Clinical trial • Phase IV • Musculoskeletal
VAMOROLONE for Duchenne muscular dystrophy
Phase IV trial of VAMOROLONE for Duchenne muscular dystrophy. open-label, none/not specified-controlled. 39 participants.
Overview
- Trial Therapeutic Area
- Musculoskeletal
- Trial Disease
- Duchenne muscular dystrophy
- Trial Stage
- Phase IV
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 27-06-2024
- First CTIS Authorization Date
- 04-10-2024
Trial design
open-label, none/not specified-controlled Phase IV trial in Greece, Netherlands, Czechia and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 39
- Trial Duration For Participant
- 1461
Eligibility
Recruits 39 paediatric patients.
- Vulnerable Population
- Vulnerable population: male minors (boys) with Duchenne muscular dystrophy. Informed consent is obtained from the subject and/or the subject’s parent(s) or legal guardian. Age-appropriate assent and information documents are provided (examples in documentation: Information/assent forms for ages 6–9, 10–11, 12–15, 16–17). Country-specific SIS/ICF and assent forms are included in multiple languages.
Inclusion criteria
- {"criterion_text":"- Subject and/or subject’s parent(s) or legal guardian has provided written informed consent"}
- {"criterion_text":"- Subject has previously completed either the VBP15-LTE or VBP15-004 study, and transitioned through the CUP, NPP or EAP"}
- {"criterion_text":"- Subject is on vamorolone on the day of enrolment"}
- {"criterion_text":"- Subject and parent / legal guardian are willing and able to comply with the protocol schedule, assessments and requirements"}
Exclusion criteria
- {"criterion_text":"- Any medical condition, which in the opinion of the Investigator, would affect study participation, performance or interpretation of study assessments"}
- {"criterion_text":"- Vamorolone treatment discontinued for ≥6 months within the year prior to enrolment for a non-safety reason, or vamorolone treatment previously discontinued at any time for a safety reason"}
- {"criterion_text":"- Severe hepatic impairment"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Number of vertebral fractures per 1000 person-years based on X- ray central reading","definition_or_measurement_approach":"Based on X-ray central reading; expressed as number of vertebral fractures per 1000 person-years."}
Secondary endpoints
- {"endpoint_text":"- Time to first vertebral fractures (cumulative incidence)","definition_or_measurement_approach":"Cumulative incidence measurement of time to first vertebral fracture."}
- {"endpoint_text":"- Number of non-vertebral fractures per 1000 person-years based on investigator reporting","definition_or_measurement_approach":"Count per 1000 person-years based on investigator reporting."}
- {"endpoint_text":"- Time to first non-vertebral fractures (cumulative incidence)","definition_or_measurement_approach":"Cumulative incidence measurement of time to first non-vertebral fracture."}
- {"endpoint_text":"- Number of cataracts per 1000 person-years based on ophthalmologist assessment","definition_or_measurement_approach":"Count per 1000 person-years based on ophthalmologist assessment."}
- {"endpoint_text":"- Number of subjects not reaching Tanner stage 2 by 15 years of age","definition_or_measurement_approach":"Count of subjects who have not reached Tanner stage 2 by age 15."}
- {"endpoint_text":"- Frequency of adverse events (AEs) and serious adverse events (SAEs)","definition_or_measurement_approach":"Frequency counts of AEs and SAEs as reported during study."}
- {"endpoint_text":"- Change from baseline in body weight, height and body mass index (BMI)","definition_or_measurement_approach":"Change-from-baseline measures for weight, height and BMI."}
- {"endpoint_text":"- Number of subjects with clinically relevant laboratory abnormalities including glycosylated haemoglobin (HbA1c), and morning cortisol","definition_or_measurement_approach":"Count of subjects with clinically relevant lab abnormalities, including HbA1c and morning cortisol."}
- {"endpoint_text":"- Change from baseline in Time to Stand Test (TTSTAND) velocity","definition_or_measurement_approach":"Change-from-baseline in TTSTAND velocity."}
- {"endpoint_text":"- 6-Minute Walk Test (6MWT) distance","definition_or_measurement_approach":"Absolute 6MWT distance measurement."}
- {"endpoint_text":"- Change from baseline in 6MWT distance","definition_or_measurement_approach":"Change-from-baseline in 6MWT distance."}
- {"endpoint_text":"- NorthStar Ambulatory Assessment (NSAA) scores","definition_or_measurement_approach":"NSAA score assessments."}
- {"endpoint_text":"- Age at ambulatory and non-ambulatory milestones","definition_or_measurement_approach":"Recorded age at achievement of ambulatory and non-ambulatory milestones."}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 39
- Recruitment Window Months
- 48
- Consent Approach
- Informed consent is required from the subject and/or the subject’s parent(s) or legal guardian. Age-appropriate assent and information sheets are provided; documentation includes assent/information forms for age groups 6–9, 10–11, 12–15, and 16–17 and parental/legal guardian ICFs. Multiple language versions are provided across countries (examples include English, Greek, Spanish, Dutch, French, Czech and country-specific ICF/SIS documents). Separate forms for pregnant partners (adult and minor) are included.
Methods
- Recruitment arrangements documents (K1_Recruitment arrangements) available for multiple countries (documents listed in CTIS document list).
- Scout e-mail (document: L2_Other subject information material_Scout e-mail) referenced in recruitment materials.
- Scout Study Brochure (document: L2_Other subject information material_Scout Study Brochure) included in recruitment materials.
- Scout Taxable Payments Letter (document: L2_Other subject information material_Scout Taxable Payments Letter) included in recruitment materials.
- Patient-facing materials such as patient card and patient diaries (documents listed) used as part of recruitment/participant information.
Geography
- Total Number Of Sites
- 11
- Total Number Of Participants
- 26
Greece
- Earliest CTIS Part Ii Submission Date
- 08-01-2025
- Latest Decision Or Authorization Date
- 13-10-2025
- Processing Time Days
- 278
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Nosokomeio Paidon I Agia Sofia
- Department Name
- Neurological Department
- Principal Investigator Name
- Marina Katsalouli
- Principal Investigator Email
- mkatsalouli@hotmail.com
- Contact Person Name
- Marina Katsalouli
- Contact Person Email
- mkatsalouli@hotmail.com
Netherlands
- Earliest CTIS Part Ii Submission Date
- 27-01-2025
- Latest Decision Or Authorization Date
- 29-10-2025
- Processing Time Days
- 275
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Radboud universitair medisch centrum Stichting
- Department Name
- Rehabilitation
- Principal Investigator Name
- Saskia Houwen
- Principal Investigator Email
- saskia.houwen@radboudumc.nl
- Contact Person Name
- Saskia Houwen
- Contact Person Email
- saskia.houwen@radboudumc.nl
- Site Name
- Leids Universitair Medisch Centrum (LUMC)
- Department Name
- Neurology
- Principal Investigator Name
- Erik Niks
- Principal Investigator Email
- e.niks@lumc.nl
- Contact Person Name
- Erik Niks
- Contact Person Email
- e.niks@lumc.nl
Czechia
- Earliest CTIS Part Ii Submission Date
- 16-09-2024
- Latest Decision Or Authorization Date
- 17-09-2025
- Processing Time Days
- 366
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- Fakultni Nemocnice V Motole
- Department Name
- Paediatric Neurology
- Principal Investigator Name
- Jana Haberlova
- Principal Investigator Email
- Jana.haberlova@fnmotol.cz
- Contact Person Name
- Jana Haberlova
- Contact Person Email
- Jana.haberlova@fnmotol.cz
- Site Name
- Fakultni Nemocnice Brno
- Department Name
- Paediatric Neurology
- Principal Investigator Name
- Lenka Jurikova
- Principal Investigator Email
- jurikova.lenka@fnbrno.cz
- Contact Person Name
- Lenka Jurikova
- Contact Person Email
- jurikova.lenka@fnbrno.cz
Belgium
- Earliest CTIS Part Ii Submission Date
- 16-09-2024
- Latest Decision Or Authorization Date
- 24-10-2025
- Processing Time Days
- 403
- Number Of Sites
- 2
- Number Of Participants
- 5
Sites
- Site Name
- Universiteit Gent
- Department Name
- Neuromusculair referentiecentrum
- Principal Investigator Name
- Nicolas Deconinck
- Principal Investigator Email
- nicolas.deconinck@huderf.be
- Contact Person Name
- Nicolas Deconinck
- Contact Person Email
- nicolas.deconinck@huderf.be
- Site Name
- UZ Leuven
- Department Name
- Pediatric Neurology
- Principal Investigator Name
- Liesbeth De Waele
- Principal Investigator Email
- Liesbeth.dewaele@uzleuven.be
- Contact Person Name
- Liesbeth De Waele
- Contact Person Email
- Liesbeth.dewaele@uzleuven.be
Spain
- Earliest CTIS Part Ii Submission Date
- 06-09-2024
- Latest Decision Or Authorization Date
- 30-09-2025
- Processing Time Days
- 389
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Hospital Universitario Puerta De Hierro De Majadahonda
- Department Name
- Pediatrics and Neonatology
- Principal Investigator Name
- Gemma Iglesias Escalera
- Principal Investigator Email
- gemma.iglesias@salud.madrid.org
- Contact Person Name
- Gemma Iglesias Escalera
- Contact Person Email
- gemma.iglesias@salud.madrid.org
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Pediatrics
- Principal Investigator Name
- Immaculada Pitarch Castellano
- Principal Investigator Email
- pitarch_inmcas@gva.es
- Contact Person Name
- Immaculada Pitarch Castellano
- Contact Person Email
- pitarch_inmcas@gva.es
Ireland
- Earliest CTIS Part Ii Submission Date
- 21-01-2025
- Latest Decision Or Authorization Date
- 08-01-2026
- Processing Time Days
- 352
- Number Of Sites
- 2
- Number Of Participants
- 1
Sites
- Site Name
- Children's Health Ireland
- Department Name
- Department of Paediatric Neurodisability
- Principal Investigator Name
- Denise McDonald
- Principal Investigator Email
- Denise.Mcdonald@tuh.ie
- Contact Person Name
- Denise McDonald
- Contact Person Email
- Denise.Mcdonald@tuh.ie
- Site Name
- Children's Health Ireland
- Department Name
- Department of Paediatric Neurodisability
- Principal Investigator Name
- Denise McDonald
- Principal Investigator Email
- denise.mcdonald@tuh.ie
- Contact Person Name
- Denise McDonald
- Contact Person Email
- denise.mcdonald@tuh.ie
Sponsor
Primary sponsor
- Full Name
- Santhera Pharmaceuticals (Schweiz) AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Investigational products
- Investigational Product Name
- AGAMREE 40 mg/ml oral suspension
- Active Substance
- VAMOROLONE
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL USE
- Authorisation Status
- Authorised (marketing authorisation EU/1/23/1776/001)
- Orphan Designation
- Yes
- Maximum Dose
- 240 mg per day and up to 6 mg/kg (as listed: maxDailyDoseAmount 240 mg; maxTotalDoseAmount 6 mg/kg)
Related trials
Other published trials that may interest you.
- ENTR-601-44 for Duchenne muscular dystrophy
- EX VIVO FUSED NORMAL ALLOGENEIC HUMAN MYOBLAST WITH AUTOLOGOUS HUMAN MYOBLAST DERIVED FROM DUCHENNE MUSCULAR DYSTROPHY AFFECTED DONOR for Duchenne muscular dystrophy
- SAT-3247 OXALATE for Duchenne muscular dystrophy
- ENTR-601-45 for Duchenne muscular dystrophy
- DMD06-MAB for Duchenne muscular dystrophy