteria. Subjects who meet the requirements will be randomly assigned on Day 1 and start the study administration. The administration period of both groups is 20 days (Day 1\~Day 20), in which the experimental group receives CBT102-A with 4 dose levels, and the control group receives placebo administration, with the same mode, frequency, time, cycle and dose as the experimental group. 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）. Change of blood Phe concentration and occurrence of Treatment-Emergent Adverse Events(TEAE) with PKU will be used to evaluate the efficacy and safety of the CBT102-A.

Conditions

• Phenylketonuria

Interventions

Type	Name	Description
BIOLOGICAL	CBT102-A capsule	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）.
OTHER	Placebo capsule	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）.

Timeline

• Start date: 2023-09-02
• Primary completion: 2023-11-25
• Completion: 2024-03-07
• First posted: 2023-07-17
• Last updated: 2026-02-27

Locations

1 site across 1 country: China