Clinical trial • Phase III|Phase IV • Endocrinology|Rare Disease
PARIGLASGENE BRECAPARVOVEC for Glycogen storage disease type Ia (GSDIa)
Phase III|Phase IV trial of PARIGLASGENE BRECAPARVOVEC for Glycogen storage disease type Ia (GSDIa). open-label. 134 participants.
Overview
- Trial Therapeutic Area
- Endocrinology|Rare Disease
- Trial Disease
- Glycogen storage disease type Ia (GSDIa)
- Trial Stage
- Phase III|Phase IV
- Drug Modality
- Gene therapy
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 24-05-2024
- First CTIS Authorization Date
- 16-09-2024
Trial design
open-label Phase III|Phase IV trial in Netherlands, Germany, Spain and others.
- Open Label
- Yes
- Target Sample Size
- 134
- Trial Duration For Participant
- 3650
Eligibility
Recruits 134 paediatric patients.
- Vulnerable Population
- If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures.
Inclusion criteria
- {"criterion_text":"- Patient who had • DTX401 (full or partial dose) administered in a parent clinical study (Group 1) or • Prescribed DTX401 (full or partial dose) administered in a post-marketing setting (Group 2)\n- Patient is willing and able to provide informed consent after the study has been explained, and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures."}
Exclusion criteria
- {"criterion_text":"- Presence of any condition that would interfere with study participation, interpretation of results or affect patient’s safety in the opinion of the Investigator"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Incidence and severity of SAEs assessed as related to DTX401 by the Investigator","definition_or_measurement_approach":"Incidence and severity of serious adverse events (SAEs) judged by the Investigator to be related to DTX401; collected and assessed by the Investigator."}
- {"endpoint_text":"- Incidence, relationship, severity, and seriousness of AESIs for AAV therapies (vector-induced hepatic effects [eg, increased aminotransferase levels], malignancy [new or worsening of pre-existing malignancies], thrombotic microangiopathy, dorsal root ganglion/peripheral nerve effects, and any new potential risks of AAV therapies identified over time)","definition_or_measurement_approach":"Collection of adverse events of special interest (AESIs) related to AAV therapies, including specified event types (vector-induced hepatic effects, malignancy, thrombotic microangiopathy, dorsal root ganglion/peripheral nerve effects) with assessment of incidence, relationship, severity and seriousness over time."}
- {"endpoint_text":"- Incidence and outcomes of pregnancy in patients treated with DTX401 or patient’s partner","definition_or_measurement_approach":"Recording of pregnancy occurrences and their outcomes in patients treated with DTX401 or in partners of patients; incidence and pregnancy outcomes collected."}
- {"endpoint_text":"- Incidence and severity of SAEs of infusion-related reactions including hypersensitivitya","definition_or_measurement_approach":"Collection and assessment of infusion-related serious adverse events, including hypersensitivity reactions; incidence and severity recorded."}
- {"endpoint_text":"- Incidence and severity of SAEs assessed by the Investigator as related to concomitant immunomodulatory therapies (eg, steroids) including adrenal insufficiency and iatrogenic Cushing’s syndromea","definition_or_measurement_approach":"Investigator-assessed incidence and severity of SAEs related to concomitant immunomodulatory therapies (e.g., steroids), with specific interest in adrenal insufficiency and iatrogenic Cushing’s syndrome."}
Secondary endpoints
- {"endpoint_text":"- Nutritional Assessment;Clinical/CGM/Laboratory/Imaging; Major Clinical Events (MCEs); Patient Experience Clinical Interview; HEOR:","definition_or_measurement_approach":"Includes nutritional assessments, clinical/continuous glucose monitoring (CGM), laboratory and imaging assessments, capture of major clinical events (MCEs), patient experience clinical interviews, and health economics and outcomes research (HEOR) measures."}
Recruitment
- Planned Sample Size
- 134
- Recruitment Window Months
- 146
- Consent Approach
- Patients must be willing and able to provide informed consent after the study has been explained and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is expected to provide assent (if possible) and a legally authorized representative will provide informed consent. Age-specific assent/consent documents are provided (examples in the dossier include forms for under 5, 5-7, 8-11, 12-14, 15-17, and adult/parent forms). Patient-facing materials and ICFs are available in multiple languages (Spanish, Dutch, Italian, German, Danish and English as indicated in the submitted documents).
Geography
- Total Number Of Sites
- 6
- Total Number Of Participants
- 14
Netherlands
- Earliest CTIS Part Ii Submission Date
- 09-09-2024
- Latest Decision Or Authorization Date
- 09-02-2026
- Processing Time Days
- 518
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Universitair Medisch Centrum Groningen
- Department Name
- Dept. of Metabolic Diseases
- Principal Investigator Name
- Terry Derks
- Principal Investigator Email
- t.g.j.derks@umcg.nl
- Contact Person Name
- Terry Derks
- Contact Person Email
- t.g.j.derks@umcg.nl
Germany
- Earliest CTIS Part Ii Submission Date
- 10-02-2025
- Latest Decision Or Authorization Date
- 03-02-2026
- Processing Time Days
- 358
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- University Medical Center Hamburg-Eppendorf
- Department Name
- pediatric and adolescent medicine
- Principal Investigator Name
- Nicole Muschol
- Principal Investigator Email
- muschol@uke.de
- Contact Person Name
- Nicole Muschol
- Contact Person Email
- muschol@uke.de
Spain
- Earliest CTIS Part Ii Submission Date
- 19-08-2024
- Latest Decision Or Authorization Date
- 29-01-2026
- Processing Time Days
- 528
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Universidade De Santiago De Compostela
- Department Name
- Unit for Diagnosis and Treatment of Metabolic Diseases
- Principal Investigator Name
- Maria Luz Couce Pico
- Principal Investigator Email
- Maria.Luz.Couce.Pico@sergas.es
- Contact Person Name
- Maria Luz Couce Pico
- Contact Person Email
- Maria.Luz.Couce.Pico@sergas.es
Italy
- Earliest CTIS Part Ii Submission Date
- 04-02-2025
- Latest Decision Or Authorization Date
- 03-02-2026
- Processing Time Days
- 364
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Azienda Ospedaliera Universitaria Federico II Di Napoli
- Department Name
- Mother Child Department
- Principal Investigator Name
- Alessandro Rossi
- Principal Investigator Email
- alessandro.rossi@unina.it
- Contact Person Name
- Alessandro Rossi
- Contact Person Email
- alessandro.rossi@unina.it
- Site Name
- IRCCS Istituto Giannina Gaslini
- Department Name
- Pediatric gastroenterology and digestive endoscopy Unit
- Principal Investigator Name
- Alessandro La Rosa
- Principal Investigator Email
- alessandrolarosa@gaslini.org
- Contact Person Name
- Alessandro La Rosa
- Contact Person Email
- alessandrolarosa@gaslini.org
Denmark
- Earliest CTIS Part Ii Submission Date
- 06-03-2025
- Latest Decision Or Authorization Date
- 29-01-2026
- Processing Time Days
- 329
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Rigshospitalet
- Department Name
- Department of Paediatrics and Clinical Genetics
- Principal Investigator Name
- Allan Meldgaard Lund
- Principal Investigator Email
- allan.lundh@regionh.dk
- Contact Person Name
- Allan Meldgaard Lund
- Contact Person Email
- allan.lundh@regionh.dk
Sponsor
Primary sponsor
- Full Name
- Ultragenyx Pharmaceutical Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Third parties
- {"country":"Croatia","full_name":"Primevigilance Zagreb d.o.o.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Stichting EuroQol Research Foundation","duties_or_roles":"Provides EQ-5D-5L/Y questionnaire (paper)","organisation_type":"Patient organisation/association"}
- {"country":"United Kingdom","full_name":"Dietary Assessment Limited","duties_or_roles":"Myfood24 is a robust nutrition platform enabling accurate tracking and monitoring of dietary intake.","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Pariglasgene brecaparvovec
- Active Substance
- PARIGLASGENE BRECAPARVOVEC
- Modality
- Gene therapy
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Orphan Designation
- Yes
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