Duchenne Muscular DystrophyPhase 2NCT02329769
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
A Phase 2 study testing PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg and PRO044 IV 9 mg/kg in people with Duchenne Muscular Dystrophy, run by BioMarin Pharmaceutical at 5 sites in 4 countries.
Trial summary
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein. Read more about this study
- Who is it for?
- Ages 9 to 20 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg and PRO044 IV 9 mg/kg
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 5 sitesin 4 countries
- Study dates
- Dec 2014to 31 Aug 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 9 to 20 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects previously treated with PRO044.
- 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 the dose regimen or cessation of glucocorticoids will be at the discretion of the Principle Investigator (PI) in consultation with the subject/parent and the Medical Monitor. If the subject is not on steroids, involvement in the study needs to be discussed with the medical monitor
Who cannot join
- Current, or history of, liver or renal disease.
- Acute illness within 4 weeks prior to the first dose of PRO044 (Week 1) which may interfere with the measurements.
- Severe cardiac myopathy which in the opinion of the Investigator prohibits participation in this study
- Need for daytime mechanical ventilation.
- Screening aPTT above the upper limit of normal (ULN).
- Screening platelet count below the lower limit of normal (LLN).
Show all 9Show fewer
- Use of anticoagulants, antithrombotics or antiplatelet agents.
- Use of any investigational product within 6 months prior to the start of Screening for the study.
- 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 Universitario Agostino Gemelli
Roma
About this study
Official title: A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular Dystrophy
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
Read the detailed description
A Phase II, open-label, extesion study. Following a Screening period of up to one month, subjects previously treated with PRO044, and eligible for enrolment in PRO044-CLIN-02, will be allocated to one of three groups to receive either 6 mg/kg or 9 mg/kg PRO044 weekly by IV infusion or 6 mg/kg weekly by SC injection for 48 weeks.
Safety and tolerability, pharmacokinetics (PK), pharmacodynamic (PD) and efficacy assessments will be conducted at regular intervals throughout the study.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg
- Keywords
- Duchenne muscular dystrophy, DMD, BioMarin, PRO044
- 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.
PRO044 SC 6 mg/kgDrug
PRO044 IV 6 mg/kgDrug
PRO044 IV 9 mg/kgDrug
| Study group | Role | What they receive |
|---|---|---|
| PRO044 SC 6 mg/kg | Experimental | Weekly subcutaneous (SC) dosing with 6 mg/kg Receives: Drug: PRO044 SC 6 mg/kg |
| PRO044 IV 6 mg/kg | Experimental | Weekly intravenous (IV) dosing with 6 mg/kg Receives: Drug: PRO044 IV 6 mg/kg |
| PRO044 SC 9 mg/kg | Experimental | Weekly intravenous (IV) dosing with 9 mg/kg Receives: Drug: PRO044 IV 9 mg/kg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of PRO044 (composite of several measures)
Efficacy parameters:
Muscle Function
- 6 Minute Walk Distance (6MWD)
- North Star Ambulatory Assessment
- Timed tests (10-meter walk/run, rising from floor, stair climb)
- DMD Functional Outcomes Questionnaire (DMD-FOS) -for ambulant subjects only
- Egen Klassification - for non-ambulant subjects.
Muscle strength
- Pulmonary Function (Spirometry)
- Handheld myometry.
Exploratory:
- Performance Upper Limb (PUL).
- Patient Reported Outcome measure (PROM).
Measured: After 48 weeks of treatment
Safety and tolerability of PRO044 (treatement emergent adverse events)
Number of subjects with 1 or more treatement emergent adverse events following SC or IV PRO044 dosing
Measured: After 48 weeks of treatment
Secondary outcomes (1)
Assess the pharmacokinetics of PRO044 (composite of several measures)
Pharmacokinetic parameters:
- t ½
- AUC: 0-24h, 0-∞ (where applicable)
- Cmax
- tmax
- CL (for IV subjects) or CL/F (for SC subjects)
- PRO044 concentrations in muscle tissue.
Measured: After 48 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jan 2015 (estimated)
- Study startDec 2014
- Primary completion1 Jul 2016
- Study completion31 Aug 2016
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
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02329769
- Sponsor's protocol ID
- PRO044-CLIN-02
- Status last verified
- Dec 2017
- First submitted
- 22 Dec 2014
- Last updated
- 8 Dec 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02329769
- Acronym / sponsor ID
- PRO044-CLIN-02
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne muscular dystrophy, DMD, BioMarin, PRO044
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 15 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Dec 2014 (actual)
- Primary completion
- 1 Jul 2016 (actual)
- Study completion
- 31 Aug 2016 (actual)
- First posted
- 1 Jan 2015 (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 purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
Detailed description
A Phase II, open-label, extesion study. Following a Screening period of up to one month, subjects previously treated with PRO044, and eligible for enrolment in PRO044-CLIN-02, will be allocated to one of three groups to receive either 6 mg/kg or 9 mg/kg PRO044 weekly by IV infusion or 6 mg/kg weekly by SC injection for 48 weeks.
Safety and tolerability, pharmacokinetics (PK), pharmacodynamic (PD) and efficacy assessments will be conducted at regular intervals throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| PRO044 SC 6 mg/kg | Experimental | Drug: PRO044 SC 6 mg/kgWeekly subcutaneous (SC) dosing with 6 mg/kg |
| PRO044 IV 6 mg/kg | Experimental | Drug: PRO044 IV 6 mg/kgWeekly intravenous (IV) dosing with 6 mg/kg |
| PRO044 SC 9 mg/kg | Experimental | Drug: PRO044 IV 9 mg/kgWeekly intravenous (IV) dosing with 9 mg/kg |
Who may qualify
- Ages
- 9 to 20 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Subjects previously treated with PRO044.
- 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 the dose regimen or cessation of glucocorticoids will be at the discretion of the Principle Investigator (PI) in consultation with the subject/parent and the Medical Monitor. If the subject is not on steroids, involvement in the study needs to be discussed with the medical monitor
Key exclusion criteria (9 listed)
- Current, or history of, liver or renal disease.
- Acute illness within 4 weeks prior to the first dose of PRO044 (Week 1) which may interfere with the measurements.
- Severe cardiac myopathy which in the opinion of the Investigator prohibits participation in this study
- Need for daytime mechanical ventilation.
- Screening 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 |
|---|---|---|
| PRO044 SC 6 mg/kg | Drug | PRO044 SC 6 mg/kg |
| PRO044 IV 6 mg/kg | Drug | PRO044 IV 6 mg/kg |
| PRO044 IV 9 mg/kg | Drug | PRO044 SC 9 mg/kg |
Primary outcome
Efficacy of PRO044 (composite of several measures)
Time frame: After 48 weeks of treatment
Efficacy parameters: Muscle Function * 6 Minute Walk Distance (6MWD) * North Star Ambulatory Assessment * Timed tests (10-meter walk/run, rising from floor, stair climb) * DMD Functional Outcomes Questionnaire (DMD-FOS) -for ambulant subjects only * Egen Klassification - for non-ambulant subjects. Muscle strength * Pulmonary Function (Spirometry) * Handheld myometry. Exploratory: * Performance Upper Limb (PUL). * Patient Reported Outcome measure (PROM).
Safety and tolerability of PRO044 (treatement emergent adverse events)
Time frame: After 48 weeks of treatment
Number of subjects with 1 or more treatement emergent adverse events following SC or IV PRO044 dosing
1 secondary outcome
Assess the pharmacokinetics of PRO044 (composite of several measures)
Time frame: After 48 weeks of treatment
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 8 Dec 2017.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg and PRO044 IV 9 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 2 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 8 Dec 2017. 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. 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?
Efficacy of PRO044 (composite of several measures) (time frame: After 48 weeks of treatment). The study lists 2 primary outcomes in all. 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 8 Dec 2017.