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Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2017 by NS Pharma, Inc.
Nippon Shinyaku Co Ltd
Cooperative International Neuromuscular Research Group (CINRG)
Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)
Information provided by (Responsible Party):
NS Pharma, Inc. Identifier:
First received: March 23, 2016
Last updated: April 24, 2017
Last verified: April 2017
The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53 skipping. Additional objectives include tolerability, muscle function and strength, pharmacokinetics and pharmacodynamics.

Condition Intervention Phase
Duchenne Muscular Dystrophy
Drug: NS-065/NCNP-01
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Care Provider, Investigator, Outcomes Assessor
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by NS Pharma, Inc.:

Primary Outcome Measures:
  • Incidence of Adverse Events as assessed by CTCAE v4.0. [ Time Frame: 24 weeks of treatment phase ]
  • Dystrophin protein in muscle as measured by mass spectrometry. [ Time Frame: 20-24 weeks of treatment ]
  • Drug concentration in plasma. [ Time Frame: 20-24 weeks of treatment ]

Secondary Outcome Measures:
  • Induction of dystrophin messenger ribonucleic acid (mRNA) in muscle as measured by real time polymerase chain reaction (RT-PCR) for mRNA analysis. [ Time Frame: 20-24 weeks of treatment ]
  • Induction of dystrophin protein in muscle as measured by western blot and immunofluorescence methods for protein analysis. [ Time Frame: 20-24 weeks of treatment ]
  • Muscle strength as measured by Quantitative Muscle Testing (QMT). [ Time Frame: 20-24 weeks of treatment ]
  • Change in distance traveled in the Six-Minute Walk Test (6MWT). [ Time Frame: baseline to 20-24 weeks of treatment ]
  • Change in Time to Climb 4 Stairs (TTCLIMB). [ Time Frame: baseline to 20-24 weeks of treatment ]
  • Change in Time to Run/Walk 10 meters (TTRW). [ Time Frame: baseline to 20-24 weeks of treatment ]
  • Change in Time to Stand (TTSTAND) [ Time Frame: baseline to 20-24 weeks of treatment ]
  • North Star Ambulatory Assessment (NSAA) results. [ Time Frame: baseline to 20-24 weeks of treatment ]

Estimated Enrollment: 16
Study Start Date: December 2016
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NS-065/NCNP-01 40mg/kg
Six patients with confirmed DMD with genetic deletions amenable to exon 53 skipping will be administered an intravenous infusion of NS-065/NCNP-01 40mg/kg dose once a week for 24 weeks
Drug: NS-065/NCNP-01
Experimental: NS-065/NCNP-01 80mg/kg
Six patients with confirmed DMD with genetic deletions amenable to exon 53 skipping will be administered an intravenous infusion of NS-065/NCNP-01 80mg/kg once a week for 24 weeks
Drug: NS-065/NCNP-01
Placebo Comparator: Placebo
Two patients in each of the dose groups will be administered placebo as an intravenous infusion once a week for 4 weeks followed by 20 weeks of open label treatment
Drug: Placebo

Detailed Description:

This is a Phase II, multiple center, 2-period, randomized, placebo-controlled, dose finding study of NS-065/NCNP-01 administered by infusion once weekly for 24 weeks to ambulant boys ages 4-<10 years with DMD. Two dose level cohorts will be enrolled. Period 1 of this study will be conducted in a double-blind fashion. Randomized patients will receive weekly IV infusions of NS-065/NCNP-01 or placebo for the first 4 weeks of their participation (Period 1) and NS-065/NCNP-01 by IV infusion for weeks 5-24 (20 weeks of active treatment - Period 2). Analysis of safety data from Period 1 of the 40mg/kg dose cohort will be completed prior to enrolling patients in the 80mg/kg dose cohort.

Patients completing the 24-week study will be eligible for an open-label extension study.

Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six-minute walk test (6MWT), time to stand (TTSTAND), time to run/walk 10 meters (TTRW), time to climb 4 stairs (TTCLIMB) and quantitative muscle testing (QMT). All patients will undergo a muscle biopsy of the bicep at baseline and a second muscle biopsy at Week 24.

Safety will be assessed through the collection of adverse events (AEs), blood and urine laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study.

Serial blood samples will be taken at four of the study visits to assess the pharmacokinetics of the study drug.


Ages Eligible for Study:   4 Years to 9 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male ≥ 4 years and <10 years of age
  • Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame;
  • Able to walk independently without assistive devices;
  • Ability to complete the time to stand, time to run/walk and time to climb assessments;
  • Stable dose of glucocorticoid for at least 3 months

Exclusion Criteria:

  • Acute illness within 4 weeks prior to the first dose of study medication;
  • Evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
  • Severe allergy or hypersensitivity to medications;
  • Severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
  • Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator;
  • Patient is taking any other investigational drug currently or within 3 months prior to the start of study treatment; or
  • Patient has had surgery within the 3 months prior to the first anticipated administration of study medication or surgery is planned for anytime during the duration of the study;
  • Patient has previously participated in this study or any other study during which NS-065/NCNP-01 was administered.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02740972

Contact: Lauren Morgenroth 412-224-2030
Contact: Sara Misplay

United States, California
UC Davis Medical Center Recruiting
Sacramento, California, United States
Contact: Erica Goude    916-734-0384   
Principal Investigator: Craig McDonald, MD         
United States, Florida
University of Florida Health Recruiting
Gainesville, Florida, United States
Contact: Gee Kim    352-273-7573   
Principal Investigator: Barry Byrne, MD         
United States, Illinois
Lurie Children's Hospital Recruiting
Chicago, Illinois, United States
Contact: Theresa Oswald    312-227-3019   
Principal Investigator: Vamshi Rao, MD         
United States, Missouri
Washington University Recruiting
Saint Louis, Missouri, United States
Contact: Pallavi Anand    314-362-2490   
Principal Investigator: Anne M Connolly, MD         
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States
Contact: Karen Cornett    919-684-1143   
Principal Investigator: Edward C Smith, MD         
United States, Virginia
Children's Hospital of Richmond at VCU Recruiting
Richmond, Virginia, United States
Contact: Kathy O'Hara    804-828-3862   
Principal Investigator: Amy Harper, MD         
Canada, Alberta
Alberta Children's Hospital Not yet recruiting
Calgary, Alberta, Canada
Contact: Tiffany Haig    403-955-3192   
Principal Investigator: Jean Mah, MD         
Sponsors and Collaborators
NS Pharma, Inc.
Nippon Shinyaku Co Ltd
Cooperative International Neuromuscular Research Group (CINRG)
Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)
Study Chair: Paula R. Clemens, MD University of Pittsburgh
  More Information

Responsible Party: NS Pharma, Inc. Identifier: NCT02740972     History of Changes
Other Study ID Numbers: NS-065/NCNP-01-201
Study First Received: March 23, 2016
Last Updated: April 24, 2017

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked processed this record on April 28, 2017