Clinical trial NCT03167255: Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Official title: A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy
Lead sponsor: NS Pharma, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Nippon Shinyaku Co., Ltd.
name: Cooperative International Neuromuscular Research Group
name: Therapeutic Research in Neuromuscular Disorders Solutions
First posted: 2017-05-25
Enrollment: 16
Sex: MALE
Minimum age (years): 4
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Low Dose cohort of 40 mg/kg and High Dose cohort of 80 mg/kg
Primary purpose: TREATMENT

Brief summary: This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.

Detailed description: This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201. This study will evaluate the safety, tolerability, and clinical efficacy of NS-065/NCNP-01 at dose levels of up to 80 mg/kg/week administered by weekly IV infusion over an additional treatment period of 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.

Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.

Eligibility criteria: Inclusion Criteria:

1. Completed Study NS-065/NCNP-01-201 through Week 25.
2. Willing and able to comply with scheduled visits, investigational product administration plan, and study procedures.
3. Stable dose of glucocorticoid (GC), and is expected to remain on the stable dose for the duration of the study.

Exclusion Criteria:

1. Serious or severe adverse event in Study NS-065/NCNP-01-201 that precludes safe use of NS-065/NCNP-01.
2. Patient had a treatment which was made for the purpose of dystrophin or its related protein induction after completion of Study NS-065/NCNP-01-201.
3. Patient took any other investigational drugs after completion of Study NS-065/NCNP-01-201.
4. Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.

Primary outcomes:
measure: Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
measure: Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
measure: Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.
timeFrame: Up to 192 weeks of treatment
Secondary outcomes:
measure: Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
measure: Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
measure: Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
measure: Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
measure: Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202
measure: Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202
measure: Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
measure: Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
measure: Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
measure: Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
measure: Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls
timeFrame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: UC Davis
state: California
city: Chicago
country: United States
facility: Lurie Children's Hospital
state: Illinois
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Richmond
country: United States
facility: Children's Hospital of Richmond at VCU
state: Virginia
city: Calgary
country: Canada
facility: Alberta Children's Hospital
state: Alberta
Study officials:
affiliation: University of Pittsburgh
name: Paula R. Clemens, MD
role: STUDY_CHAIR
References: