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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02740972 |
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Date of registration:
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23/03/2016 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
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Scientific title:
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Date of first enrolment:
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December 2016 |
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Target sample size:
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16 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02740972 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 2
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Countries of recruitment
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Canada
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United States
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Contacts
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Name:
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Paula R. Clemens, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Pittsburgh |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Male = 4 years and <10 years of age
- Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon
53 to restore the dystrophin mRNA reading frame;
- Able to walk independently without assistive devices;
- Ability to complete the time to stand, time to run/walk and time to climb assessments;
- Stable dose of glucocorticoid for at least 3 months
Exclusion Criteria:
- Acute illness within 4 weeks prior to the first dose of study medication;
- Evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on
investigation would not be exclusionary];
- Severe allergy or hypersensitivity to medications;
- Severe behavioral or cognitive problems that preclude participation in the study, in
the opinion of the Investigator;
- Previous or ongoing medical condition, medical history, physical findings or
laboratory abnormalities that could affect safety, make it unlikely that treatment and
follow-up will be correctly completed or impair the assessment of study results, in
the opinion of the Investigator;
- Patient is taking any other investigational drug currently or within 3 months prior to
the start of study treatment; or
- Patient has had surgery within the 3 months prior to the first anticipated
administration of study medication or surgery is planned for anytime during the
duration of the study;
- Patient has previously participated in this study or any other study during which
NS-065/NCNP-01 was administered.
Age minimum:
4 Years
Age maximum:
9 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Duchenne Muscular Dystrophy
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Intervention(s)
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Drug: NS-065/NCNP-01
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Drug: Placebo
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Primary Outcome(s)
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Incidence of Adverse Events as assessed by CTCAE v4.0.
[Time Frame: 24 weeks of treatment phase]
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Dystrophin protein in muscle as measured by western blot.
[Time Frame: 20-24 weeks of treatment]
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Drug concentration in plasma.
[Time Frame: 20-24 weeks of treatment]
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Secondary Outcome(s)
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Change in Time to Climb 4 Stairs (TTCLIMB).
[Time Frame: baseline to 20-24 weeks of treatment]
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Change in Time to Run/Walk 10 meters (TTRW).
[Time Frame: baseline to 20-24 weeks of treatment]
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Induction of dystrophin messenger ribonucleic acid (mRNA) in muscle as measured by real time polymerase chain reaction (RT-PCR) for mRNA analysis.
[Time Frame: 20-24 weeks of treatment]
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Muscle strength as measured by Quantitative Muscle Testing (QMT).
[Time Frame: 20-24 weeks of treatment]
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Change in distance traveled in the Six-Minute Walk Test (6MWT).
[Time Frame: baseline to 20-24 weeks of treatment]
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Change in Time to Stand (TTSTAND)
[Time Frame: baseline to 20-24 weeks of treatment]
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Induction of dystrophin protein in muscle as measured by mass spectrometry and immunofluorescence staining methods for protein analysis.
[Time Frame: 20-24 weeks of treatment]
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North Star Ambulatory Assessment (NSAA) results.
[Time Frame: baseline to 20-24 weeks of treatment]
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Secondary ID(s)
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NS-065/NCNP-01-201
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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