Muscular DystrophiesPhase 3NCT01803412

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

A Phase 3 study testing Drisapersen in people with Muscular Dystrophies, run by BioMarin Pharmaceutical at 13 sites in 2 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Who is it for?: Ages 5 years and older, men only
Conditions the study is for: Muscular Dystrophies
What is being tested?: Drisapersen
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 53 participants
Where: 13 sitesin 2 countries
Study dates: 1 May 2013to 1 Oct 2016
Phase: Phase 3
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 5 years and older
Men only
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Participation in an eligible drisapersen study as follows:
Prior DMD114876 subjects: Subjects who completed both the 24 week double-blind treatment and 24 week post-treatment phases in study DMD114876 OR Subjects who withdrew from the treatment portion of study DMD114876 due to meeting laboratory safety stopping criteria may be eligible to enrol in the extension study if: the laboratory parameters that led to stopping have resolved; the principal investigator (PI) considers the benefit of further treatment with drisapersen outweighs the risk to the individual subject; and following consultation with the Medical Monitor (B) Prior DMD114044 Subjects: US subjects who completed study DMD114044 in another country and who want to return to the US to participate in study DMD115501, upon agreement by a DMD115501 Investigator OR US citizens who participated in DMD114044 but who had to withdraw from the study due to meeting laboratory safety stopping criteria may be eligible to enrol in DMD115501 if: the laboratory parameters that led to stopping have resolved; the PI considers the benefit of further treatment with drisapersen outweighs the risk to the individual subject; and following consultation with the Medical Monitor and upon agreement by a DMD115501 investigator (C) Prior DMD114349 Subjects: US subjects who participated in and completed study DMD114044 in another country and who entered into the ongoing open-label extension study DMD114349 in a country outside the US who wish to withdraw from DMD114349 and return to the US to participate in study DMD115501, upon agreement by a DMD115501 investigator.
Canadian subjects who participated in the DMD114349 study OR Canadian subjects who withdrew from the treatment portion of the study DMD114349 due to meeting laboratory safety stopping criteria may be eligible to enroll in the extension study if the laboratory parameters that led to stopping have resolved; the PI considers the benefit of further treatment with drisapersen outweighs the risk to the individual subject; and following consultation with the Medical Monitor.
Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to or cessation of glucocorticoids will be at the discretion of the PI in consultation with the subject/parent and the Medical Monitor. If subject is not on steroids, involvement in the study needs to be discussed with the medical monitor.
Willing and able to comply with all protocol 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 signed by the subject and/or parent(s)/legal guardian (according to local regulations)

Who cannot join

Subject had a serious adverse experience or who met safety stopping criteria that remains unresolved from studies DMD114876, DMD114044, or DMD114349, which in the opinion of the investigator could have been attributable to study medication, and which is ongoing. Once resolved, subject may be eligible to enrol following PI consultation with the Medical Monitor
Use of anticoagulants, antithrombotics or antiplatelet agents, or previous treatment with investigational drugs except for drisapersen, within 28 days of the first administration of study medication
Participation in any other investigational clinical trial within 30 days prior to the start of screening, or during this clinical study. If subject has participated in any other study within 6 months, this should be discussed with the medical monitor prior to study entry.
History of significant medical disorder which may confound the interpretation of either efficacy or 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 Screening, the investigator should discuss inclusion of subject in the study with the medical monitor
A platelet count under the lower limit of normal at screening. A re-test within the screening period is permissible and if within normal range the subject may enter the study.

Trial locations and contacts

13 sites in 2 countries.

Country

City, postcode or hospital

13 sites in 2 countries

United States10 sites

Kennedy Krieger Institute
Baltimore, Maryland, 21205
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
Duke University Medical Center
Durham, North Carolina, 27710
University of Iowa
Iowa City, Iowa, 52242
University of Minnesota
Minneapolis, Minnesota, 55455
Columbia University Medical Center
New York, New York, 10032
Shriners Hospitals For Children
Portland, Oregon, 97239
UC Davis Medical Center
Sacramento, California, 95817
Shriner's Hospital for Children Tampa
Tampa, Florida, 12502

Other trials in United States

Canada3 sites

Children's Hospital London Health Sciences Centre
London, Ontario, N6A 4G5
CHU Ste-Justine
Montreal, Quebec, H3T 1C5
Children's & Women's Health Centre of BC
Vancouver, British Columbia, V6H 3V4

Other trials in Canada

About this study

Official title: An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in US and Canadian Subjects With Duchenne Muscular Dystrophy.

This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects with DMD open to eligible US and Canadian subjects who previously participated in the following studies of drisapersen: DMD114876, DMD114044 and DMD114349. Subjects will receive 6mg/kg subcutaneous drisapersen on a weekly basis. For subjects who have previously experienced significant safety or tolerability issues or who experience these during the study, there is the potential of an alternate intermittent dosing arm that will be given as a regimen of 6 mg/kg weekly for 8 weeks followed by 4 weeks off treatment. For subjects who experience or have previously experienced significant safety/tolerability issues, side effects or reactions or intermittent dosing, intravenous dosing will be made available.

Read the detailed description

In Part A of the study (DMD115501, original protocol), 21 subjects entered the study at 3 US sites and completed up to 14 weeks of treatment, and up to 22 weeks of follow-up. This protocol amendment, Part B of the study will include up to 13 more US and Canadian centers, and up to 51 more subjects. In total the study will enroll approximately 72 subjects. All subjects will commence Part B at screening and follow the study schedule.

The primary dosing arm is drisapersen 6 mg/kg as SC injection(s) once a week. For subjects who have previously experienced significant safety or tolerability issues or who experience these during the study, there is the potential of an alternate intermittent dosing arm that will be given as a regimen of 6 mg/kg/wk for 8 weeks followed by 4 weeks of treatment. For subjects who experience or have previously experienced significant safety/tolerability issues, intravenous dosing will be made available.

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 in this study until such a time that they withdraw based on protocol-defined criteria, or BioMarin stops the study.

Conditions: Muscular Dystrophies
Being tested: Drisapersen
Keywords: GSK2402968, open-label extension, safety, drisapersen, Duchenne Muscular Dystrophy, tolerability, Quality of Life
Medical subject headings: Muscular Dystrophies, Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

DrisapersenDrug
Also called BMN 051
Drisapersen will be supplied as 3 mL (milliliter) vials containing 1 mL sterile solution of 200 mg/mL strength. Each subject will receive drisapersen 3 mg/kg administered IV once a week, continuously throughout their duration of participation
DrisapersenDrug
Also called BMN 051
Drisapersen will be supplied as 3 mL (milliliter) vials containing 1 mL sterile solution of 220 mg/mL strength. Each subject will receive drisapersen 6 mg/kg administered SC once a week, either continuously or intermittently (for 8 weeks, followed by 4 weeks of no dosing) throughout their duration of participation
DrisapersenDrug
Also called BMN 051
Drisapersen will be supplied as 3 mL (milliliter) vials containing 1 mL sterile solution of 220 mg/mL strength. Each subject will receive drisapersen 6 mg/kg administered SC once a week, either continuously or intermittently (for 8 weeks, followed by 4 weeks of no dosing) throughout their duration of participation

Study groups in NCT01803412
Study group	Role	What they receive
Alternate Intravenous Dosing Arm	Experimental	Subjects will receive drisapersen as a regimen of 3 mg/kg over 1 hr IV weekly throughout the duration of participation. Receives: Drug: Drisapersen
Primary continuous Dosing Arm	Experimental	Subjects will receive drisapersen 6 mg/kg as SC injection(s) once a week, continuously throughout their duration of participation. Receives: Drug: Drisapersen
Alternate Intermittent Dosing Arm	Experimental	Subjects will receive drisapersen intermittently, as a regimen of 6 mg/kg as SC injection(s) once a week for 8 weeks followed by 4 weeks of no dosing, throughout their duration of participation. Receives: Drug: Drisapersen

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence and severity of Adverse Events (AEs)
AEs will be assessed from Pre-baseline visit until 20 weeks after the subject has either completed the study or withdrawn from treatment early. AEs are any untoward medical occurrence in a patient or clinical investigation subject, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product
Measured: Up to 48 weeks
Systolic and diastolic blood pressure measurements to assess the safety and tolerability
Measured: Up to 48 weeks
Pulse rate and respiratory rate measurements to assess the safety and tolerability
Measured: Up to 48 weeks
Body temperature measurements to assess the safety and tolerability
Measured: Up to 48 weeks
12-Lead Electrocardiogram (ECG) measurements to assess the safety and tolerability
The following parameters will be assessed: heart rate, intervals, corrected QT (QTc) interval (Bazett). In addition, an assessment of abnormal morphology will be made
Measured: Up to 48 weeks
Echocardiogram measurements to assess the safety and tolerability
The following parameters will be assessed: Left ventricular end-diastolic/end-systolic wall thickness (septum, posterior wall), fractional shortening (SF) and ejection fraction (LVEF) will be derived from M-mode (from the parasternal long-axis or short-axis view) for quantitative measurements
Measured: Up to 48 weeks
Laboratory tests to assess the safety and tolerability
Laboratory tests will include hematology, biochemistry and urinalysis parameters
Measured: Up to 48 weeks

Secondary outcomes (5)

Muscle function assessment using 6-minute walking distance (6MWD) test
Subjects will be asked to walk, at their own preferred speed, up and down a fixed distance until they are told to stop after 6 minutes. The subjects are warned of the time and are told that they may stop earlier if they feel unable to continue. The total distance walked within 6 minutes (or until the subject stopped in case of early termination of the test) will be recorded in meters, as well as any falls. Subjects who became non-ambulant in the prior study or who become non-ambulant during this study will not be able to perform this
Measured: Up to 48 weeks
North Star Ambulatory Assessment (NSAA)
The NSAA is a functional scale devised from the Hammersmith Scale of Motor Ability specifically for use in ambulant children with DMD. It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). The scale assesses activities that are required for ambulatory activity and includes items that are rarely achieved in untreated DMD (jump, hop, raise head) as well as items that are known to progressively deteriorate over time (stand from a chair, walk)
Measured: Up to 48 weeks
Pulmonary function assessment
Non-invasive spirometry will be conducted to determine actual and percent values for Forced Vital Capacity (FVC) and Forced Expiratory Volume (FEV1)
Measured: Up to 48 weeks
Time to major disease milestones
Major disease milestones are defined as those events that occurred since the last time they were assessed and include the following muscular dystrophy-related milestones: Achilles tendon contracture, hamstring contracture, lumbar lordosis, limb skeletal deformity, loss of ambulation, respiratory support during the day, respiratory support during sleep, scoliosis, use of leg braces, use of orthoses, use of special shoes, using Gower's maneuver or other milestone (to be specified)
Measured: Up to 48 weeks
Functional Outcomes assessment
One assessments will be completed to observe the changes in the ability of the subject to perform usual day-to-day activities during the study: Functional Outcomes Survey - by the family/caregivers who attends the scheduled clinic visit.
Measured: Up to 48 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 May 20131 Oct 2016

First posted4 Mar 2013 (estimated)
Study start1 May 2013
Primary completion1 Jun 2016
Study completion1 Oct 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Muscular DystrophiesStudies of the same condition, open and under way.
More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 53 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01803412
Sponsor's protocol ID: DMD115501
Status last verified: Jun 2018
First submitted: 28 Feb 2013
Last updated: 14 Jun 2018
Results posted: No
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 3
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1), DM1
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingObservational study
Preclinical Assessment of an Engineered HMGB1 Protein as a Novel Companion Therapy for Muscular Dystrophies
LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy

All Muscular Dystrophies trials

Study at a glance

From the registry record, last updated by the sponsor on 14 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in US and Canadian Subjects With Duchenne Muscular Dystrophy.
ClinicalTrials.gov ID: NCT01803412
Acronym / sponsor ID: DMD115501
Conditions: Muscular Dystrophies
Keywords: GSK2402968, open-label extension, safety, drisapersen, Duchenne Muscular Dystrophy, tolerability, Quality of Life
Phase: Phase 3
Study type: Interventional
Recruitment status: Terminated
Enrolment: 53 participants (actual)
Lead sponsor: BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
Study start: 1 May 2013 (actual)
Primary completion: 1 Jun 2016 (actual)
Study completion: 1 Oct 2016 (actual)
First posted: 4 Mar 2013 (estimated)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 3

Arm	Type	Receives
Alternate Intravenous Dosing Arm	Experimental	Drug: DrisapersenSubjects will receive drisapersen as a regimen of 3 mg/kg over 1 hr IV weekly throughout the duration of participation.
Primary continuous Dosing Arm	Experimental	Drug: DrisapersenSubjects will receive drisapersen 6 mg/kg as SC injection(s) once a week, continuously throughout their duration of participation.
Alternate Intermittent Dosing Arm	Experimental	Drug: DrisapersenSubjects will receive drisapersen intermittently, as a regimen of 6 mg/kg as SC injection(s) once a week for 8 weeks followed by 4 weeks of no dosing, throughout their duration of participation.

Who may qualify

Ages: 5 years and older
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (6 listed)

Participation in an eligible drisapersen study as follows:
Prior DMD114876 subjects: Subjects who completed both the 24 week double-blind treatment and 24 week post-treatment phases in study DMD114876 OR Subjects who withdrew from the treatment portion of study DMD114876 due to meeting laboratory safety stopping criteria may be eligible to enrol in the extension study if: the laboratory parameters that led to stopping have resolved; the principal investigator (PI) considers the benefit of further treatment with drisapersen outweighs the risk to the individual subject; and following consultation with the Medical Monitor (B) Prior DMD114044 Subjects: US subjects who completed study DMD114044 in another country and who want to return to the US to participate in study DMD115501, upon agreement by a DMD115501 Investigator OR US citizens who participated in DMD114044 but who had to withdraw from the study due to meeting laboratory safety stopping criteria may be eligible to enrol in DMD115501 if: the laboratory parameters that led to stopping have resolved; the PI considers the benefit of further treatment with drisapersen outweighs the risk to the individual subject; and following consultation with the Medical Monitor and upon agreement by a DMD115501 investigator (C) Prior DMD114349 Subjects: US subjects who participated in and completed study DMD114044 in another country and who entered into the ongoing open-label extension study DMD114349 in a country outside the US who wish to withdraw from DMD114349 and return to the US to participate in study DMD115501, upon agreement by a DMD115501 investigator.
Canadian subjects who participated in the DMD114349 study OR Canadian subjects who withdrew from the treatment portion of the study DMD114349 due to meeting laboratory safety stopping criteria may be eligible to enroll in the extension study if the laboratory parameters that led to stopping have resolved; the PI considers the benefit of further treatment with drisapersen outweighs the risk to the individual subject; and following consultation with the Medical Monitor.
Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to or cessation of glucocorticoids will be at the discretion of the PI in consultation with the subject/parent and the Medical Monitor. If subject is not on steroids, involvement in the study needs to be discussed with the medical monitor.
Willing and able to comply with all protocol requirements and procedures (with the exception of those assessments requiring a subject to be ambulant, for those subjects who have lost ambulation)

Key exclusion criteria (6 listed)

Subject had a serious adverse experience or who met safety stopping criteria that remains unresolved from studies DMD114876, DMD114044, or DMD114349, which in the opinion of the investigator could have been attributable to study medication, and which is ongoing. Once resolved, subject may be eligible to enrol following PI consultation with the Medical Monitor
Use of anticoagulants, antithrombotics or antiplatelet agents, or previous treatment with investigational drugs except for drisapersen, within 28 days of the first administration of study medication
Participation in any other investigational clinical trial within 30 days prior to the start of screening, or during this clinical study. If subject has participated in any other study within 6 months, this should be discussed with the medical monitor prior to study entry.
History of significant medical disorder which may confound the interpretation of either efficacy or 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 Screening, the investigator should discuss inclusion of subject in the study with the medical monitor

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
DrisapersenAlso: BMN 051Drisapersen will be supplied as 3 mL (milliliter) vials containing 1 mL sterile solution of 200 mg/mL strength. Each subject will receive drisapersen 3 mg/kg administered IV once a week, continuously throughout their duration of participation	Drug	Alternate Intravenous Dosing Arm
DrisapersenAlso: BMN 051Drisapersen will be supplied as 3 mL (milliliter) vials containing 1 mL sterile solution of 220 mg/mL strength. Each subject will receive drisapersen 6 mg/kg administered SC once a week, either continuously or intermittently (for 8 weeks, followed by 4 weeks of no dosing) throughout their duration of participation	Drug	Primary continuous Dosing Arm
DrisapersenAlso: BMN 051Drisapersen will be supplied as 3 mL (milliliter) vials containing 1 mL sterile solution of 220 mg/mL strength. Each subject will receive drisapersen 6 mg/kg administered SC once a week, either continuously or intermittently (for 8 weeks, followed by 4 weeks of no dosing) throughout their duration of participation	Drug	Alternate Intermittent Dosing Arm

Primary outcome

Incidence and severity of Adverse Events (AEs)
Time frame: Up to 48 weeks
AEs will be assessed from Pre-baseline visit until 20 weeks after the subject has either completed the study or withdrawn from treatment early. AEs are any untoward medical occurrence in a patient or clinical investigation subject, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product
Systolic and diastolic blood pressure measurements to assess the safety and tolerability
Time frame: Up to 48 weeks
Pulse rate and respiratory rate measurements to assess the safety and tolerability
Time frame: Up to 48 weeks
Body temperature measurements to assess the safety and tolerability
Time frame: Up to 48 weeks
12-Lead Electrocardiogram (ECG) measurements to assess the safety and tolerability
Time frame: Up to 48 weeks
The following parameters will be assessed: heart rate, intervals, corrected QT (QTc) interval (Bazett). In addition, an assessment of abnormal morphology will be made
Echocardiogram measurements to assess the safety and tolerability
Time frame: Up to 48 weeks
The following parameters will be assessed: Left ventricular end-diastolic/end-systolic wall thickness (septum, posterior wall), fractional shortening (SF) and ejection fraction (LVEF) will be derived from M-mode (from the parasternal long-axis or short-axis view) for quantitative measurements
Laboratory tests to assess the safety and tolerability
Time frame: Up to 48 weeks
Laboratory tests will include hematology, biochemistry and urinalysis parameters

5 secondary outcomes

Muscle function assessment using 6-minute walking distance (6MWD) test
Time frame: Up to 48 weeks
North Star Ambulatory Assessment (NSAA)
Time frame: Up to 48 weeks
Pulmonary function assessment
Time frame: Up to 48 weeks
Time to major disease milestones
Time frame: Up to 48 weeks
Functional Outcomes assessment
Time frame: Up to 48 weeks

Results and safety

The study was stopped early. Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 Jun 2018.

Related trials for Muscular Dystrophies

RecruitingPhase 3
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1), DM1
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingObservational study
Preclinical Assessment of an Engineered HMGB1 Protein as a Novel Companion Therapy for Muscular Dystrophies
LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

BioMarin Clinical Trials study director

BioMarin Pharmaceutical

13 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Drisapersen in people with Muscular Dystrophies, run by BioMarin Pharmaceutical at 13 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 14 Jun 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early. Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence and severity of Adverse Events (AEs) (time frame: Up to 48 weeks). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01803412.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jun 2018.