Actively Recruiting
A Phase I/II Study for the Safety and Efficacy of Intravenous Infusion With NGGT002 in Adults Patients With Classic Phenylketonuria
Led by NGGT (Suzhou) Biotechnology Co., Ltd. · Updated on 2024-11-14
18
Participants Needed
2
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of NGGT002, a gene therapy using a viral vector carrying the human PAH gene, in adults diagnosed with classic Phenylketonuria (PKU). This condition is marked by severe deficiency of the PAH enzyme, leading to high phenylalanine levels. The study is a Phase 1/2, open-label trial conducted at multiple centers involving adult participants with classic PKU. Participants will receive a single intravenous infusion of NGGT002 at one of three increasing dose levels. The study will enroll six to eighteen patients divided among these dose cohorts to assess safety and efficacy. After administration, participants will be monitored for five years to evaluate treatment effects and any adverse events. Throughout the study, participants will undergo regular assessments including blood phenylalanine levels, quality of life questionnaires, and safety laboratory tests. Researchers will track adverse events up to one year and changes in plasma phenylalanine concentrations at weeks 12, 28, and 52. The study includes dietary management and long-term follow-up to monitor sustained effects and participant well-being over five years.
CONDITIONS
Brief Title
Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Voluntarily participating and signing informed consent
- Any gender; must carry biallelic pathogenic or likely pathogenic variants in the PAH gene
- Adults aged 18 to 55 years
- At least two blood phenylalanine measurements ≥600 µmol/L in past 24 months, with one within 6 months before screening
- Willing and able to manage diet
- Willing and able to comply with study procedures
- Women of childbearing potential must have negative serum HCG test within 7 days before dosing and agree to use effective contraception for at least 1 year after treatment
You will not qualify if you...
- Presence of anti-AAV8 neutralizing antibodies (≥1:5)
- Disease well-controlled with existing therapies like Sapropterin Dihydrochloride or Pegvaliase-pqpz
- Elevated liver enzymes (ALT or AST >1.5×ULN), ALP >1.5×ULN, bilirubin >1.5×ULN, INR >1.5, creatinine >1.5×ULN
- Hematological values outside normal range (low hemoglobin, white blood cells, neutrophils, or platelets)
- HbA1c >6% or fasting blood glucose >6.1 mmol/L
- Significant abnormal vital signs, lab results, or findings making participation unsuitable
- Contraindications to corticosteroids or conditions worsening health
- Active or latent infections including hepatitis, HIV, syphilis, or tuberculosis
- Significant liver disease within 6 months except Gilbert's syndrome
- History of malignant tumors
- Severe liver diseases by imaging
- Serious systemic diseases before screening
- Allergy to human serum albumin
- History of substance abuse
- Previous gene therapy
- Participation in other non-gene therapy drug trials within 3 months
- Elevated alpha-fetoprotein
- Severe comorbidities associated with PKU
- Weight over 100 kg
- Excessive daily natural protein intake (>2 g/kg/day)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 52 weeks
Participants receive NGGT002 gene therapy through intravenous infusion to evaluate safety and efficacy in classic PKU.
Visits at Week 12, Week 28, and Week 52 for assessments
Trial Site Locations
Total: 2 locations
1
First Affiliated Hospital of Bengbu Medical College
Bengbu, Anhui, China
Actively Recruiting
2
Xinhua Hospital Affifiated to Shanghai Jiao Tong University School of Medicine
Shanghai, China
Actively Recruiting
Research Team
H
Huan Zhou, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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