Overview
Sponsor-declared trial summary
Phase
Phase I and Phase II (Integrated) - Other
Status
Ended
Participants planned
12
Countries
2
Sites
2
Glycogen storage disease type Ia
To determine the long-term safety of DTX401 following a single IV dose in adults with GSDIa
Key facts
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Trial duration
- 18 Oct 2021 → 25 Feb 2025
- Decision date (initial)
- 2024-02-07
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
- Funding sources
- Ultragenyx Pharmaceutical Inc.
External identifiers
- EU CT number
- 2023-504004-29-00
- EudraCT number
- 2018-004473-27
- ClinicalTrials.gov
- NCT03970278
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety
To determine the long-term safety of DTX401 following a single IV dose in adults
with GSDIa
Secondary objectives 1
- To evaluate the long-term effect of DTX401 on symptom-free euglycemia in a setting of a controlled fasting challenge (CFC)
Conditions and MedDRA coding
Glycogen storage disease type Ia
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.1 | LLT | 10056911 | Glycogen storage disease type IA | 10010331 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | LTFU group Subjects who received DTX401 in Study 401GSDIA01 will be followed up for safety and efficacy.
|
Not Applicable | None |
Regulatory references
- Scientific advice from competent authorities
- Finnish Medicines Agency, National Agency For The Safety Of Medicine And Health Products, European Medicines Agency
- Plan to share IPD
- No
- IPD plan description
- Due to the rarity of GSDIa and the small number of subjects in this trial, individual patient date will not be shared in order to safeguard patient privacy, consistent with the data sharing commitment statement listed on Ultragenyx.com
| EU CT number | Title | Sponsor |
|---|---|---|
| 2016-003023-30 | A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease Type Ia (GSDIa), Estudio de fase 1/2, abierto, de seguridad y búsqueda de dosis de la transferencia del gen de la glucosa-6-fosfatasa (G6Pasa) mediada por el virus adenoasociado (AAV) de serotipo 8 (AAV8) en adultos con glucogenosis de tipo Ia (GSD-Ia) |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- Individuals eligible to participate in this study must meet all of the following criteria: 1. Received DTX401 in Study 401GSDIA01
- Willing and able to provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures being performed
- Willing and able to comply with all scheduled study visits, procedures, and requirements
Exclusion criteria 2
- Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study: 1. Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study
- Presence or history of any condition that, in the view of the Investigator, poses a risk to subject safety or places the subject at high risk of poor compliance or not completing the study or that would significantly affect the interpretation of study results
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The incidence of AEs and SAEs for each cohort and overall assessed by severity and relationship to IP
Secondary endpoints 1
- The change from Day 0 (Study 401GSDIA01) in time to first hypoglycemic event during a controlled fasting challenge over time by cohort and overall, following IV administration of DTX401
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD7389681 · Product
- Active substance
- Pariglasgene Brecaparvovec
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS
- Authorisation status
- Not Authorised
- MA holder
- ULTRAGENYX PHARMACEUTICAL INC.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/16/1771
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Ultragenyx Pharmaceutical Inc.
- Sponsor organisation
- Ultragenyx Pharmaceutical Inc.
- Address
- 60 Leveroni Court Suite 200
- City
- Novato
- Postcode
- 94949-5746
- Country
- United States
Scientific contact point
- Organisation
- Ultragenyx Pharmaceutical Inc.
- Contact name
- Medical Information
Public contact point
- Organisation
- Ultragenyx Pharmaceutical Inc.
- Contact name
- Ultragenyx trial information group
Third parties 11
| Organisation | City, country | Duties |
|---|---|---|
| Charles River Laboratories International Inc. ORG-100041066
|
Mattawan, United States | Laboratory analysis |
| Biotel Research LLC ORG-100039864
|
Rochester, United States | Other |
| Pharma Start LLC ORG-100042396
|
Chicago, United States | Other |
| IQVIA Limited ORG-100008655
|
Reading, United Kingdom | Code 14, Other, Interactive response technologies (IRT) |
| Precision For Medicine Inc. ORG-100041895
|
Frederick, United States | Laboratory analysis |
| PPD International Holdings LLC ORG-100007655
|
Zaventem, Belgium | Laboratory analysis |
| Genosafe S.A.S. ORG-100013179
|
Evry Cedex, France | Laboratory analysis |
| Cellular Technology Ltd. ORG-100046556
|
Shaker Heights, United States | Laboratory analysis |
| PPD Development LP ORG-100011560
|
Wilmington, United States | On site monitoring, Code 12, Code 13, Other, Laboratory analysis, Code 5, E-data capture, Code 8 |
| Primevigilance Zagreb d.o.o. ORG-100041973
|
Zagreb, Croatia | Code 8 |
| Gray Consulting Inc. ORG-100044159
|
Philadelphia, United States | Other |
Locations
2 EU/EEA countries · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Netherlands | Ended | 2 | 1 |
| Spain | Ended | 1 | 1 |
| Rest of world
United States, Canada
|
— | 9 | — |
Investigational sites
Universitair Medisch Centrum Groningen
Department of Metabolic Diseases, Hanzeplein 1, 9713 GZ, Groningen
Complexo Hospitalario Universitario De Santiago
Unidad de Diagnóstico y Tratamiento de Enfermedades Metabólicas Congénitas, Calle Choupana Da S/n, 15706, Santiago De Compostela
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Netherlands | 2021-10-18 | 2024-11-26 | 2021-10-18 | 2021-11-01 | |
| Spain | 2021-10-25 | 2024-11-25 | 2021-10-25 | 2021-10-25 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Summary of results Art. 37(4) CTR
| Title | Submission date | Status | Type |
|---|---|---|---|
| 401GSDIA02_Summary of Results SUM-120706
|
2026-02-24T16:38:48 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| 401GSDIA02_Lay Summary | 2026-02-24T16:39:03 | Submitted | Laypersons Summary of Results |
| 401GSDIA02_Lay Summary_FR | 2026-05-02T00:07:23 | Submitted | Laypersons Summary of Results |
| 401GSDIA02_Lay Summary_NL | 2026-05-02T00:07:12 | Submitted | Laypersons Summary of Results |
| 401GSDIA02_Lay Summary_ES | 2026-05-02T00:06:59 | Submitted | Laypersons Summary of Results |
Documents 10 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | 401GSDIA02_Lay Summary | 1 |
| Laypersons summary of results (for publication) | 401GSDIA02_Lay Summary_ES | 1 |
| Laypersons summary of results (for publication) | 401GSDIA02_Lay Summary_FR | 1 |
| Laypersons summary of results (for publication) | 401GSDIA02_Lay Summary_NL | 1 |
| Protocol (for publication) | D1_Ultragenyx_401GSDIA02_Protocol_2023-504004-29-00_Public | 4 |
| Protocol (for publication) | D4_Ultragenyx_401GSDIA02_Morning Glucose Level Worksheet_ENG_Public | N/A |
| Protocol (for publication) | D4_Ultragenyx_401GSDIA02_Morning Glucose Level Worksheet_SPA_Public | N/A |
| Summary of results (for publication) | 401GSDIA02__Summary of Results | 1 |
| Synopsis of the protocol (for publication) | D1_Ultragenyx_401GSDIA02_Protocol Synopsis_2023-504004-29-00_ENG_Public | 4 |
| Synopsis of the protocol (for publication) | D1_Ultragenyx_401GSDIA02_Protocol Synopsis_2023-504004-29-00_SPA_Public | 4 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-12-15 | Spain | Acceptable 2024-02-07
|
2024-02-07 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-01-06 | Spain | Acceptable 2024-02-07
|
2025-01-06 |