Clinical trial • Phase III • Rare Disease
apazunersen for Angelman syndrome
Phase III trial of apazunersen for Angelman syndrome. open-label, none/not specified-controlled. 60 participants.
Overview
- Trial Therapeutic Area
- Rare Disease
- Trial Disease
- Angelman syndrome
- Trial Stage
- Phase III
- Drug Modality
- Oligonucleotide
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 15-04-2024
- First CTIS Authorization Date
- 18-07-2024
Trial design
open-label, none/not specified-controlled Phase III trial across 6 sites in Germany, Spain, France.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 60
Eligibility
Recruits 60 paediatric patients.
- Pregnancy Exclusion
- Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study.
- Vulnerable Population
- Pediatric subjects are included (isVulnerablePopulationSelected = true). Informed consent must be signed by parent(s) or legal guardian(s). No specific assent process text is provided in the available data.
Inclusion criteria
- {"criterion_text":"- Signed informed consent from parent(s) or legal guardian(s)."}
- {"criterion_text":"- Completed a final study visit in a prior clinical trial with GTX-102. If the subject has an ongoing adverse event (AE), Medical Monitor approval is required to rollover. The Screening visit must occur within 6 months of the last visit in the prior GTX-102 study."}
- {"criterion_text":"- From the time of informed consent through the end of the study and for at least 6 months after the final dose of GTX-102, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102."}
Exclusion criteria
- {"criterion_text":"- Discontinued from prior GTX-102 trial due to treatment-emergent adverse event assessed as related to GTX-102 and where the risk of study participation outweighs the benefits as deemed by the clinical judgment of the Investigator."}
- {"criterion_text":"- History or evidence of any other medical, neurological, or psychological condition that would expose the subject to an undue risk of a significant AE or interfere with study assessments during the course of the trial as determined by the clinical judgment of the Investigator."}
- {"criterion_text":"- Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects."}
- {"criterion_text":"- Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Treatment-emergent AEs and SAEs, frequency, severity, and relationship to investigational product throughout the study","definition_or_measurement_approach":"Monitoring and recording of treatment-emergent adverse events (AEs) and serious adverse events (SAEs) with assessment of frequency, severity, and relationship to the investigational product throughout the study."}
Secondary endpoints
- {"endpoint_text":"- Change from LTE Month 0 and pretreatment (when available) throughout the study in: • Bayley-4 Cognitive raw score • Bayley-4 Receptive Communication raw score • Bayley-4 Expressive Communication raw score • Bayley-4 Gross Motor raw score • Bayley-4 Fine Motor raw score","definition_or_measurement_approach":"Change from LTE Month 0 and, when available, pre-treatment baseline measured using Bayley-4 raw scores for cognitive, receptive communication, expressive communication, gross motor, and fine motor domains."}
Recruitment
- Planned Sample Size
- 60
- Recruitment Window Months
- 60
- Consent Approach
- Signed informed consent must be provided by parent(s) or legal guardian(s). Parent/guardian ICFs and subject information are available in multiple languages (documents listed include German, Spanish, French; patient-facing documents are available in English as well). No specific assent procedure is provided in the available documents.
Geography
- Total Number Of Sites
- 6
- Total Number Of Participants
- 38
Germany
- Earliest CTIS Part Ii Submission Date
- 09-07-2024
- Latest Decision Or Authorization Date
- 12-11-2025
- Processing Time Days
- 491
- Number Of Sites
- 2
- Number Of Participants
- 10
Sites
- Site Name
- University Medical Center Hamburg-Eppendorf
- Department Name
- Pediatric Neurology
- Contact Person Name
- Deike Weiss
- Contact Person Email
- d.weiss@uke.de
- Site Name
- Universitaet Leipzig
- Department Name
- Pediatric Neurology
- Contact Person Name
- Andreas Merkenschlager
- Contact Person Email
- andreas.merkenschlager@medizin.uni-leipzig.de
Spain
- Earliest CTIS Part Ii Submission Date
- 01-07-2024
- Latest Decision Or Authorization Date
- 13-11-2025
- Processing Time Days
- 500
- Number Of Sites
- 2
- Number Of Participants
- 17
Sites
- Site Name
- Hospital Universitario Puerta De Hierro De Majadahonda
- Department Name
- Pediatric
- Contact Person Name
- Maria Lorenzo Ruiz
- Contact Person Email
- maria.lorenzo@salud.madrid.org
- Site Name
- Parc Tauli Hospital Universitari
- Department Name
- Pediatric
- Contact Person Name
- Ana Roche Martinez
- Contact Person Email
- aroche@tauli.cat
France
- Earliest CTIS Part Ii Submission Date
- 10-07-2024
- Latest Decision Or Authorization Date
- 16-02-2026
- Processing Time Days
- 586
- Number Of Sites
- 2
- Number Of Participants
- 11
Sites
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Hôpital d’enfants de la Timone
- Contact Person Name
- Mathieu MILH
- Contact Person Email
- mathieu.milh@ap-hm.fr
- Site Name
- Hopital Necker Enfants Malades
- Department Name
- Neurologie Pédiatrique
- Contact Person Name
- Nadia BAHI-BUISON
- Contact Person Email
- nadia.bahi-buisson@aphp.fr
Sponsor
Primary sponsor
- Full Name
- Ultragenyx Pharmaceutical Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Primevigilance Limited
- Responsibilities
- 8
- Name
- Suvoda LLC
- Responsibilities
- 3
- Name
- Almac Clinical Services Limited
- Responsibilities
- 14
- Name
- Icon Clinical Research Limited
- Responsibilities
- 4
- Name
- Worldwide Clinical Trials d.o.o.
- Responsibilities
- 1,12,5,6
- Name
- Charles River Laboratories Montreal ULC
- Responsibilities
- 4
- Name
- Gray Consulting Inc.
- Responsibilities
- 15: Patient reimbursement
- Name
- Yprime LLC
- Responsibilities
- 15: Rater training; 7
- Name
- Clario eResearch Technology GmbH
- Responsibilities
- 15: ECG
- Name
- Veeva Systems Inc.
- Responsibilities
- 15: eTMF
- Name
- Medidata Solutions Inc.
- Responsibilities
- 7
- Name
- EPL Pathology Archives LLC
- Responsibilities
- 15: Laboratory Sample Storage
Third parties
- {"country":"United Kingdom","full_name":"Primevigilance Limited","duties_or_roles":"8","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"3","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"14","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Yprime LLC","duties_or_roles":"15: Rater training; 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Croatia","full_name":"Worldwide Clinical Trials d.o.o.","duties_or_roles":"1,12,5,6","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Gray Consulting Inc.","duties_or_roles":"15: Patient reimbursement","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Canada","full_name":"Charles River Laboratories Montreal ULC","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Clario eResearch Technology GmbH","duties_or_roles":"15: ECG","organisation_type":"Industry"}
- {"country":"United States","full_name":"Veeva Systems Inc.","duties_or_roles":"15: eTMF","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"EPL Pathology Archives LLC","duties_or_roles":"15: Laboratory Sample Storage","organisation_type":"Laboratory/Research/Testing facility"}
Investigational products
- Investigational Product Name
- GTX-102
- Active Substance
- apazunersen
- Modality
- Oligonucleotide
- Routes Of Administration
- Intrathecal use
- Route
- Intrathecal
- Orphan Designation
- Yes
- Investigational Product Name
- GTX/UX Diluent and Flush Solution
- Active Substance
- sodium dihydrogen phosphate dihydrate; disodium phosphate; potassium chloride; sodium chloride; calcium chloride dihydrate; magnesium sulfate heptahydrate
- Modality
- Small molecule
- Routes Of Administration
- Intrathecal use
- Route
- Intrathecal
- Investigational Product Name
- ELLIOTTS B SOLUTION (buffered intrathecal electrolyte/dextrose injection)
- Active Substance
- calcium chloride; glucose anhydrous; sodium hydrogen carbonate; potassium chloride; sodium chloride; sodium phosphate; magnesium sulfate
- Modality
- Small molecule
- Routes Of Administration
- Intrathecal use
- Route
- Intrathecal
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