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

  • End date
    Jul 15, 2023
  • participants needed
  • sponsor
    PTC Therapeutics
Updated on 12 August 2022


This study will have a trial phase and an extension phase. The primary objective of the trial phase is to assess the safety of the SmartFlow® 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, including changes in motor development, AADC-specific symptoms, and other pharmacodynamic (PD) measures.

Condition AADC Deficiency
Treatment Eladocagene Exuparvovec
Clinical Study IdentifierNCT04903288
SponsorPTC Therapeutics
Last Modified on12 August 2022


Yes No Not Sure

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 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
Unable to ambulate independently (with or without assistive device)
Baseline hematology, chemistry, and coagulation values within the normal pediatric laboratory value ranges, unless in the investigator's opinion the out of range values are not clinically significant with respect to the participant's suitability for surgery
Participant must test negative for coronavirus disease of 2019 (COVID-19) a maximum of 72 hours prior to receiving gene therapy
Females of childbearing potential must have a negative pregnancy test at screening and baseline and agree to abstinence or double-barrier form of contraception for the duration of the study following discharge from the hospital (acceptable methods will be determined by the site)
Males sexually active with females of childbearing potential must agree to use a barrier method of birth control during the study following discharge from the hospital
Parent(s)/legal guardian(s) of the participant must agree to comply with the requirements of the study, including the need for frequent and prolonged follow up
Parent(s)/legal guardian(s) with custody of the participant must give their consent for the participant to enroll in the study

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
Contraindication for imaging studies (computed tomography [CT] scan, PET, or magnetic resonance imaging [MRI]), including sedation limitations or metal that would interfere with a brain MRI
Anti-adeno-associated virus, serotype 2 (anti-AAV2) antibody titer higher than 1:1200 or >1 optical density value by enzyme-linked immunosorbent assay
Participants who have received treatment with other experimental therapies within the last 24 weeks prior to planned gene therapy administration, or any treatment ever with a gene therapy
Evidence of a clinically active infection
Females who are pregnant or breast feeding
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