Exploratory Study of NS-089/NCNP-02 in DMD

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-12-02

Study Completion Date

2022-05-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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NS-089/NCNP-02

Group Type EXPERIMENTAL

NS-089/NCNP-02

Intervention Type DRUG

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."

Interventions

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NS-089/NCNP-02

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."

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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.
* DNA sequencing of exon 44 confirms that no DNA polymorphisms occur that could compromise duplex formation between NS-089/NCNP-02 and pre-mRNA.
* 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.
* 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.
* Life expectancy of at least 1 year
* Able to ambulate. Non-ambulant subject can be enrolled according to the circumstances.
* Have intact muscles, which have adequate quality for biopsy. (No lacks or severe atrophy of biceps brachii or tibialis anterior muscle)
* QTc \<450 msec (based on 12-lead ECGs), or \<480 msec for subject with Bundle Branch Block.
* 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.

Exclusion Criteria

* 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.
* A forced vital capacity (FVC) \< 50% of predicted.
* Continuous use of artificial respirator (except for use of NPPV while sleeping)
* A left ventricular ejection fraction (EF) \< 40% or fractional shortening (FS) \< 25% based on echocardiogram (ECHO).
* 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.
* Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at screening.
* Current diagnosis of any immune deficiency or autoimmune disease.
* Current diagnosis of any active or uncontrolled infection, cardiomyopathy, or liver or renal disease.
* Use of any other investigational agents and/or experimental agents within 3 months prior to the first anticipated administration of study medication.
* History of any severe drug allergy.

Minimum Eligible Age

4 Years

Maximum Eligible Age

17 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No