Clinical trial NCT06713135: A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy
Official title: An Open-label Study to Collect Safety and Effectiveness Information on Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy Who Have Completed Prior Studies With Vamorolone
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne
vamorolone
santhera
long-term safety
Lead sponsor: Santhera Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2024-12-03
Enrollment: 80
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study aims to assess safety and effectivness of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy (DMD) who have completed prior studies with vamorolone.

Detailed description: All subjects in this study have completed previous studies with vamorolone and continued to receive vamorolone under special programs: Compassionate Use Program \[CUP\], Named Patient Program \[NPP\] or Expanded Access Protocol \[EAP\]. All subjects will continue treatment with vamorolone under Guardian protocol instead. The primary objective of this study is to evaluate the safety of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy regarding vertebral fractures. Secondary study objectives will evaluate the safety of long-term treatment with vamorolone on non-vertebral fractures, cataracts, delayed puberty, overall safety as well as ambulatory and non-ambulatory function.

Eligibility criteria: Inclusion Criteria:

* Subject and/or subject's parent(s) or legal guardian has provided written informed consent
* Subject has previously completed either the VBP15-LTE or VBP15-004 study, and transitioned through the Compassionate Use Program, Named Patient Program or Expanded Acess Protocol
* Subject is on vamorolone on day of enrolment
* Subject and parent / legal guardian are willing and able to comply with the protocol schedule, assessments and requirements

Exclusion Criteria:

* Any medical condition, which in the opinion of the Investigator, would affect study participation, performance or interpretation of study assessments
* 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
* Severe hepatic impairment

Primary outcomes:
measure: Number of vertebral fractures per 1000 person-years based on X-ray central reading.
timeFrame: At Enrolment and every 2 years during a Full visit
Secondary outcomes:
measure: Time to first vertebral fractures (cumulative incidence)
timeFrame: From enrolment up to at least 2 years
measure: Number of non-vertebral fractures per 1000 person-years based on investigator reporting
timeFrame: From enrolment until up to at least 3 years
measure: Time to first non-vertebral fractures (cumulative incidence)
timeFrame: From enrolment until up to at least 3 years
measure: Number of cataracts per 1000 person-years based on ophthalmologist assessment
timeFrame: From enrolment until up to at least 3 years
measure: Number of subjects not reaching Tanner stage 2 by 15 years of age
timeFrame: From enrolment until up to at least 3 years
measure: Frequency of adverse events (AEs) and serious adverse events (SAEs)
timeFrame: From enrolment until up to at least 3 years
measure: Change from baseline in body weight
timeFrame: From enrolment until up to at least 3 years
measure: Number of subjects with clinically relevant laboratory abnormalities
timeFrame: From enrolment until up to at least 3 years
measure: Change from baseline in Time to Stand (TTSTAND) velocity
timeFrame: From enrolment until up to at least 3 years
measure: Six-minute Walk Test (6MWT)
timeFrame: From enrolment until up to at least 3 years
measure: Change from baseline in 6MWT distance
timeFrame: From enrolment until up to at least 3 years
measure: Age at ambulatory and non-ambulatory milestones
timeFrame: From enrolment until up to at least 3 years
measure: North Star Ambulatory Assessment (NSAA) scores
timeFrame: At enrolment and each Full visit (Month 12, 24, 36, etc / End-of -Treatment) until End of Study
measure: Change from baseline in body height
timeFrame: From enrolment until up to at least 3 years
measure: Change from baseline in Body Mass Index (BMI)
timeFrame: From enrolment until up to at least 3 years
Other outcomes:
Locations:
city: Gent
country: Belgium
facility: UZ Gent (Universitair Ziekenhuis Gent)
city: Leuven
country: Belgium
facility: UZ Leuven (Universitair Ziekenhuis Leuven)
city: Brno
country: Czechia
facility: University Hospital Brno
city: Prague
country: Czechia
facility: Fakultni Nemocnice Motol
city: Madrid
country: Spain
facility: Hospital Universitario Puerta de Hierro Majadahonda
city: Valencia
country: Spain
facility: Hospital Universitario y Politecnico de La Fe
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital
state: Lanarkshire
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's Hospital
state: Merseyside
city: Leeds
country: United Kingdom
facility: Leeds Teaching Hospitals NHS Trust
state: West Yorkshire
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham NHS Foundation Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children NHS Foundation Trust
city: Newcastle
country: United Kingdom
facility: The John Walton Muscular Dystrophy Research Centre
Study officials:
affiliation: Leeds Teaching Hospital
name: A Child, MD
role: PRINCIPAL_INVESTIGATOR
References:
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