A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy

Phase 4Active Not RecruitingNCT06713135

Santhera Pharmaceuticals80 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

vamorolone 40 mg/mL oral suspensionDRUG

Vamorolone is administered at a dose range between 2 mg/kg/day and 6 mg/kg/day for boys weighing \<40 kg. For boys weighing 40 kg or above, the dose range will be 80 mg to 240 mg once daily. Doses can be adjusted within the dose range as determined by the Investigator based on tolerability. The highest tolerated dose should be used.

Eligibility

Sex: MALE

Medical Language ↔ Plain English

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

Locations (18)

UZ Gent (Universitair Ziekenhuis Gent)

Ghent, Belgium

UZ Leuven (Universitair Ziekenhuis Leuven)

Leuven, Belgium

Outcomes

Primary Outcomes

Number of vertebral fractures per 1000 person-years based on X-ray central reading.

Lateral thoracolumbar spine X-Rays will be collected and sent to a central reader for evaluation of vertebral fractures

Time frame: At Enrolment and every 2 years during a Full visit

Secondary Outcomes

Time to first vertebral fractures (cumulative incidence)

Time frame: From enrolment up to at least 2 years

Number of non-vertebral fractures per 1000 person-years based on investigator reporting

Non-vertebral fractures will be reported by investigators and not reviewed centrally

Time frame: From enrolment until up to at least 3 years

Time to first non-vertebral fractures (cumulative incidence)

Time frame: From enrolment until up to at least 3 years

Number of cataracts per 1000 person-years based on ophthalmologist assessment

An ophthalmologist assessment including assessment of posterior capsular cataracts by slit lamp will be performed yearly

Time frame: From enrolment until up to at least 3 years

Number of subjects not reaching Tanner stage 2 by 15 years of age

Qualified personnel (eg, an individual, part of the endocrinology team and trained to assess Tanner stages) will provide an assessment of the puberty status of the subject, including testicular volume.

Time frame: From enrolment until up to at least 3 years

Frequency of adverse events (AEs) and serious adverse events (SAEs)

The occurrence of AEs should be sought by questioning of the subject and/or his caregiver at each visit or phone call during the study. All SAEs occurring from signature of the ICF up to 30 days after last dose of study medication must be reported, irrespective of severity or whether or not considered related to study medication. All SAEs must be reported within 24 hours of becoming aware of the event, whether or not the SAE is considered to be related to the study medication.

Data from ClinicalTrials.gov

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