Duchenne Muscular DystrophyPhase 2NCT02958202
Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
A Phase 2 study testing BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg and BMN 044 SC 6 mg/kg in people with Duchenne Muscular Dystrophy, run by BioMarin Pharmaceutical at 5 sites in 4 countries.
Trial summary
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a longer treatment period. Read more about this study
- Who is it for?
- Ages 5 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg and BMN 044 SC 6 mg/kg
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 5 sitesin 4 countries
- Study dates
- Apr 2016to Sep 2016
- Phase
- Phase 2
- 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
- Subjects previously treated with BMN 044 or a comparator treatment in a BMN 044 Sponsored Study or Investigator Initiated Trial and who are not eligible for another ongoing BMN 044 study.
- Continued use of glucocorticosteroids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on glucocorticosteroids for the duration of this study.
- Willing and able to comply with all study requirements and procedures.
- Willing and able to provide written, signed informed consent, or in the case of subjects under the age of 18 years(or 16 years, depending on the region), provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to the conduct of any research-related procedures.
Who cannot join
- Subjects who have previously been treated with BMN 044 who had a serious adverse experience or met safety stopping criteria, that remains unresolved, which in the opinion of the Investigator could have been attributable to BMN 044.
- History of significant medical disorder which may confound the interpretation of safety data
- Acute illness within 4 weeks prior to the first dose of BMN 044 (Week 1) which may interfere with the measurements.
- Symptomatic cardiomyopathy.
- Baseline aPTT above the upper limit of normal (ULN).
- Baseline platelet count below the lower limit of normal (LLN).
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- Use of anti coagulants, anti thrombotics or anti platelet agents within 28 days of the baseline visit.
- Prior use of any investigational product (other than BMN 044) or investigational medical device must be discussed with the Medical Monitor prior to screening.
- Current or history of drug and/or alcohol abuse.
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
Italy2 sites
S.Anna Hospital
Ferrara
Policlinico Univsersitario Agostino Gemelli
Rome
About this study
Official title: A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular Dystrophy
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a longer treatment period.
Read the detailed description
This is a phase 2 multi center, multi national, open label, long term extension study. Up to approximately 50 male subjects with Duchenne Muscular Dystrophy (DMD) who have previously been treated with BMN 044 will be enrolled. Subjects will receive either IV infusions or SC injections at pre-defined doses.
Safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy assessments will be conducted at regular intervals throughout the study.
Subjects will be permitted to continue in this study until the subject meets any of the defined withdrawal criteria, BioMarin decides to halt the clinical development of BMN 044, or BMN 044 receives marketing authorization.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg, BMN 044 SC 6 mg/kg
- Keywords
- Duchenne Muscular Dystrophy, DMD, Dystrophin, BMN 044, PRO044, BioMarin
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BMN 044 IV 6 mg/kgDrug
Also called PRO044
BMN 044 IV 9 mg/kgDrug
Also called PRO044
BMN 044 SC 6 mg/kgDrug
Also called PRO044
| Study group | Role | What they receive |
|---|---|---|
| BMN 044 IV 6 mg/kg | Experimental | Weekly intravenous (IV) dosing with 6 mg/kg Receives: Drug: BMN 044 IV 6 mg/kg |
| BMN 044 IV 9 mg/kg | Experimental | Weekly intravenous (IV) dosing with 9 mg/kg Receives: Drug: BMN 044 IV 9 mg/kg |
| BMN 044 SC 6 mg/kg | Experimental | Weekly subcutaneous (SC) dosing with 6 mg/kg Receives: Drug: BMN 044 SC 6 mg/kg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing
Measured: Through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2016 (estimated)
- Study startApr 2016
- Primary completionSep 2016
- Study completionSep 2016
Why it stopped: Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Paulatsya Joshi, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02958202
- Sponsor's protocol ID
- BMN-044-201
- Other IDs
- 2015-003681-87, 2015
- Status last verified
- Jan 2018
- First submitted
- 27 Oct 2016
- Last updated
- 26 Jan 2018
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02958202
- Acronym / sponsor ID
- BMN-044-201
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, DMD, Dystrophin, BMN 044, PRO044, BioMarin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD.
- Enrolment
- 7 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Apr 2016
- Primary completion
- Sep 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 8 Nov 2016 (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.
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a longer treatment period.
Detailed description
This is a phase 2 multi center, multi national, open label, long term extension study. Up to approximately 50 male subjects with Duchenne Muscular Dystrophy (DMD) who have previously been treated with BMN 044 will be enrolled. Subjects will receive either IV infusions or SC injections at pre-defined doses.
Safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy assessments will be conducted at regular intervals throughout the study.
Subjects will be permitted to continue in this study until the subject meets any of the defined withdrawal criteria, BioMarin decides to halt the clinical development of BMN 044, or BMN 044 receives marketing authorization.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| BMN 044 IV 6 mg/kg | Experimental | Drug: BMN 044 IV 6 mg/kgWeekly intravenous (IV) dosing with 6 mg/kg |
| BMN 044 IV 9 mg/kg | Experimental | Drug: BMN 044 IV 9 mg/kgWeekly intravenous (IV) dosing with 9 mg/kg |
| BMN 044 SC 6 mg/kg | Experimental | Drug: BMN 044 SC 6 mg/kgWeekly subcutaneous (SC) dosing with 6 mg/kg |
Who may qualify
- Ages
- 5 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Subjects previously treated with BMN 044 or a comparator treatment in a BMN 044 Sponsored Study or Investigator Initiated Trial and who are not eligible for another ongoing BMN 044 study.
- Continued use of glucocorticosteroids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on glucocorticosteroids for the duration of this study.
- Willing and able to comply with all study requirements and procedures.
- Willing and able to provide written, signed informed consent, or in the case of subjects under the age of 18 years(or 16 years, depending on the region), provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to the conduct of any research-related procedures.
Key exclusion criteria (9 listed)
- Subjects who have previously been treated with BMN 044 who had a serious adverse experience or met safety stopping criteria, that remains unresolved, which in the opinion of the Investigator could have been attributable to BMN 044.
- History of significant medical disorder which may confound the interpretation of safety data
- Acute illness within 4 weeks prior to the first dose of BMN 044 (Week 1) which may interfere with the measurements.
- Symptomatic cardiomyopathy.
- Baseline aPTT above the upper limit of normal (ULN).
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 |
|---|---|---|
| BMN 044 IV 6 mg/kgAlso: PRO044 | Drug | BMN 044 IV 6 mg/kg |
| BMN 044 IV 9 mg/kgAlso: PRO044 | Drug | BMN 044 IV 9 mg/kg |
| BMN 044 SC 6 mg/kgAlso: PRO044 | Drug | BMN 044 SC 6 mg/kg |
Primary outcome
Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing
Time frame: Through study completion, an average of 1 year
Results and safety
The study was stopped early (reason given: Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2018.
- Individual participant dataSharing planned: No
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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.
Paulatsya Joshi study director
BioMarin Pharmaceutical
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg and BMN 044 SC 6 mg/kg in people with Duchenne Muscular Dystrophy, run by BioMarin Pharmaceutical at 5 sites in 4 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 4 inclusion and 9 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 26 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Italy, Belgium, Netherlands, Sweden. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 (reason given: Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD.). 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?
Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing (time frame: Through study completion, an average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 26 Jan 2018.