The PubMed entry documents a Phase 3 randomized trial evaluating DTX401, an AAV gene therapy, for the treatment of Glycogen Storage Disease type Ia (GSDIa). The record is indexed as a Clinical Trial in the Journal of Inherited Metabolic Disease, published September 2026 (J Inherit Metab Dis. 2026 Sep;49(5):e70241), with DOI 10.1002/jimd.70241 and PubMed ID (PMID) 42674977.
The title explicitly identifies the investigational agent (DTX401), the vector type (AAV gene therapy), the target condition (GSDIa), and the trial phase (Phase 3 randomized trial). Beyond these high-level identifiers, the supplied source text does not include the trial abstract or the manuscript body. Therefore, protocol details, eligibility criteria, primary and secondary endpoints, randomization schema, sample size, follow-up duration, efficacy outcomes, safety results, and statistical analyses were not reported in the provided PubMed excerpt.
The published record lists a broad, international author group representing multiple pediatric metabolic and academic centers. Lead and coauthors include clinicians and investigators affiliated with institutions such as Montreal Children's Hospital; Rady Children's Health; University Medical Center Groningen (Beatrix Children's Hospital); Mount Sinai; Hospital Clínico Universitario de Santiago de Compostela; Children's Hospital of Philadelphia; Duke University Medical Center; University of Connecticut; University of Utah; Rigshospitalet (Copenhagen); Children’s Hospital Colorado; Hospital de Clinicas de Porto Alegre; University Medical Center Hamburg-Eppendorf; Cleveland Clinic Children's; McGovern Medical School at UTHealth Houston; IRCCS Istituto Giannina Gaslini; University of Naples Federico II; and Boston Children's Hospital.
The author list also includes contributors affiliated with Ultragenyx Pharmaceutical Inc., Novato, California, indicating industry involvement among the authors. The supplied metadata does not specify the nature or extent of industry roles (for example, sponsor, funder, or data analysis partner) within the trial; those specifics were not reported in the provided source text.
This work is cataloged in PubMed with the following bibliographic identifiers:
The PubMed entry classifies the item as a Clinical Trial. The record supplies full author names and institutional affiliations, but the provided excerpt does not include the article abstract text or main manuscript content.
The PubMed excerpt accessed here functions primarily as a bibliographic record. In this SOURCE excerpt, the abstract and detailed trial results are absent. Consequently, essential information typically needed to evaluate a randomized Phase 3 trial — including primary and secondary endpoints, efficacy outcomes, adverse events, statistical significance, subgroup analyses, and trial conduct details — are not available in the supplied material.
Readers seeking the complete trial data, methods, and authors' conclusions should consult the full text of the published article via the journal, the DOI link, or the publisher's website. The PubMed record provides the DOI and PMID to facilitate retrieval of the complete manuscript. If institutional access is required, readers may use interlibrary resources or contact the corresponding author or sponsoring organization for access or supplementary materials.
Interpretation limits:
Recommended next steps for clinicians, researchers, and other readers:
Retrieve and review the full published manuscript (DOI: 10.1002/jimd.70241) for complete trial methodology, outcomes, safety analyses, and authors' interpretations.
Evaluate the trial's primary and secondary endpoints, statistical methods, and population characteristics before applying findings to clinical practice or designing further research.
Review disclosures and funding statements in the full article to clarify Ultragenyx Pharmaceutical Inc.'s role and any potential conflicts of interest.
If needed, consult supplementary materials, clinical trial registry entries (trial identifier if available in the full text), and regulatory communications for additional context on trial conduct and subsequent regulatory actions.
Summary note
This PubMed entry confirms the existence and bibliographic details of a Phase 3 randomized trial of DTX401 AAV gene therapy for GSDIa published in September 2026, authored by a large international group including academic and industry contributors. The supplied SOURCE excerpt does not contain the trial abstract or outcome data; therefore, no trial results or clinical conclusions can be reported from this material alone. For complete clinical assessment, consult the full-text article and associated supporting documents.