A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency

Launched by SHANGHAI VITALGEN BIOPHARMA CO., LTD. · May 22, 2024

Keywords

ClinConnect Summary

This clinical trial is designed to test a new treatment called VGN-R09b for children with a rare condition known as severe AADC deficiency, which affects how the brain uses certain important chemicals that help with movement and mood. The trial is in its early phase and aims to find out if VGN-R09b is safe and effective. Researchers will gradually increase the dose given to participants to determine the best amount that works without causing serious side effects.

To be eligible for this study, children must be between 18 months and 8 years old and have been diagnosed with AADC deficiency based on both their symptoms and specific genetic tests. They should also have very low levels of a certain enzyme in their blood that is related to this condition and have not had enough success with other standard treatments. Parents or guardians must agree to their child participating and provide necessary information throughout the study. It's important to note that children with certain serious health issues or those who have had previous surgeries related to their condition may not be able to join. Participants will receive close monitoring during the trial, and the goal is to see how well the treatment works and if it is safe for young patients.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. The child patient has to be ≥18 months old and \< 8 years old, and a head circumference big enough for surgery as judged by investigator.
• • 2. Historical diagnosis of AADC deficiency with clinical symptoms consistency, AND with Molecular genetic confirmation of homozygous or compound heterozygous mutation point of IVS6+4A\>T in DDC gene.
• • 3. With Plasma AADC activity less than or equal to 12 pmol/min/mL.
• • 4. Motor development at baseline \<3 months (head fully uncontrollable at baseline）, and Failed to benefit from standard medical therapy (dopamine agonists, monoamine oxidase inhibitor or related form of Vitamin B6) at discretion of investigators.
• • 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. Contraindication to sedation during surgery or imaging studies (PET or MRI).