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Eladocagene Exuparvovec · 1 trial · 1 indication
HVA is a main metabolite of dopamine and HVA CSF levels are recognized as a proxy for dopamine levels in the brain.
An AE is any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered related to the drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease in a study participant who was administered gene therapy in this study. Number of participants with AEs related to the SmartFlow MR-compatible ventricular cannula used to administer eladocagene exuparvovec to pediatric participants at the end of Trial Phase (8 weeks after administration) are reported. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module."
| Arm | Type | Description |
|---|---|---|
| Eladocagene Exuparvovec | EXPERIMENTAL | Participants will receive eladocagene exuparvovec intraoperatively at 1.8×10\^11 vector genomes (vg) via SmartFlow® MR Compatible Ventricular Cannula in a single operative session. Participants will receive standard of care for their AADC deficiency during the study. |
| Name | Type | Description |
|---|---|---|
| Eladocagene Exuparvovec | GENETIC | Four 0.08 milliliters (mL) infusions at a dose of 0.45×10\^11 vg and a volume of 80 microliters (μl) per site to 4 sites (2 per putamen), for the total dose of 1.8×10\^11 vg and a total volume of 320 μl per participant. |
Inclusion Criteria: * Pediatric participants must have genetically-confirmed AADC deficiency with typical clinical characteristics and decreased AADC enzyme activity in plasma. * Cranium sufficiently developed to allow placement of ClearPoint® system for stereotactic surgery. * Persistent neurologi...
Eladocagene Exuparvovec is an investigational gene therapy being developed for the treatment of AADC deficiency, a rare genetic disorder. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
Eladocagene Exuparvovec is a gene therapy that belongs to the gene therapy class of treatments. It is designed to address the underlying genetic cause of AADC deficiency by delivering a functional gene to affected cells.
Eladocagene Exuparvovec is being developed by PTC Therapeutics, Inc., a biopharmaceutical company. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational gene therapy for AADC deficiency.
Eladocagene Exuparvovec is currently in Phase 2 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory agencies and is still being studied in clinical trials.
Eladocagene Exuparvovec is being studied in a Phase 2 clinical trial identified as NCT04903288. This trial is evaluating the use of a SmartFlow Magnetic Resonance compatible ventricular cannula for administering the drug to pediatric participants with AADC deficiency.
Eladocagene Exuparvovec is a distinct investigational gene therapy developed by PTC Therapeutics. It is not known to be the same as any other approved or marketed drug, and it is being studied specifically for AADC deficiency.