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

NCT06713135 | Active Not Recruiting Phase 4

• 1 other identifier: SNT-IV-VAM-011
• Study Type: interventional
• Target: 80
• Locations: 9 countries
• Sites: 18

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne; vamorolone; santhera; long-term safety

Outcome Measures

Primary Outcomes (1)

• 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

  At Enrolment and every 2 years during a Full visit

Secondary Outcomes (15)

• Time to first vertebral fractures (cumulative incidence)

  From enrolment up to at least 2 years

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

  From enrolment until up to at least 3 years

• Time to first non-vertebral fractures (cumulative incidence)

  From enrolment until up to at least 3 years

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

  From enrolment until up to at least 3 years

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

  From enrolment until up to at least 3 years

Study Arms (1)

vamorolone

EXPERIMENTAL

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.

Drug: vamorolone 40 mg/mL oral suspension

Interventions

vamorolone 40 mg/mL oral suspension 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.

Also known as: vamorolone

vamorolone

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Santhera Pharmaceuticals: lead

Study Sites (18)

UZ Gent (Universitair Ziekenhuis Gent)

Ghent, 9000, Belgium

Location

UZ Leuven (Universitair Ziekenhuis Leuven)

Leuven, 3000, Belgium

Location

University Hospital Brno

Brno, Czechia

Location

Fakultni Nemocnice Motol

Prague, 150 06, Czechia

Location

Children's Hospital Agia Sofia

Athens, 115 27, Greece

Location

Children's Health Ireland at Tallaght, Tallaght University Hospital

Dublin, D24TN3C, Ireland

Location

Schneider Children's Medical Center

Petah Tikva, 4920235, Israel

Location

Leiden University Medical Center

Leiden, 2333 ZA, Netherlands

Location

Radboud University Nijmegen

Nijmegen, 6525 HB, Netherlands

Location

Te Wao Nui - Child Health Service, Wellington Hospital

Wellington, 6021, New Zealand

Location

Hospital Universitario Puerta de Hierro Majadahonda

Madrid, 28222, Spain

Location

Hospital Universitario y Politecnico de La Fe

Valencia, 46026, Spain

Location

Queen Elizabeth University Hospital

Glasgow, Lanarkshire, United Kingdom

Location

Alder Hey Children's Hospital

Liverpool, Merseyside, L14 5AB, United Kingdom

Location

Leeds Teaching Hospitals NHS Trust

Leeds, West Yorkshire, LS1 3EX, United Kingdom

Location

University Hospitals Birmingham NHS Foundation Trust

Birmingham, B9 5SS, United Kingdom

Location

Great Ormond Street Hospital for Children NHS Foundation Trust

London, WC1N 3JH, United Kingdom

Location

The John Walton Muscular Dystrophy Research Centre

Newcastle, NE1 3BZ, United Kingdom

Location

Related Publications (6)

• 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.

  PMID: 36036925 BACKGROUND

• 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.

  PMID: 38335499 BACKGROUND

• 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.

  PMID: 31451516 BACKGROUND

• 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.

  PMID: 35076703 BACKGROUND

• 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.

  PMID: 30219580 BACKGROUND

• 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

  RESULT

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

VBP15 compound; Suspensions

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Colloids; Complex Mixtures; Dosage Forms; Pharmaceutical Preparations

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2024
• First Posted: December 3, 2024
• Study Start: November 10, 2024
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): September 1, 2028
• Last Updated: April 23, 2026

Record last verified: 2026-04

Locations

ES (2); CZ (2); GR (1); BE (2); NL (2); NZ (1); IL (1); GB (6); IE (1)