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
We will connect you to the doctor responsible for this trial and help you get more information.
Locations near you
Unfortunately, there are no recruiting locations near you. Please check the list with all locations below.
Full eligibility criteria for NCT02636686
Ages eligible for Study
5 Years to 80 Years
Genders eligible for Study
Accepts Healthy Volunteers
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)
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)
Symptomatic cardiomyopathy. If subject has a left ventricular ejection fraction <45% at start of this study, the investigator should discuss inclusion of subject in this study with the Medical Monitor.
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.
All locations for NCT02636686
United States (1)
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Buenos Aires, Argentina, C1425AWC
Royal Children's Hosital, Children's Neuroscience Centre
Parkville, Victoria, Australia, 3052
Institute for Neuromuscular Research
Westmead, Australia, 2145
Queen Fabiola Children's University Hospital
Brussels, Belgium, 1020
Universitair Ziekenhuis Gent, Afdeling Neurologie
Gent, Belgium, 9000
Universitair Ziekenhuis Gasthuisberg
Leuven, Belgium, 3000
Hôpital de La Citadelle, Centre de référence des Maladies
Liege, Belgium, 4000
Sofia, Bulgaria, 1431
CHU de Nantes - Hôtel Dieu
Nantes cedex 01, France, 44093
Hopital Armand Trousseau
Paris Cedex 12, France, 75571
Centre hospitalier de Pau
Pau, France, 64000
CHU de Toulouse - Hôpital des Enfants
Toulouse cedex 9, France, 31059
Dr. von Haunersches Kinderspital
Bayern, Muenchen, Germany, 80337
Essen, Germany, 45122
Freiburg, Germany, 79106
Hadassah, Hebrew University Medical Center
Jerusalem, Israel, 91240
Azienda Universitaria Ospedaliera
Messina, Italy, 98125
IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
Milano, Italy, 20122
IRCCS Ospedale Pediatrico Bambino Gesù
Roma, Italy, 00165
Fondazione IRCCS Policlinico Gemelli
Roma, Italy, 00168
Kobe University Hospital
Hyogo, Japan, 650-0017
Kumamoto University Hospital
Kumamoto, Japan, 860-8556
National Hospital Organization
Saitama, Japan, 349-0196
National Center Hospital of Neurology and Psychiatry
Korea, Republic of (1)
Seoul National University Children's Hospital
Seoul, Korea, Republic of, 110-744
Leiden University Medical Center
Leiden, Netherlands, 2333 ZA
UMCN St. Radboud
Nijmegen, Netherlands, 6525 GA
Oslo, Norway, 0027
SPCSK Uniwersytet Medyczny w
Warszawa, Poland, 02-097
Russian Federation (1)
Moscow Pediatrics and Children
Moscow, Russian Federation, 125412
Hospital Sant Joan de Deu
Barcelona, Spain, 08950
Hospital Infantil La Paz
Madrid, Spain, 28046
Hospital Universitari la Fe
Valencia, Spain, 46009
Kaohsiung Medical University Hospital
Kaohsiung, Taiwan, 80708
Hacettepe Children's Hospsital
Ankara, Turkey, 06100
United Kingdom (1)
UCL Institute of Child Health
London, United Kingdom, WC1N 1EH
Brno, Czechia, 613 00
Praha 5, Czechia
View full eligibility
Tris trial is registered with FDA with number: NCT02636686. The sponsor of the trial is BioMarin Pharmaceutical and it is looking for 0 volunteers for the current phase.
Official trial title: An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in Subjects With Duchenne Muscular Dystrophy.
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