Eladocagene Exuparvovec for AADC Deficiency

Phase-Based Progress Estimates
AADC Deficiency
Eladocagene Exuparvovec - Genetic
< 18
All Sexes
What conditions do you have?

Study Summary

This trial will assess the safety of using a SmartFlow cannula to administer eladocagene exuparvovec to children with AADC deficiency. The extension phase will capture additional information on how the drug affects motor development, symptoms, and other measures.

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 1 Secondary · Reporting Duration: Baseline (Day 1), Week 8

Week 8
Number of Participants With Adverse Events (AEs) Associated With the Surgical Administration of Eladocagene Exuparvovec Using the SmartFlow® MR Compatible Ventricular Cannula
Week 8
Change From Baseline in Positron Emission Tomography (PET) Imaging of Putaminal-Specific L-6-[18F] Fluoro-3,4-Dihydroxyphenylalnine (18F-DOPA) PET Uptake at the End of the Trial Phase (Week 8)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

1 Treatment Group

Eladocagene Exuparvovec
1 of 1
Experimental Treatment

3 Total Participants · 1 Treatment Group

Primary Treatment: Eladocagene Exuparvovec · No Placebo Group · Phase 2

Eladocagene Exuparvovec
Experimental Group · 1 Intervention: Eladocagene Exuparvovec · Intervention Types: Genetic

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline (day 1), week 8

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor
70 Previous Clinical Trials
6,237 Total Patients Enrolled

Eligibility Criteria

Age < 18 · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
The cranial vault is sufficiently developed to allow placement of the ClearPoint® system for stereotactic surgery.
You have persistent neurological defects secondary to AADC deficiency despite standard medical therapy (dopamine agonists, monoamine oxidase inhibitor, pyridoxine, or other forms of vitamin B6) in the opinion of the investigator.
You are unable to walk without assistance.
You are a male who is sexually active with females of childbearing potential

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 28th, 2021

Last Reviewed: October 30th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.