Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as KEBILIDI™
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 studied 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. The therapy is designed to address the underlying genetic cause of the condition.
Eladocagene Exuparvovec targets the DDC gene, which provides instructions for making the enzyme aromatic L-amino acid decarboxylase. By delivering a functional copy of this gene, the therapy aims to restore enzyme activity in patients with AADC deficiency. It is classified as an exogenous gene therapy.
Eladocagene Exuparvovec is being developed by PTC Therapeutics, Inc., a biopharmaceutical company. PTC Therapeutics is publicly traded on the NASDAQ under the ticker symbol PTCT. The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for AADC deficiency.
Eladocagene Exuparvovec is currently in Phase 2 clinical development. It is an investigational therapy, meaning it has not yet received regulatory approval for commercial use. The ongoing Phase 2 study is evaluating the delivery of the therapy using a specialized cannula in pediatric participants with AADC deficiency.
Eladocagene Exuparvovec is being studied in a Phase 2 clinical trial with the identifier NCT04903288. This trial is titled 'A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants.' The study is active but not recruiting, with an enrollment of 13 participants.
Eladocagene Exuparvovec is also known by the brand name Upstaza in some regions, though this information is not provided in the current data. The drug is being developed under the name Eladocagene Exuparvovec by PTC Therapeutics for the treatment of AADC deficiency.