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

Santhera

Status and phase: Enrolling; Phase 4

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: vamorolone 40 mg/mL oral suspension

Study type: Interventional

Funder types: Industry

Identifiers

NCT06713135

SNT-IV-VAM-011

Details and patient eligibility

About

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.

Full 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.

Enrollment: 80 estimated patients

Sex: Male

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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80 participants in 1 patient group

vamorolone

Experimental group

Description:

On Day 1, Subjects will roll over from a previous vamorolone program and continue treatment with vamorolone under this protocol. During the study, vamorolone will be 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.

Treatment:

Drug: vamorolone 40 mg/mL oral suspension