Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05228145 | Gene Therapy Study for Children With CLN5 Batten Disease | PHASE1 | ACTIVE NOT_RECRUITING | 6 | — | — | Jan 31, 2022 | Nov 1, 2028 | Aug 12, 2024 | 2 | United States, United Kingdom |
Incidence, type, severity, and frequency of TEAEs
Incidence, type, severity, and frequency of SAEs
Incidence, type, severity, and frequency of clinical laboratory abnormalities
Incidence, type, severity, and frequency of new nerve conduction study (NCS) abnormalities
Incidence, type, severity, and frequency of new physical and neurologic exam abnormalities
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | The study treatment is a recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt). |
| Cohort 2 | EXPERIMENTAL | The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt). |
| Cohort 3 | EXPERIMENTAL | The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon- optimized human CLN5 transgene (hCLN5opt). |
| Name | Type | Description |
|---|---|---|
| NGN-101 | GENETIC | Participants with confirmed mutations in the CLN5 gene who meet all the inclusion and none of the exclusion criteria will be treated with a single intracerebroventricular (ICV) dose and a single intravitreal (IVT) dose of the study treatment. |
Inclusion Criteria * Age from 3 to 9 years (Child) * Molecular genetic diagnosis of the CLN5 gene * Confirmed clinical diagnosis of CLN5 disease * Impaired motor and/or language function and/or impaired visual acuity * Written informed consent from parent or legal guardian and assent from study par...