NCT05765981 An Early Clinical Trial to Evaluate VGN-R09b for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency.
| NCT ID | NCT05765981 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | Shanghai Jiao Tong University School of Medicine |
| Condition | Aromatic L-amino Acid Decarboxylase (AADC) Deficiency |
| Study Type | INTERVENTIONAL |
| Enrollment | 6 participants |
| Start Date | 2023-01-30 |
| Primary Completion | 2025-02-20 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 6 participants in total. It began in 2023-01-30 with a primary completion date of 2025-02-20.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This early Phase trial is to prove the safety and efficacy of VGN-R09b to treat patients with AADC deficiency.
Eligibility Criteria
Inclusion Criteria: 1. The child patient has to be ≥2 years old and \< 8 years old, or a head circumference big enough for surgery. 2. Historical diagnosis of AADC deficiency with clinical symptoms consistency, AND confirmed by one of the lab tests: (1) CSF neurotransmitter profile demonstrating reduced HVA and 5-HIAA, and elevated L-Dopa concentrations; (2) Plasma AADC activity less than or equal to 5 pmol/min/mL, AND with Molecular genetic confirmation of homozygous or compound heterozygous mutation of IVS6+4A\>T in DDC. 3. Motor development at baseline ≤3 months, and Failed to benefit from standard medical therapy (dopamine agonists, monoamine oxidase inhibitor or related form of Vitamin B6) at discretion of investigators. 4. Stable medication regimen for treatment of AADC deficiency: (i.e. no new medications introduced for at least 6 months, and no existing medication dose changes for at least 3 months prior to Baseline). 5. Parent(s)/legal guardian(s) with custody of subject must give their consent for subject to enroll in the study 6. Parent(s)/legal guardian(s) of the subject must agree to comply with the requirements of the study, including providing disease information and support disease assessment of symptoms. Exclusion Criteria: 1. Intracranial neoplasm or any structural brain abnormality or lesion (e.g., severe brain atrophy, white matter degenerative changes), which, in the opinion of the study investigators, would confer excessive risk and/or inadequate potential for benefit. 2. Presence of other significant medical or neurological conditions that would create an unacceptable operative or anesthetic risk (including congenital heart disease, respiratory disease with home oxygen requirement, history of serious anesthesia complications during previous elective procedures, history of cardiorespiratory arrest), liver or renal failure, malignancy, or HIV positive. 3. Severe coagulopathy, or need for ongoing anticoagulant therapy. 4. clinically active infection or with severe infection within 12 weeks before screening (e.g. adenovirus or herpes virus, pneumonia, sepsis, central nervous system infection). 5. Previous stereotactic neurosurgery, or any gene/cell therapy. 6. Received live vaccination within 4 weeks. 7. Patients with anti-AAV9 neutralizing antibody titer over 1,200 folds. 8. Contraindication to sedation during surgery or imaging studies (PET or MRI).
Contact & Investigator
Jiwen Wang, Doctor
STUDY CHAIR
Shanghai Children's Medical Center, affiliated to Shanghai Jiao Tong University School of Medicine
Frequently Asked Questions
Who can join the NCT05765981 clinical trial?
This trial is open to participants of all sexes, aged 24 Months or older, up to 7 Years, studying Aromatic L-amino Acid Decarboxylase (AADC) Deficiency. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05765981 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05765981 currently recruiting?
Yes, NCT05765981 is actively recruiting participants. Contact the research team at wangjiwen@scmc.com.cn for enrollment information.
Where is the NCT05765981 trial being conducted?
This trial is being conducted at Shanghai, China.
Who is sponsoring the NCT05765981 clinical trial?
NCT05765981 is sponsored by Shanghai Jiao Tong University School of Medicine. The principal investigator is Jiwen Wang, Doctor at Shanghai Children's Medical Center, affiliated to Shanghai Jiao Tong University School of Medicine. The trial plans to enroll 6 participants.