A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants

This study will have a trial phase, extension phase, and a long-term extension phase. The primary objectives of the trial phase are to assess the pharmacodynamics (PD) of eladocagene exuparvovec treatment by evaluation of homovanillic acid (HVA) levels and to assess the safety of the SmartFlow® magnetic resonance (MR) Compatible Ventricular Cannula for administering eladocagene exuparvovec to pediatric participants with aromatic L-amino acid decarboxylase (AADC) deficiency. The extension phase is designed to capture additional clinical information for eladocagene exuparvovec through study evaluations, changes in motor development, AADC-specific symptoms, and other PD measures. The long-term extension phase is designed to capture long-term safety and efficacy data from participants treated with eladocagene exuparvovec.

PTC Identifier:
PTC-AADC-GT-002
ClinicalTrials.gov Identifier:
NCT04903288
EudraCT Identifier:
N/A
Not Recruiting

Trial Purpose

Age
1 - 17 Years
Sex
Both (Female and Male)
Healthy Volunteers
No

Key Participation Requirements

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.

Exclusion Criteria:

  • The participant has presence of other significant medical or neurological conditions that would create an unacceptable operative or anesthetic risk.
  • Participants with pyridoxine 5'-phosphate oxidase or tetrahydrobiopterin (BH4) deficiency.

Trial Summary

Total Participants

This is the number of individuals who are taking part in the trial.

Total Participants
13

Trial Dates

These are the start and end dates for the trial. Note that future dates are estimates that may be updated before or during the trial.

Trial Dates
May 2021 - May 2023

Phase

These are the different steps that a treatment goes through during clinical development before it is approved for use.

Phase
Phase 2

Placebo Controlled

In this type of study, some participants receive a “placebo,” which looks like the study treatment, but does not actually contain the active treatment.

Placebo Controlled
No

Product

This is any type of drug, device, or other treatment that is being studied in the trial.

Product
N/A

Accepts Healthy Volunteers

Healthy volunteers are individuals who participate in the trial and do not have that specific disease or condition.

Accepts Healthy Volunteers
No
Discuss with the trial center for details

Trial Design

Trial Type

This is the general nature of the study.

Trial Type
Interventional

Interventional

Participants in these studies are subject to an intervention by researchers. These trials are used to understand the effects of a particular intervention.

Discuss with the trial center for details

Blinding

Study participants know which treatments they might get if they join the trial, however during a trial they may not be informed which treatment they are actually taking. The act of not disclosing this information to the participant is known as “blinding.”

Blinding
N/A
Discuss with the trial center for details

Intervention Type

This is a medicine, device, or other type of treatment that is being studied or is already approved for use. Interventions can also include behavior changes, like diet and exercise.

Intervention Type
Genetic

Allocation

This determines how study participants are assigned their treatment.

Allocation
N/A

Trial Purpose

This is the rationale behind why the clinical trial is being performed.

Trial Purpose
Treatment

Trial Arms

These are the groups of participants in a clinical study. Each different “arm” is assigned a different treatment to use. Different types of trial arms in studies can include:

Trial Arms
Experimental

Experimental arm

Experimental arm: The group assigned to use the study treatment

Primary Outcome

Primary Outcome

This is the most important measurement of the study and is used to help researchers evaluate the effect of a treatment.

Change From Baseline in HVA Metabolite Levels at the End of the Trial Phase

Timeframe: Baseline (Day 1), Week 8

Number of Participants With Adverse Events (AEs) Associated With the Surgical Administration of Eladocagene Exuparvovec Using the SmartFlow® MR-Compatible Ventricular Cannula

Timeframe: Baseline (Day 1) up to Week 8

Secondary Outcome

Secondary Outcome

This measurement is a secondary measurement and is still important in helping researchers evaluate the effect of a treatment.

Change From Baseline in Neurotransmitter Cerebrospinal Fluid (CSF) Metabolite HVA at Week 48

Timeframe: Baseline (Day 1), Week 48

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) and the Extension Phase (Week 48)

Timeframe: Baseline (Day 1), Week 8, Week 48

Change From Baseline in Neurotransmitter CSF Metabolites 5-hydroxyindoleacetic acid (5-HIAA), and 3-O-methyldopa (3-OMD) at Weeks 8 and 48

Timeframe: Baseline (Day 1), Weeks 8 and 48

Number of Participants who Attain Motor Milestones

Timeframe: Baseline (Day 1) up to Week 260

Change in Peabody Developmental Motor Scale, Second Edition (PDMS-2)

Timeframe: Baseline (Day 1), Week 260

Change in Bayley Scale of Infant Development, Third Edition (Bayley-III)

Timeframe: Baseline (Day 1), Week 260

Change in EuroQol-5 Dimensions Youth Version (EQ-5D-Y)

Timeframe: Baseline (Day 1), Week 260

Change in Body Weight

Timeframe: Baseline (Day 1), Week 260

Number of Participants With AADC-Specific Symptoms

Timeframe: Baseline (Day 1) up to Week 260

Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

Timeframe: Baseline (Day 1) up to Week 260

Contact Information

Location
Status
Location
Texas Children’s Hospital
Houston, TX, United States, 77030
Status
Active, not recruiting
Location
Boston Children’s Hospital
Boston, MA, United States, 02115
Status
Recruiting
Location
Duke University Hospital
Durham, NC, United States, 27705
Status
Recruiting
Location
Cincinnati Children’s Hospital Medical Center
Cincinnati, OH, United States, 45229
Status
Recruiting
Location
Chaim Sheba Medical Center
Ramat Gan, Israel, 5262000
Status
Recruiting
Location
National Taiwan University Hospital, Department of Pediatrics and Medical Genetics
Taipei, Taiwan, 10041
Status
Recruiting