Printed from Voxsanity (voxsanity.com.au) — NCT06061614 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.
Back to Phenylketonuria
Early Phase 1 Phenylketonuria Trial, Recruiting
NCT06061614
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You are willing to voluntarily sign a consent form to take part in the study.
- You have been diagnosed with Phenylketonuria (PKU) confirmed to be caused by a specific gene mutation in the PAH gene.
- You are 18 years old or older.
- Your blood phenylalanine (Phe) level has been recorded at 600 μmol/L or above at least once in the past 2 years, with one measurement confirmed within the last 6 months.
- You are able to keep your normal diet the same throughout the study and are willing to follow the study team's dietary guidance.
- You agree to check with the study doctor before taking any other medicines during the trial.
- In the study team's opinion, you are willing and able to follow all study procedures and requirements.
- If you are a woman who could become pregnant, you must have a negative pregnancy blood test within 7 days before starting, have avoided unprotected sex for at least 14 days before your first dose, and be willing to use a highly effective form of contraception for at least 12 months after receiving the study treatment.
Who may not be able to join:
- You have a high level of antibodies against a specific virus (AAV8) used in the study treatment (confirm with trial site).
- You have previously received any type of gene therapy.
- You have tested positive for Hepatitis B, Hepatitis C, HIV, or syphilis.
- Your liver function blood tests show results that are too high above the normal range (confirm with trial site).
- Your blood count results fall outside the normal range, such as low red blood cells, white blood cells, or platelets (confirm with trial site).
- Your blood sugar control levels are above a certain threshold, suggesting possible diabetes or pre-diabetes (confirm with trial site).
- You have any other significant abnormal findings in tests or physical checks that the study doctor feels make you unsuitable for the trial.
- You have any condition or reason why you should not take steroid medications (such as uncontrolled infections, epilepsy, severe bone loss, recent fractures, or allergy to steroids).
- You have a known allergy to human serum albumin, a substance used in some medicines.
- You have been diagnosed with any type of cancer, either in the past or currently.
- You have a serious condition affecting your heart, lungs, digestive system, hormones, kidneys, blood, nervous system, or mental health.
- You have a history of liver disease, such as hepatitis, liver cirrhosis, or liver cancer.
- You have taken part in another clinical trial or received a trial drug within the last 3 months.
- The study doctors have any other concerns that make them feel you are not suitable for this trial.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Early Phase 1 studies are small, early exploratory studies that come before the main Phase 1 to 3 testing, so a standard phase success rate does not apply.
Contact this trial
Phone: 008613665527160
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Status
Recruiting
Phase
Early Phase 1
Sponsor
The First Affiliated Hospital of Bengbu Medical University
Registry
ClinicalTrials.gov
Start date
30 March 2023
Est. completion
30 December 2028
Where this trial is recruiting
🇨🇳 China
Primary endpoints
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.
Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.