Mucopolysaccharidosis Type IIIB (MPS IIIB)
Conditions
Interventions
Experimental Group:Intracerebroventricular Injection of RDGT-101 Injection
Sponsors
The Seventh Medical Center of Chinese PLA General Hospital
Eligibility
Sex/Gender
All
Age
No minimum to 12 Years
Inclusion criteria
Inclusion criteria: 1. Age = 30 (assessed using the Infant-Child Mental Development Scale), indicating early-stage disease; 5. The patient and/or the patient’s guardian is capable of understanding and willing to comply with the study protocol requirements and procedures, voluntarily agrees to participate, and provides signed informed consent.
Exclusion criteria
Exclusion criteria: 1. Rapid disease progression or end-stage disease, such as severe visual or hearing impairment precluding neurodevelopmental testing, poorly controlled seizures, or loss of independent walking ability; 2. Evidence of symptomatic improvement of MPS IIIB; 3. Presence of central nervous system damage or behavioral disorders unrelated to MPS IIIB that may affect the interpretation of study results; 4. Prior history of hematopoietic stem cell transplantation, current participation in other clinical trials, or previous use of other cell/gene therapy products; 5. Serum anti-AAV9 neutralizing antibody titer >.= 1:100 during the screening period; 6. Hematologic abnormalities, including: absolute neutrophil count (ANC) = 3×upper limit of normal (ULN), total bilirubin (TBil) >= 1.5×ULN, creatinine (CRE) >= 1.5×ULN; 8. Cardiomyopathy or significant congenital heart abnormalities; 9. Clinically significant active bacterial, viral, fungal, parasitic, or prion-related infections; 10. Contraindications to corticosteroid use, such as severe hypertension, diabetes, systemic fungal infection, glaucoma, osteoporosis, gastric ulcer, tuberculosis, etc.; 11. Any condition preventing enhanced MRI examination (including allergy to anesthetics or contrast agents); 12. Contraindications to intracerebroventricular injection or lumbar puncture; 13. Any medical condition that may interfere with result interpretation or subject safety, including but not limited to organ dysfunction, acute infectious diseases, primary/acquired immunodeficiency, severe cardiovascular/cerebrovascular or gastrointestinal diseases, diabetes, history of meningitis or family history of psychiatric disorders, cerebrospinal fluid circulation disorders, etc.; 14. Positive for HIV antibody, hepatitis B surface antigen, hepatitis C antibody, or Treponema pallidum antibody; 15. Vaccination within 2 weeks prior to dosing; 16. Any other condition considered by the investigator as unsuitable for study participation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of adverse events within 52 weeks following a single intracerebroventricular injection of RDGT-101.; | — |
Secondary
| Measure | Time frame |
|---|---|
| Changes in biomarkers within 52 weeks post-dose (HS in plasma, urine, and CSF; NAGLU enzyme activity in plasma and CSF).;Changes in neurocognitive function and quality of life from baseline at 52 weeks post-dose (including scores for Developmental Quotient [DQ], Intelligence Quotient [IQ], cognitive age, adaptive age equivalent, and the total score of the Pediatric Quality of Life Inventory? [PedsQL?] Generic Core Scales).;Pharmacokinetic profile within 52 weeks post-dose (copy number of the target gene in blood, urine, saliva, and stool).;Drug immunogenicity (serum levels of anti-AAV9 and anti-NAGLU protein antibodies at baseline and within 52 weeks post-dose).; | — |
Countries
China
Contacts
Public ContactZhichun Feng
The Seventh Medical Center of Chinese PLA General Hospital
Outcome results
None listed