Extension Study of Drisapersen in DMD Subjects

NCT02636686 | Unknown

• 1 other identifier: BMN-051-302
• Study Type: expanded_access
• Target: N/A
• Locations: 20 countries
• Sites: 39

Brief Summary

This is a phase IIIb, multi-centre, open-label extension study in male subjects with DMD who previously have been treated with drisapersen, aiming at assessing the safety and efficacy of drisapersen.

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD; Duchenne Muscular Dystrophy; Drisapersen; Kyndrisa; exon-skipping; exon-51; BMN-051-302; 051-302

Interventions

Drisapersen DRUG

Subjects will receive 6 mg/kg of drisapersen by subcutaneous injection once weekly. If subjects have experienced an intolerable injection site reaction(s), in consultation with the investigator, the subject may be allowed intermittent injections (8 weeks on/4 weeks off) or weekly intravenous infusions of 3 or 6 mg/kg

Also known as: PRO051

Eligibility Criteria

• Age: 5 Years - 80 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Any subject who has been previously treated with an exon 51 skipping antisense oligonucleotide (drisapersen or eteplirsen) and is not eligible for another ongoing drisapersen study. Subjects who withdrew from the previous studies due to meeting laboratory safety stopping criteria may be eligible to enroll if:
• The laboratory parameters that led to stopping have resolved; benefit of further treatment with drisapersen outweighs the risk to the individual subject; and following consultation with the Medical Monitor.
• Subjects with DMD mutation/deletion within the dystrophin gene and correctable by drisapersen-induced DMD exon 51 skipping.
• Male subjects age \>5 at screening in whom the investigator considers treatment with drisapersen is likely to lead to improvement or prevent worsening of the condition.
• Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on glucocorticoids for the duration of this study. Changes to or cessation of glucocorticoids will be at the discretion of the investigator conducting this study in consultation with the subject/parent and Medical Monitor.
• Willing and able to comply with all study requirements and procedures (with the exception of those assessments requiring a subject to be ambulant, for those subjects who have lost ambulation).
• Able to give informed assent and/or consent in writing by the subject and/or parent(s)/legal guardian (according to local regulations)

You may not qualify if:

• Subjects who have previously been treated with drisapersen and who had a serious adverse experience or who met safety stopping criteria that remains unresolved, which in the opinion of the investigator could have been attributable to drisapersen. Once resolved, subject may be eligible to enter the study following investigator consultation with the Medical Monitor.
• Use of anticoagulants, anti-thrombotics or antiplatelet agents within 28 days of the first re-dosing of drisapersen. Chronic use of anticoagulants, anti-thrombotics or antiplatelet agents is prohibited during the study. As needed dosing (pro re nata - PRN) may be acceptable (except for aspirin) following discussion with the Medical Monitor.
• Participation in any investigational clinical trial within 3 months prior to start or during this study (except for other drisapersen studies). If subjects have participated in any other study within the last 6 months this should be discussed with the Medical Monitor prior to start of this study.
• History of significant medical disorder which may confound the interpretation of safety data (e.g. current or history of renal or liver disease/impairment, history of inflammatory illness)
• A platelet count under the lower limit of normal (LLN) at start of this study. A re-test is possible at a later stage, and if within normal range, the subject may enter the study.

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (39)

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

IMAI Research

Buenos Aires, C1425AWC, Argentina

Location

Royal Children's Hosital, Children's Neuroscience Centre

Parkville, Victoria, 3052, Australia

Location

Institute for Neuromuscular Research

Westmead, 2145, Australia

Location

Queen Fabiola Children's University Hospital

Brussels, 1020, Belgium

Location

Universitair Ziekenhuis Gent, Afdeling Neurologie

Ghent, 9000, Belgium

Location

Universitair Ziekenhuis Gasthuisberg

Leuven, 3000, Belgium

Location

Hôpital de La Citadelle, Centre de référence des Maladies

Liège, 4000, Belgium

Location

MHAT "Alexandrovska

Sofia, 1431, Bulgaria

Location

Detska Nemocnice

Brno, 613 00, Czechia

Location

FN Motol

Prague, Czechia

Location

CHU de Nantes - Hôtel Dieu

Nantes, 44093, France

Location

Hopital Armand Trousseau

Paris, 75571, France

Location

Centre hospitalier de Pau

Pau, 64000, France

Location

CHU de Toulouse - Hôpital des Enfants

Toulouse, 31059, France

Location

Dr. von Haunersches Kinderspital

Bayern, Muenchen, 80337, Germany

Location

Universitaetsklinikum Essen

Essen, 45122, Germany

Location

Universitaetsklinikum Freiburg

Freiburg im Breisgau, 79106, Germany

Location

Hadassah, Hebrew University Medical Center

Jerusalem, 91240, Israel

Location

Azienda Universitaria Ospedaliera

Messina, 98125, Italy

Location

IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena

Milan, 20122, Italy

Location

IRCCS Ospedale Pediatrico Bambino Gesù

Roma, 00165, Italy

Location

Fondazione IRCCS Policlinico Gemelli

Roma, 00168, Italy

Location

Kobe University Hospital

Hyōgo, 650-0017, Japan

Location

Kumamoto University Hospital

Kumamoto, 860-8556, Japan

Location

National Hospital Organization

Saitama, 349-0196, Japan

Location

National Center Hospital of Neurology and Psychiatry

Tokyo, Japan

Location

Leiden University Medical Center

Leiden, 2333 ZA, Netherlands

Location

UMCN St. Radboud

Nijmegen, 6525 GA, Netherlands

Location

Oslo Universitetssykehus

Oslo, 0027, Norway

Location

SPCSK Uniwersytet Medyczny w

Warsaw, 02-097, Poland

Location

Moscow Pediatrics and Children

Moscow, 125412, Russia

Location

Seoul National University Children's Hospital

Seoul, 110-744, South Korea

Location

Hospital Sant Joan de Deu

Barcelona, 08950, Spain

Location

Hospital Infantil La Paz

Madrid, 28046, Spain

Location

Hospital Universitari la Fe

Valencia, 46009, Spain

Location

Kaohsiung Medical University Hospital

Kaohsiung City, 80708, Taiwan

Location

Hacettepe Children's Hospsital

Ankara, 06100, Turkey (Türkiye)

Location

UCL Institute of Child Health

London, WC1N 1EH, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

PRO051

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

Study Officials

• Derry Ridgway, MD

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 9, 2015
• First Posted: December 22, 2015
• Last Updated: January 24, 2018

Record last verified: 2018-01

Locations

RU (1); NL (2); IL (1); AU (2); BU (1); ES (3); AR (1); US (1); NO (1); IT (4); PL (1); KR (1); TU (1); CZ (2); JP (4); TW (1); DE (3); BE (4); FR (4); GB (1)