Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 11:28 PM
Ignite Modification Date:
2025-12-24 @ 11:28 PM
NCT ID:
NCT03952156
Eligibility Criteria:
Key Inclusion Criteria:
* Adults 18-55 years of age at the time of informed consent
* Diagnosis of phenylketonuria (PKU) due to PAH deficiency
* Two plasma Phe values with a concentration of ≥ 600 μmol/L drawn at least 72 hours apart during the screening period and at least one historical value ≥ 600 μmol/L in the preceding 24 months.
* Subject has the ability and willingness to maintain their baseline diet, whether Phe-restricted or unrestricted for the duration of the trial, unless otherwise directed
Key Exclusion Criteria:
* Subjects with PKU that is not due to PAH deficiency
* Presence of anti-AAVHSC15 neutralizing antibodies
* ALT \> ULN and AST \> ULN
* Alkaline phosphatase \> ULN.
* Total bilirubin \> ULN, direct bilirubin \> ULN
* Serum creatinine \>1.5x ULN
* International normalized ratio (INR) \> 1.2
* Hematology values outside of the normal range (hemoglobin \<11.0 g/dL for males or \<10.0 g/dL for females; white blood cells (WBC) \<3,000/μL; absolute neutrophils \<1500/μL; platelets \<100,000/μL)
* Hemoglobin A1c \>6.5% or fasting glucose \>126 mg/dL
* Any clinically significant abnormal laboratory result at screening, in the opinion of the Investigator
* Contraindication to corticosteroid use or conditions that could worsen in the presence of corticosteroids, as assessed and determined by the investigator
* Previously received gene therapy for the treatment of any condition.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
18 Years
Maximum Age:
55 Years
Study:
NCT03952156
Study Brief:
Protocol Section:
NCT03952156