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Extension Study of Drisapersen in DMD Subjects

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02636686
Expanded Access Status : No longer available (Due to a company decision to stop the development of exon-skipping Duchenne muscular dystrophy therapy.)
First Posted : December 22, 2015
Last Update Posted : January 24, 2018
Information provided by (Responsible Party):
BioMarin Pharmaceutical

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.

Condition or disease Intervention/treatment
Duchenne Muscular Dystrophy Drug: Drisapersen

Detailed Description:

This is a phase IIIb, multi-centre, open-label extension study in male subjects with DMD who have previously been treated with drisapersen.

This study aims to enroll up to approximately 220 subjects. The primary dosing arm is drisapersen 6 mg/kg as subcutaneous (SC) injection(s) once a week. All subjects starting with subcutaneous injections will receive a loading dose of twice weekly 6mg/kg drisapersen for the first three weeks of treatment. This study does not have a minimum duration of participation. Subjects will have varying times of study participation depending on when they enter from one of the eligible studies and will be permitted to continue the study until such a time that they withdraw based on protocol-defined criteria, or BioMarin stops the study. Subjects naïve to treatment are not eligible for participation in this study

For subjects who have previously experienced significant safety or tolerability issues in one of the eligible studies, or who experience these during this study, there is the potential of an alternate intermittent dosing arm. This will be agreed in advance with the Medical Monitor.

For subjects who have previously experienced significant injection site reactions in an earlier drisapersen study, or who experience similar reaction(s) during this study, there is the potential to be dosed intravenously.

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
Official Title: An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in Subjects With Duchenne Muscular Dystrophy.

Intervention Details:
  • Drug: Drisapersen
    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
    Other Name: PRO051

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 80 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. 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:
  2. 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.
  3. Subjects with DMD mutation/deletion within the dystrophin gene and correctable by drisapersen-induced DMD exon 51 skipping.
  4. 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.
  5. 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.
  6. 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).
  7. Able to give informed assent and/or consent in writing by the subject and/or parent(s)/legal guardian (according to local regulations)

Exclusion Criteria:

  1. 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.
  2. 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.
  3. 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.
  4. 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)
  5. 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.
  6. 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02636686

  Hide Study Locations
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United States, Maryland
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
IMAI Research
Buenos Aires, Argentina, C1425AWC
Australia, Victoria
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
MHAT "Alexandrovska
Sofia, Bulgaria, 1431
Detska Nemocnice
Brno, Czechia, 613 00
FN Motol
Praha 5, Czechia
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
Universitaetsklinikum Essen
Essen, Germany, 45122
Universitaetsklinikum Freiburg
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
Tokyo, Japan
Korea, Republic of
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 Universitetssykehus
Oslo, Norway, 0027
SPCSK Uniwersytet Medyczny w
Warszawa, Poland, 02-097
Russian Federation
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
UCL Institute of Child Health
London, United Kingdom, WC1N 1EH
Sponsors and Collaborators
BioMarin Pharmaceutical
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Study Director: Derry Ridgway, MD BioMarin Pharmaceutical

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Responsible Party: BioMarin Pharmaceutical Identifier: NCT02636686     History of Changes
Other Study ID Numbers: BMN-051-302
First Posted: December 22, 2015    Key Record Dates
Last Update Posted: January 24, 2018
Last Verified: January 2018
Keywords provided by BioMarin Pharmaceutical:
Duchenne Muscular Dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked