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

NCT ID: NCT06713135

Last Updated: 2026-01-09

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 80 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-11-10
• Study Completion Date: 2028-09-30

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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

vamorolone

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.

Group Type: EXPERIMENTAL

vamorolone 40 mg/mL oral suspension

Intervention Type: DRUG

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.

Interventions

vamorolone 40 mg/mL oral suspension

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.

Intervention Type: DRUG

Other Intervention Names

vamorolone

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

• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Santhera Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

A Child, MD

Role: PRINCIPAL_INVESTIGATOR

Leeds Teaching Hospital

Locations

UZ Gent (Universitair Ziekenhuis Gent)

Ghent, , Belgium

UZ Leuven (Universitair Ziekenhuis Leuven)

Leuven, , Belgium

University Hospital Brno

Brno, , Czechia

Fakultni Nemocnice Motol

Prague, , Czechia

Children's Hospital Agia Sofia

Athens, , Greece

Children's Health Ireland at Tallaght, Tallaght University Hospital

Dublin, , Ireland

Schneider Children's Medical Center

Petah Tikva, , Israel

Leiden University Medical Center

Leiden, , Netherlands

Radboud University Nijmegen

Nijmegen, , Netherlands

Te Wao Nui - Child Health Service, Wellington Hospital

Wellington, , New Zealand

Hospital Universitario Puerta de Hierro Majadahonda

Madrid, , Spain

Hospital Universitario y Politecnico de La Fe

Valencia, , Spain

Queen Elizabeth University Hospital

Glasgow, Lanarkshire, United Kingdom

Alder Hey Children's Hospital

Liverpool, Merseyside, United Kingdom

Leeds Teaching Hospitals NHS Trust

Leeds, West Yorkshire, United Kingdom

University Hospitals Birmingham NHS Foundation Trust

Birmingham, , United Kingdom

Great Ormond Street Hospital for Children NHS Foundation Trust

London, , United Kingdom

The John Walton Muscular Dystrophy Research Centre

Newcastle, , United Kingdom

Countries

Belgium; Czechia; Greece; Ireland; Israel; Netherlands; New Zealand; Spain; United Kingdom

References

Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans N, Haberlova J, Straub V, Mengle-Gaw LJ, Schwartz BD, Harper AD, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster R, McMillan HJ, Kuntz NL, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez-Padilla JJ, Nascimento-Osorio A, Niks EH, de Groot IJM, Katsalouli M, James MK, van den Anker J, Damsker JM, Ahmet A, Ward LM, Jaros M, Shale P, Dang UJ, Hoffman EP. Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA Neurol. 2022 Oct 1;79(10):1005-1014. doi: 10.1001/jamaneurol.2022.2480.

Reference Type: BACKGROUND

PMID: 36036925 (View on PubMed)

Dang UJ, Damsker JM, Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans NM, Haberlova J, Straub V, Mengle-Gaw L, Schwartz BD, Harper A, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster RI, Mcmillan HJ, Kuntz N, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez JJ, Nascimento-Osorio A, Niks EH, De Groot IJM, Katsalouli M, Van Den Anker JN, Ward LM, Leinonen M, D'Alessandro AL, Hoffman EP. Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial. Neurology. 2024 Mar 12;102(5):e208112. doi: 10.1212/WNL.0000000000208112. Epub 2024 Feb 9.

Reference Type: BACKGROUND

PMID: 38335499 (View on PubMed)

Hoffman EP, Schwartz BD, Mengle-Gaw LJ, Smith EC, Castro D, Mah JK, McDonald CM, Kuntz NL, Finkel RS, Guglieri M, Bushby K, Tulinius M, Nevo Y, Ryan MM, Webster R, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Siener C, Jaros M, Shale P, McCall JM, Nagaraju K, van den Anker J, Conklin LS, Cnaan A, Gordish-Dressman H, Damsker JM, Clemens PR; Cooperative International Neuromuscular Research Group. Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology. 2019 Sep 24;93(13):e1312-e1323. doi: 10.1212/WNL.0000000000008168. Epub 2019 Aug 26.

Reference Type: BACKGROUND

PMID: 31451516 (View on PubMed)

Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ; NorthStar UK Network and CINRG DNHS Investigators. Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial. JAMA Netw Open. 2022 Jan 4;5(1):e2144178. doi: 10.1001/jamanetworkopen.2021.44178.

Reference Type: BACKGROUND

PMID: 35076703 (View on PubMed)

Conklin LS, Damsker JM, Hoffman EP, Jusko WJ, Mavroudis PD, Schwartz BD, Mengle-Gaw LJ, Smith EC, Mah JK, Guglieri M, Nevo Y, Kuntz N, McDonald CM, Tulinius M, Ryan MM, Webster R, Castro D, Finkel RS, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, McCall JM, Hathout Y, Nagaraju K, van den Anker J, Ward LM, Ahmet A, Cornish MR, Clemens PR. Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug. Pharmacol Res. 2018 Oct;136:140-150. doi: 10.1016/j.phrs.2018.09.007. Epub 2018 Sep 13.

Reference Type: BACKGROUND

PMID: 30219580 (View on PubMed)

K. Nip, A. de Vera, S. Hasham, P. Charef, S. Wong. An open-label study to collect long-term safety and efficacy data from boys with DMD who have completed prior studies with vamorolone (GUARDIAN Study). Neuromuscular Disorders Volume 43, Supplement 1, October 2024, 104441.298

Reference Type: RESULT

Other Identifiers

SNT-IV-VAM-011

Identifier Type: -

Identifier Source: org_study_id