---
title: "Phase 3 Randomized Trial of DTX401 AAV Gene Therapy for GSDIa — Trial Report"
id: "pubmed-42674977"
canonical_url: "https://medichelpline.com/clinical-feed/pubmed-42674977"
content_type: "clinical_feed_article"
specialty: "Pharmacology"
source_name: "PubMed / NCBI"
source_url: "https://pubmed.ncbi.nlm.nih.gov/42674977/"
doi: "10.1002/jimd.70241"
published_at: "2026-09-01T00:00:00.000Z"
evidence_level: "Journal Article"
license: "CC-BY-NC-4.0 / Informational Use"
---
# Phase 3 Randomized Trial of DTX401 AAV Gene Therapy for GSDIa — Trial Report
## Provenance & Clinical Metadata
- **Canonical URL:** https://medichelpline.com/clinical-feed/pubmed-42674977
- **Specialty:** [Pharmacology](https://medichelpline.com/clinical-feed/pharmacology.md)
- **Primary Source:** PubMed / NCBI
- **Source URL:** [Original Journal Publication](https://pubmed.ncbi.nlm.nih.gov/42674977/)
- **DOI:** [10.1002/jimd.70241](https://doi.org/10.1002%2Fjimd.70241)
- **Published At:** 2026-09-01T00:00:00.000Z
- **Evidence Rating:** Journal Article
## Executive GIST (TL;DR)
- The article reports a **Phase 3 randomized trial** evaluating **DTX401**, an **AAV gene therapy**, for the treatment of **Glycogen Storage Disease type Ia (GSDIa)**, published in J Inherit Metab Dis (Sep 2026) with DOI 10.1002/jimd.70241 and PMID 42674977. - The publication lists a large, international, multicenter author group spanning pediatric metabolic centers and academic hospitals across North America, Europe, and Latin America, with involvement from Ultragenyx Pharmaceutical Inc. - Lead and contributing authors are affiliated with institutions including Montreal Children’s Hospital, Rady Children’s Health, University Medical Center Groningen, Mount Sinai, Children’s Hospital of Philadelphia, Duke University, University of Connecticut, University of Utah, Rigshospitalet Copenhagen, and others. - The citation identifies this work as a Clinical Trial report in the Journal of Inherited Metabolic Disease, volume 49, issue 5, article e70241 (September 2026). - The PubMed entry provides bibliographic metadata (authors, affiliations, journal, DOI, PMID) but the SOURCE provided here does not include the trial abstract, numerical results, safety data, endpoints, randomization details, or conclusions; those details were not reported in the supplied source text. - Because outcome data and trial methods were not present in the source material, no efficacy, safety, or statistical results can be summarized from this input; readers should consult the full journal article or publisher site for complete trial findings. - The presence of industry co-authors from Ultragenyx Pharmaceutical Inc. is noted in the author list and suggests sponsor involvement, but the specific role (sponsor, funder, or contributor) is not detailed in the supplied excerpt. - This PubMed record functions primarily as a bibliographic entry; clinical interpretation or practice changes should rely on the complete published manuscript and supporting materials that were not included in the provided SOURCE text.
## Clinical Analysis & Structured Key Points
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Treatment of GSDIa [John J Mitchell](https://pubmed.ncbi.nlm.nih.gov/?term=Mitchell+JJ&cauthor_id=42674977)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-1 "Montreal Children's Hospital, Montreal, Quebec, Canada."), [Jose E Abdenur](https://pubmed.ncbi.nlm.nih.gov/?term=Abdenur+JE&cauthor_id=42674977)[ 2 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-2 "Rady Children's Health, Orange, California, USA."), [Foekje de Boer](https://pubmed.ncbi.nlm.nih.gov/?term=de+Boer+F&cauthor_id=42674977)[ 3 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-3 "University of Groningen, University Medical Center Groningen, Beatrix Children's Hospital, Section of Metabolic Diseases, Groningen, the Netherlands.")[ 4 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-4 "UMCG Center of Expertise for Carbohydrate, Fatty Acid Oxidation and Ketone Bodies Disorders, University Medical Center Groningen, Groningen, the Netherlands."), [Monica Boyer](https://pubmed.ncbi.nlm.nih.gov/?term=Boyer+M&cauthor_id=42674977)[ 2 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-2 "Rady Children's Health, Orange, California, USA."), [Margo Sheck Breilyn](https://pubmed.ncbi.nlm.nih.gov/?term=Breilyn+MS&cauthor_id=42674977)[ 5 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-5 "Mount Sinai, New York, New York, USA."), [María-Luz Couce](https://pubmed.ncbi.nlm.nih.gov/?term=Couce+ML&cauthor_id=42674977)[ 6 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-6 "Hospital Clínico Universitario de Santiago de Compostela, IDIS, CIBERER, Santiago de Compostela, Spain."), [Diva D De Leon](https://pubmed.ncbi.nlm.nih.gov/?term=De+Leon+DD&cauthor_id=42674977)[ 7 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-7 "Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA."), [Terry G Derks](https://pubmed.ncbi.nlm.nih.gov/?term=Derks+TG&cauthor_id=42674977)[ 3 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-3 "University of Groningen, University Medical Center Groningen, Beatrix Children's Hospital, Section of Metabolic Diseases, Groningen, the Netherlands.")[ 4 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-4 "UMCG Center of Expertise for Carbohydrate, Fatty Acid Oxidation and Ketone Bodies Disorders, University Medical Center Groningen, Groningen, the Netherlands."), [Areeg El-Gharbawy](https://pubmed.ncbi.nlm.nih.gov/?term=El-Gharbawy+A&cauthor_id=42674977)[ 8 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-8 "Duke University Medical Center, Durham, North Carolina, USA."), [Andrea B Haijer-Schreuder](https://pubmed.ncbi.nlm.nih.gov/?term=Haijer-Schreuder+AB&cauthor_id=42674977)[ 3 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-3 "University of Groningen, University Medical Center Groningen, Beatrix Children's Hospital, Section of Metabolic Diseases, Groningen, the Netherlands.")[ 4 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-4 "UMCG Center of Expertise for Carbohydrate, Fatty Acid Oxidation and Ketone Bodies Disorders, University Medical Center Groningen, Groningen, the Netherlands."), [Karen Loechner](https://pubmed.ncbi.nlm.nih.gov/?term=Loechner+K&cauthor_id=42674977)[ 9 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-9 "University of Connecticut, Farmington, Connecticut, USA."), [Nicola Longo](https://pubmed.ncbi.nlm.nih.gov/?term=Longo+N&cauthor_id=42674977)[ 10 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-10 "University of Utah, Salt Lake City, Utah, USA."), [Allan M Lund](https://pubmed.ncbi.nlm.nih.gov/?term=Lund+AM&cauthor_id=42674977)[ 11 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-11 "Centre for Inherited Metabolic Diseases, Rigshospitalet, Copenhagen, Denmark."), [Miguel Angel Martinez Olmos](https://pubmed.ncbi.nlm.nih.gov/?term=Olmos+MAM&cauthor_id=42674977)[ 12 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-12 "Hospital Clínico Universitario de Santiago de Compostela, IDIS, CIBEROBN, Santiago de Compostela, Spain."), [Shawn E McCandless](https://pubmed.ncbi.nlm.nih.gov/?term=McCandless+SE&cauthor_id=42674977)[ 13 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-13 "Children's Hospital Colorado, Aurora, Colorado, USA."), [Bibiana Mello de Oliveira](https://pubmed.ncbi.nlm.nih.gov/?term=de+Oliveira+BM&cauthor_id=42674977)[ 14 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-14 "Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil."), [Malaya Mount](https://pubmed.ncbi.nlm.nih.gov/?term=Mount+M&cauthor_id=42674977)[ 9 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-9 "University of Connecticut, Farmington, Connecticut, USA."), [Nicole Muschol](https://pubmed.ncbi.nlm.nih.gov/?term=Muschol+N&cauthor_id=42674977)[ 15 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-15 "University Medical Center Hamburg-Eppendorf, Hamburg, Germany."), [Kristina Pytlak](https://pubmed.ncbi.nlm.nih.gov/?term=Pytlak+K&cauthor_id=42674977)[ 16 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-16 "Cleveland Clinic Childrens, Cleveland, Ohio, USA."), [Kadakkal Radhakrishnan](https://pubmed.ncbi.nlm.nih.gov/?term=Radhakrishnan+K&cauthor_id=42674977)[ 16 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-16 "Cleveland Clinic Childrens, Cleveland, Ohio, USA."), [Rebecca Riba-Wolman](https://pubmed.ncbi.nlm.nih.gov/?term=Riba-Wolman+R&cauthor_id=42674977)[ 9 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-9 "University of Connecticut, Farmington, Connecticut, USA."), [David F Rodriguez-Buritica](https://pubmed.ncbi.nlm.nih.gov/?term=Rodriguez-Buritica+DF&cauthor_id=42674977)[ 17 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-17 "Department of Pediatrics, Division of Medical Genetics, McGovern Medical School at the University of Texas Health Science Center at Houston \(UTHealth Houston\) and Children's Memorial Hermann Hospital, Houston, Texas, USA."), [Alessandro La Rosa](https://pubmed.ncbi.nlm.nih.gov/?term=La+Rosa+A&cauthor_id=42674977)[ 18 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-18 "Pediatric Clinic and Endocrinology Unit, IRCCS Istituto Giannina Gaslini, Genoa, Italy."), [Alessandro Rossi](https://pubmed.ncbi.nlm.nih.gov/?term=Rossi+A&cauthor_id=42674977)[ 19 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-19 "Department of Translational Medicine, University of Naples Federico II, Naples, Italy."), [Heather Saavedra](https://pubmed.ncbi.nlm.nih.gov/?term=Saavedra+H&cauthor_id=42674977)[ 17 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-17 "Department of Pediatrics, Division of Medical Genetics, McGovern Medical School at the University of Texas Health Science Center at Houston \(UTHealth Houston\) and Children's Memorial Hermann Hospital, Houston, Texas, USA."), [René Santer](https://pubmed.ncbi.nlm.nih.gov/?term=Santer+R&cauthor_id=42674977)[ 15 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-15 "University Medical Center Hamburg-Eppendorf, Hamburg, Germany."), [Brian Shayota](https://pubmed.ncbi.nlm.nih.gov/?term=Shayota+B&cauthor_id=42674977)[ 10 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-10 "University of Utah, Salt Lake City, Utah, USA."), [G Peter A Smit](https://pubmed.ncbi.nlm.nih.gov/?term=Smit+GPA&cauthor_id=42674977)[ 3 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-3 "University of Groningen, University Medical Center Groningen, Beatrix Children's Hospital, Section of Metabolic Diseases, Groningen, the Netherlands.")[ 4 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-4 "UMCG Center of Expertise for Carbohydrate, Fatty Acid Oxidation and Ketone Bodies Disorders, University Medical Center Groningen, Groningen, the Netherlands."), [Carolina F Moura De Souza](https://pubmed.ncbi.nlm.nih.gov/?term=Moura+De+Souza+CF&cauthor_id=42674977)[ 14 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-14 "Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil."), [Melanie M van der Klauw](https://pubmed.ncbi.nlm.nih.gov/?term=van+der+Klauw+MM&cauthor_id=42674977)[ 20 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-20 "Department of Endocrinology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands."), [David A Weinstein](https://pubmed.ncbi.nlm.nih.gov/?term=Weinstein+DA&cauthor_id=42674977)[ 9 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-9 "University of Connecticut, Farmington, Connecticut, USA."), [Joseph I Wolfsdorf](https://pubmed.ncbi.nlm.nih.gov/?term=Wolfsdorf+JI&cauthor_id=42674977)[ 21 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-21 "Boston Children's Hospital, Boston, Massachusetts, USA."), [Anne Blake](https://pubmed.ncbi.nlm.nih.gov/?term=Blake+A&cauthor_id=42674977)[ 22 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-22 "Ultragenyx Pharmaceutical Inc., Novato, California, USA."), [Andrew A Grimm](https://pubmed.ncbi.nlm.nih.gov/?term=Grimm+AA&cauthor_id=42674977)[ 22 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-22 "Ultragenyx Pharmaceutical Inc., Novato, California, USA."), [Deepali Mitragotri](https://pubmed.ncbi.nlm.nih.gov/?term=Mitragotri+D&cauthor_id=42674977)[ 22 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-22 "Ultragenyx Pharmaceutical Inc., Novato, California, USA."), [Syeda Rahman](https://pubmed.ncbi.nlm.nih.gov/?term=Rahman+S&cauthor_id=42674977)[ 22 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-22 "Ultragenyx Pharmaceutical Inc., Novato, California, USA."), [Diane M Turner-Bowker](https://pubmed.ncbi.nlm.nih.gov/?term=Turner-Bowker+DM&cauthor_id=42674977)[ 22 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-22 "Ultragenyx Pharmaceutical Inc., Novato, California, USA."), [Richard Collis](https://pubmed.ncbi.nlm.nih.gov/?term=Collis+R&cauthor_id=42674977)[ 22 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#full-view-affiliation-22 "Ultragenyx Pharmaceutical Inc., Novato, California, USA.") Affiliations Expand ### Affiliations * 1 Montreal Children's Hospital, Montreal, Quebec, Canada. * 2 Rady Children's Health, Orange, California, USA. * 3 University of Groningen, University Medical Center Groningen, Beatrix Children's Hospital, Section of Metabolic Diseases, Groningen, the Netherlands. * 4 UMCG Center of Expertise for Carbohydrate, Fatty Acid Oxidation and Ketone Bodies Disorders, University Medical Center Groningen, Groningen, the Netherlands. * 5 Mount Sinai, New York, New York, USA. * 6 Hospital Clínico Universitario de Santiago de Compostela, IDIS, CIBERER, Santiago de Compostela, Spain. * 7 Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA. * 8 Duke University Medical Center, Durham, North Carolina, USA. * 9 University of Connecticut, Farmington, Connecticut, USA. * 10 University of Utah, Salt Lake City, Utah, USA. * 11 Centre for Inherited Metabolic Diseases, Rigshospitalet, Copenhagen, Denmark. * 12 Hospital Clínico Universitario de Santiago de Compostela, IDIS, CIBEROBN, Santiago de Compostela, Spain. * 13 Children's Hospital Colorado, Aurora, Colorado, USA. * 14 Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil. * 15 University Medical Center Hamburg-Eppendorf, Hamburg, Germany. * 16 Cleveland Clinic Childrens, Cleveland, Ohio, USA. * 17 Department of Pediatrics, Division of Medical Genetics, McGovern Medical School at the University of Texas Health Science Center at Houston (UTHealth Houston) and Children's Memorial Hermann Hospital, Houston, Texas, USA. * 18 Pediatric Clinic and Endocrinology Unit, IRCCS Istituto Giannina Gaslini, Genoa, Italy. * 19 Department of Translational Medicine, University of Naples Federico II, Naples, Italy. * 20 Department of Endocrinology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands. * 21 Boston Children's Hospital, Boston, Massachusetts, USA. * 22 Ultragenyx Pharmaceutical Inc., Novato, California, USA. * PMID: **42674977** * DOI: [ 10.1002/jimd.70241 ](https://doi.org/10.1002/jimd.70241) Item in Clipboard Clinical Trial # Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa John J Mitchell et al. J Inherit Metab Dis. 2026 Sep. Show details Display options Display options Format Abstract PubMed PMID J Inherit Metab Dis Actions * [ Search in PubMed ](https://pubmed.ncbi.nlm.nih.gov/?term=%22J+Inherit+Metab+Dis%22%5Bjour%5D&sort=date&sort_order=desc) * [ Search in NLM Catalog ](https://www.ncbi.nlm.nih.gov/nlmcatalog?term=%22J+Inherit+Metab+Dis%22%5BTitle+Abbreviation%5D) * [ Add to Search ](https://pubmed.ncbi.nlm.nih.gov/42674977/) . 2026 Sep;49(5):e70241. doi: 10.1002/jimd.70241. ### Authors [John J Mitchell](https://pubmed.ncbi.nlm.nih.gov/?term=Mitchell+JJ&cauthor_id=42674977)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#short-view-affiliation-1 "Montreal Children's Hospital, Montreal, Quebec, Canada."), [Jose E Abdenur](https://pubmed.ncbi.nlm.nih.gov/?term=Abdenur+JE&cauthor_id=42674977)[ 2 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#short-view-affiliation-2 "Rady Children's Health, Orange, California, USA."), [Foekje de Boer](https://pubmed.ncbi.nlm.nih.gov/?term=de+Boer+F&cauthor_id=42674977)[ 3 ](https://pubmed.ncbi.nlm.nih.gov/42674977/#short-view-affiliation-3 "University of Groningen, University Medical Center Groningen, Beatrix Children's Hospital, Section of Metabolic Diseases,
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