UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000038505 |
|---|---|
| Receipt number | R000043651 |
| Scientific Title | Exploratory Study of NS-089/NCNP-02 in Duchenne muscular dystrophy |
| Date of disclosure of the study information | 2019/11/06 |
| Last modified on | 2022/11/07 09:42:49 |
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Basic information
Public title
Exploratory Study of NS-089/NCNP-02 in Duchenne muscular dystrophy
Acronym
Exploratory Study of NS-089/NCNP-02
Scientific Title
Exploratory Study of NS-089/NCNP-02 in Duchenne muscular dystrophy
Scientific Title:Acronym
Exploratory Study of NS-089/NCNP-02
Region
| Japan |
Condition
Condition
Duchenne muscular dystrophy
Classification by specialty
| Neurology | Pediatrics |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
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.
Basic objectives2
Safety
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Explanatory
Developmental phase
Phase I,II
Assessment
Primary outcomes
Safety (adverse event and adverse drug reaction)
Key secondary outcomes
1. Expression of dystrophin protein
2. Detection of exon 44-skipped mRNA of dystrophin
3. Time to Stand Test (TTSTAND)
4. Time to Run/Walk 10 Meters test (TTRW)
5. Six-Minute Walk Test (6MWT) and Two-Minute Walk Test (2MWT)
6. Timed Up & Go (TUG) test
7. Performance of Upper Limb (PUL) test
8. NS-089/NCNP-02 concentration of the blood plasma
9. NS-089/NCNP-02 concentration of the urine
10. Serum Creatine kinase concentration
Base
Study type
Interventional
Study design
Basic design
Single arm
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
"[Part 1]
NS-089/NCNP-02 is administered at dose levels 1 and 3 in Cohort 1 (n=3) and at dose levels 2 and 4 in Cohort 2 (n=3)."
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."
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10
Eligibility
Age-lower limit
| 4 | years-old | <= |
Age-upper limit
| 17 | years-old | >= |
Gender
Male
Key inclusion criteria
Subject with Duchenne muscular dystrophy eligible for enrolment in the study must meet all of the following criteria:
1. Has an out of frame deletion(s) that could be corrected by skipping exon 44 as confirmed by any of methodology at the time of visit 1. If not confirmed by any of methodology that evaluates the relative copy number of all exons (i.e. MLPA etc), must be confirmed through these techniques by the time of visit 3.
2. DNA sequencing of exon 44 confirms that no DNA polymorphisms occur that could compromise duplex formation between NS-089/NCNP-02 and pre-mRNA.
3. Male and >= 8 years and < 17 years of age at the time of obtaining informed consent and/or assent. Subjects aged >= 4 years and < 8 years can be enrolled according to the circumstances.
4. Able to give informed consent in writing signed by parent(s) or legal guardian who is able to understand all of the study procedure requirements. If applicable, able to give informed assent in writing signed by the subject.
5. Life expectancy of at least 1 year
6. Able to ambulate. Non-ambulant subject can be enrolled according to the circumstances.
7. Have intact muscles, which have adequate quality for biopsy. (No lacks or severe atrophy of biceps brachii or tibialis anterior muscle)
8. QTc <450 msec (based on 12-lead ECGs), or <480 msec for subject with Bundle Branch Block.
9. Glucocorticoid-naive patients, or patients who have used systemic glucocorticoids for at least 6 months prior to enrollment in this study with no dose changes for at least 3 months prior to enrollment.
Key exclusion criteria
Subject with Duchenne muscular dystrophy meeting any of the following criteria must not be enrolled in the study:
1. Has participated in other pharmacological clinical trial that might recover dystrophin protein by the readthrough or the exon-skipping therapy, and/or upregulate the dystrophin-associated proteins such as utrophin.
2. A forced vital capacity (FVC) < 50% of predicted.
3. Continuous use of artificial respirator (except for use of NPPV while sleeping)
4. A left ventricular ejection fraction (EF) < 40% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
5. Surgery within the last 3 months prior to the first anticipated administration of study medication or planned for anytime between visit 1 of Part 1 and the last visit of Part 2.
6. Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at screening.
7. Current diagnosis of any immune deficiency or autoimmune disease.
8. Current diagnosis of any active or uncontrolled infection, cardiomyopathy, or liver or renal disease.
9. Use of any other investigational agents and/or experimental agents within 3 months prior to the first anticipated administration of study medication.
10. History of any severe drug allergy.
Target sample size
6
Research contact person
Name of lead principal investigator
| 1st name | Hirofumi |
| Middle name | |
| Last name | Komaki |
Organization
National Center of Neurology and Psychiatry
Division name
Department of Child Neurology, National Center Hospital
Zip code
187-8551
Address
4-1-1, Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan
TEL
042-341-2711
komakih@ncnp.go.jp
Public contact
Name of contact person
| 1st name | Takami |
| Middle name | |
| Last name | Ishizuka |
Organization
National Center of Neurology and Psychiatry
Division name
Section of Clinical Research Suppor, Clinical Research and Education Promotion Division
Zip code
187-8551
Address
4-1-1, Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan
TEL
042-341-2711
Homepage URL
tmc-crso@ncnp.go.jp
Sponsor or person
Institute
National Center of Neurology and Psychiatry
Institute
Department
Personal name
Funding Source
Organization
Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization
Other
Nationality of Funding Organization
Other related organizations
Co-sponsor
Nippon Shinyaku Co., Ltd.
Name of secondary funder(s)
IRB Contact (For public release)
Organization
IRB, National Center of Neurology and Psychiatry
Address
4-1-1, Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan
Tel
042-341-2711
irb-office@ncnp.go.jp
Secondary IDs
Secondary IDs
YES
Study ID_1
NCT04129294
Org. issuing International ID_1
ClinicalTrials.gov
Study ID_2
Org. issuing International ID_2
IND to MHLW
2019年10月7日
Institutions
Institutions
国立研究開発法人国立精神・神経医療研究センター（東京都）/National Center of Neurology and Psychiatry（Tokyo）、鹿児島大学病院（鹿児島県）/Kagoshima University Hospital（Kagoshima）
Other administrative information
Date of disclosure of the study information
| 2019 | Year | 11 | Month | 06 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
6
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Completed
Date of protocol fixation
| 2019 | Year | 10 | Month | 04 | Day |
Date of IRB
| 2019 | Year | 10 | Month | 03 | Day |
Anticipated trial start date
| 2019 | Year | 11 | Month | 06 | Day |
Last follow-up date
| 2021 | Year | 12 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2019 | Year | 11 | Month | 06 | Day |
Last modified on
| 2022 | Year | 11 | Month | 07 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000043651
