This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-089/NCNP-02 in subjects diagnosed with Duchenne muscular dystrophy (DMD), and to determine the dosage for subsequent studies.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
NS-089/NCNP-02 for Infusion is packaged as 50 mg/mL with 3 mL per vial. Study dosages will be infused over a 1 hour period at the following dose levels. "\[Part 1\] NS-089/NCNP-02 is administered at dose levels 1 and 3 in Cohort 1 and at dose levels 2 and 4 in Cohort 2. Dose level 1: 1.62 mg/kg once weekly for 2 weeks; Dose level 2: 10 mg/kg once weekly for 2 weeks; Dose level 3: 40 mg/kg once weekly for 2 weeks; Dose level 4: 80 mg/kg once weekly for 2 weeks \[Part 2\] Based on the results from Part 1, two dosages are selected as study dosages in Part 2. Each selected dose are administered once a week for 24 weeks."
National Center of Neurology and Psychiatry
Kodaira, Tokyo, Japan
Adverse event and adverse drug reaction [Safety and Tolerability]
adverse event and adverse drug reaction
Time frame: At the end of Part 2 (24 weeks treatment period and 12 weeks follow up period)
Expression of dystrophin protein
Expression of dystrophin protein
Time frame: At the end of the treatment period (24 weeks) of Part 2
NSAA
North Star Ambulatory Assessment
Time frame: At the end of the treatment period (24 weeks) of Part 2
TTSTAND
Time to Stand Test
Time frame: At the end of the treatment period (24 weeks) of Part 2
TTRW
Time to Run/Walk 10 Meters test
Time frame: At the end of the treatment period (24 weeks) of Part 2
6MWT and 2MWT
Six-Minute Walk Test (6MWT) and Two-Minute Walk Test (2MWT)
Time frame: At the end of the treatment period (24 weeks) of Part 2
TUG
Timed Up \& Go (TUG) test
Time frame: At the end of the treatment period (24 weeks) of Part 2
PUL
Performance of Upper Limb test
Time frame: At the end of the treatment period (24 weeks) of Part 2
Detection of exon 44-skipped mRNA of dystrophin in muscle tissue
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Detection of exon 44-skipped mRNA of dystrophin in muscle tissue
Time frame: At the end of the treatment period (24 weeks) of Part 2
NS-089/NCNP-02 concentration of the blood plasma
NS-089/NCNP-02 concentration of the blood plasma
Time frame: At the end of Part 2 (24 weeks treatment period and 12 weeks follow up period)
Serum Creatine kinase concentration
Serum Creatine kinase concentration
Time frame: At the end of Part 2 (24 weeks treatment period and 12 weeks follow up period)