Evidence map›Paper›PMID 41724580›Full record

Trial reportJournal of inherited metabolic disease2026

Pharmacodynamics, Efficacy, and Safety of Intraputaminal Eladocagene Exuparvovec Administered to Pediatric Patients With Aromatic L-Amino Acid Decarboxylase Deficiency Using an MR-Compatible Cannula: 48 Weeks of Follow-Up.

Daniel J Curry, Phillip L Pearl, Scellig S D Stone, Donald L Gilbert, Sudhakar Vadivelu, Bruria Ben-Zeev, Matthew Vestal, Muhammad Zafar, Chun-Hwei Tai, Sheng-Che Chou and 13 more

Registry-linked trialAbstract readClinical Trial, Phase IIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Journal of inherited metabolic disease, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04903288 (An Open-Label Trial to Address the Safety of the SmartFlow MR-Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Subjects), which is not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT04903288 phase2active not recruitingnot on this map

An Open-Label Trial to Address the Safety of the SmartFlow MR-Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Subjects

TypeinterventionalSponsorPTC TherapeuticsRan2021 to 2028Enrolled13ConditionsAADC DeficiencyArmsEladocagene Exuparvovec
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Trial
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

23 authors.

Daniel J CurrySection of Pediatric Neurosurgery, Department of Neurosurgery, Baylor College of Medicine, Houston, Texas, USA.
Phillip L PearlBoston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0002-6373-1068
Scellig S D StoneBoston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Donald L GilbertDivision of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.ORCID https://orcid.org/0000-0002-9245-6878
Sudhakar VadiveluDivision of Neurosurgery, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Bruria Ben-ZeevSackler School of Medicine, Tel Aviv University, Tel Aviv, Israel.
Matthew VestalDepartment of Pediatric Neurosurgery, Dartmouth Geisel School of Medicine and Dartmouth Hitchcock Medical Center, Lebanon, New Hampshire, USA.
Muhammad ZafarDepartment of Pediatrics and Neurology, Duke University Hospital, Durham, North Carolina, USA.ORCID https://orcid.org/0009-0007-1677-3955
Chun-Hwei TaiDepartment of Neurology, National Taiwan University Hospital, Taipei, Taiwan.
Sheng-Che ChouDivision of Neurosurgery, Department of Surgery, National Taiwan University Hospital, Taipei, Taiwan.
Zion ZiblyDepartment of Neurosurgery, Sheba Medical Center, Ramat Gan, Israel.
Lior UngarDepartment of Neurosurgery, Sheba Medical Center, Ramat Gan, Israel.
Mered ParnesDepartment of Pediatrics, Division of Neurology and Developmental Neuroscience, Baylor College of Medicine, Houston, Texas, USA.ORCID https://orcid.org/0000-0003-2102-4282
Mariam HullDepartment of Pediatrics, Division of Neurology and Developmental Neuroscience, Baylor College of Medicine, Houston, Texas, USA.ORCID https://orcid.org/0000-0002-7340-1660
Lisa EmrickDepartment of Pediatrics, Division of Neurology and Developmental Neuroscience, Baylor College of Medicine, Houston, Texas, USA.
Christian WernerPTC Therapeutics Germany, GmbH, Frankfurt, Germany.ORCID https://orcid.org/0000-0002-6139-7473
Alexis KrolickPTC Therapeutics, Inc., Warren, New Jersey, USA.
Vinay PenematsaPTC Therapeutics, Inc., Warren, New Jersey, USA.
Antonia WangPTC Therapeutics, Inc., Warren, New Jersey, USA.
Rezwanur RahmanPTC Therapeutics, Inc., Warren, New Jersey, USA.
Lee GoldenPTC Therapeutics, Inc., Warren, New Jersey, USA.
Yin-Hsiu ChienDepartment of Medical Genetics, National Taiwan University Hospital, Taipei, Taiwan.ORCID https://orcid.org/0000-0001-8802-5728
Paul Wuh-Liang HwuDepartment of Medical Genetics, National Taiwan University Hospital, Taipei, Taiwan.

Funding

PTC Therapeutics
6 · The paper itself

Abstract

Aromatic ʟ-amino acid decarboxylase (AADC) deficiency is a rare pediatric neurotransmitter disorder that typically necessitates lifelong care, and that carries a risk of childhood mortality. Eladocagene exuparvovec gene therapy is designed to restore AADC production. Study GT-002 (NCT04903288) is a phase 2, multicenter, open-label trial assessing the pharmacodynamics, safety, and efficacy of eladocagene exuparvovec administered to the putamen bilaterally in pediatric patients with AADC deficiency using a magnetic resonance (MR)-compatible cannula. Patients received eladocagene exuparvovec at 1.8 × 10

Indexed as

Amino Acid Metabolism, Inborn ErrorsAromatic-L-Amino-Acid DecarboxylasesGenetic TherapyAdolescentChildChild, PreschoolFemaleFollow-Up StudiesGene Therapy AgentsGenetic VectorsHomovanillic AcidHumansInfantMagnetic Resonance ImagingMalePutamenAromatic-L-Amino-Acid DecarboxylasesEladocagene exuparvovecHomovanillic AcidAADC deficiencyeladocagene exuparvovecgene therapyintraputaminal administrationMR‐compatible ventricular cannulasafety

Identifiers

PMID41724580
PMCPMC12925825

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.