This is the extension study of NS-089/NCNP-02 (Study NCNP/DMT02), which is designed to assess the safety, tolerability and efficacy of NS-089/NCNP-02 in patients with Duchenne muscular dystrophy (DMD).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
6
The same dose administered in as Part 2 of Study NCNP/DMT02 (40 mg/kg) will be administered once weekly for 288 weeks (The total treatment period is 312 weeks including Part2 of Study NCNP/DMT02).
The same dose administered in as Part 2 of Study NCNP/DMT02 (80 mg/kg) will be administered once weekly for 288 weeks (The total treatment period is 312 weeks including Part2 of Study NCNP/DMT02).
Kagoshima University Hospital
Kagoshima, Japan
National Center of Neurology and Psychiatry
Tokyo, Japan
Incidence of adverse events
Time frame: Up to Week 319
Expression of dystrophin protein (Western blot)
Time frame: Week 99
Percentage of exon 44-skipped mRNA of dystrophin
Time frame: Week 99
North Star Ambulatory Assessment (NSAA)
Time frame: Up to Week 315
Time to stand test
Time frame: Up to Week 315
Time to run/walk 10 meters test
Time frame: Up to Week 315
Six-minute walk test/Two-minute walk test
Time frame: Up to Week 315
Timed Up & Go test
Time frame: Up to Week 315
Quantitative muscle strength assessment
Time frame: Up to Week 315
Performance of Upper Limb test
Time frame: Up to Week 315
Change in serum creatine kinase concentration from baseline
Time frame: Up to Week 315
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