PhenylketonuriaEarly Phase 1NCT05948020
Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria
A Early Phase 1 study testing CBT102-A capsule in people with Phenylketonuria, run by Children's Hospital of Fudan University at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Phenylketonuria
- What is being tested?
- CBT102-A capsule
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteChina
- Study dates
- 2 Sep 2023to 7 Mar 2024
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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；
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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
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.
Read the 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
- Being tested
- CBT102-A capsule, Placebo capsule
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CBT102-A capsuleBiological
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）.
Placebo capsuleOther
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）.
| Study group | Role | What they receive |
|---|---|---|
| CBT102-A group | Experimental | 10 subjects receive oral CBT102-A with three meals per day for a total of 20 days Receives: Biological: CBT102-A capsule |
| Placebo group | Placebo comparator | 5 subjects receive oral placebo with three meals per day for a total of 20 days Receives: Other: Placebo capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From baseline to Day 51
Secondary outcomes (5)
Occurrence of Treatment-Emergent Adverse Events（TEAE）≥2 Grade
The grade of TEAE will be assessed according to Toxicity is graded in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0; TEAE ≥ 2 grade define as occurrence of age-appropriate limitations in instrumental activities of daily living; Adverse events require medical intervention.
Measured: From baseline to Day 23
Occurrence of TEAE
All treatment emerged adverse events during the whole study
Measured: From baseline to Day 51
Changing Value of Subjects Urinary Metabolites
This is a repeatedly measures outcome; During the administraion time, subjects' urine will be collected after each day on Day 8, Day 12, Day 20 and Day 23; The changes of metabolites will be detected by tandem mass spectrometry.
Measured: From baseline to Day 23
Changing Value of Subjects Fecal Metabolites
This is a repeatedly measures outcome; During the administraion time, subjects' fecal will be collected after each day on Day 8, Day 12, Day 20 and Day 23; The changes of metabolites will be detected by tandem mass spectrometry.
Measured: From baseline to Day 23
Clearance of CBT102-A from Fecal
This is a repeatedly measures outcome; CBT102-A transit through the gastrointestinal tract will be measured with fecal quantitative polymerase chain reaction (qPCR) assays from fecal samples collected at baseline,Day 20 and Day 23 during the dosing period; CBT102-A clearance reflects a test value of below the limit of quantitation (BLQ) occurring after the indicated number of days following the last dose of investigational product.
Measured: From baseline to Day 23
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2023
- Study start2 Sep 2023
- Primary completion25 Nov 2023
- Study completion7 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriaStudies of the same condition, open and under way.
- More trials run by Children's Hospital of Fudan UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Fudan University
- Sponsor type
- University, hospital or other
- Collaborators
- CommBio Therapeutics limited company
- Responsible party
- Sponsor
- Investigators
- Wenhao Zhou, Principal investigator, Children's Hospital of Fudan UniversityHuijun Wang, Principal investigator, Children's Hospital of Fudan UniversityHaitao Zhu, Principal investigator, Children's Hospital of Fudan UniversityYajie Su, Principal investigator, Children's Hospital of Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05948020
- Sponsor's protocol ID
- CBT-102
- Status last verified
- Feb 2026
- First submitted
- 9 Jul 2023
- Last updated
- 27 Feb 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05948020
- Acronym / sponsor ID
- CBT-102
- Conditions
- Phenylketonuria
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Children's Hospital of Fudan UniversityUniversity, hospital or other
- Collaborators
- CommBio Therapeutics limited company
- Study start
- 2 Sep 2023 (actual)
- Primary completion
- 25 Nov 2023 (actual)
- Study completion
- 7 Mar 2024 (actual)
- First posted
- 17 Jul 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CBT102-A group | Experimental | Biological: CBT102-A capsule10 subjects receive oral CBT102-A with three meals per day for a total of 20 days |
| Placebo group | Placebo comparator | Other: Placebo capsule5 subjects receive oral placebo with three meals per day for a total of 20 days |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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;
Key exclusion criteria (21 listed)
- 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；
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CBT102-A capsuleOrally 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）. | Biological | CBT102-A group |
| Placebo capsuleOrally 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）. | Other | Placebo group |
Primary outcome
Changes from Baseline in Blood Phe Concentration
Time frame: From baseline to Day 51
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.
5 secondary outcomes
Occurrence of Treatment-Emergent Adverse Events（TEAE）≥2 Grade
Time frame: From baseline to Day 23
Occurrence of TEAE
Time frame: From baseline to Day 51
Changing Value of Subjects Urinary Metabolites
Time frame: From baseline to Day 23
Changing Value of Subjects Fecal Metabolites
Time frame: From baseline to Day 23
Clearance of CBT102-A from Fecal
Time frame: From baseline to Day 23
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wenhao Zhou principal investigator
Children's Hospital of Fudan University
Huijun Wang principal investigator
Children's Hospital of Fudan University
Haitao Zhu principal investigator
Children's Hospital of Fudan University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing CBT102-A capsule in people with Phenylketonuria, run by Children's Hospital of Fudan University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes from Baseline in Blood Phe Concentration (time frame: From baseline to Day 51). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Feb 2026.