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Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)

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ClinicalTrials.gov Identifier: NCT04956289
Recruitment Status : Recruiting
First Posted : July 9, 2021
Last Update Posted : April 1, 2022
Sponsor:
Collaborator:
Nippon Shinyaku Co., Ltd.
Information provided by (Responsible Party):
NS Pharma, Inc.

Brief Summary:
This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Viltolarsen Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls
Actual Study Start Date : July 1, 2021
Estimated Primary Completion Date : May 2024
Estimated Study Completion Date : May 2024


Arm Intervention/treatment
Experimental: Viltolarsen
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Drug: Viltolarsen
Received during weekly intravenous infusions
Other Name: NS-065/NCNP-01




Primary Outcome Measures :
  1. Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03 [ Time Frame: baseline to up to 48 weeks of treatment ]

Secondary Outcome Measures :
  1. Peak Expiratory Flow (PEF) [ Time Frame: baseline to 48 weeks of treatment ]
  2. Forced Vital Capacity (FVC) [ Time Frame: baseline to 48 weeks of treatment ]
  3. Forced expiratory volume in 1 second (FEV1) [ Time Frame: baseline to 48 weeks of treatment ]
  4. Performance of Upper Limb (PUL) [ Time Frame: baseline to 48 weeks of treatment ]
    The PUL provides both a total score and sub-scores for the 3 domains (shoulder, middle, and distal) that in DMD are progressively involved with a proximal to distal gradient. The PUL includes 22 items with an entry item to define the starting functional level, and 21 items subdivided into shoulder level (4 items), middle level (9 items), and distal level (8 items) dimensions. Scoring options vary across the scale between 0-1 to 0-6 according to performance. Each dimension can be scored separately with a maximum score of 16 for the shoulder level, 34 for the middle level, and 24 for the distal level. A total score can be achieved by adding the 3 level scores (maximum total score 74).

  5. Brooke scale [ Time Frame: baseline to 48 weeks of treatment ]
    The Brooke scale for upper limb has grades ranging from 1 to 6. Grade 1 is given to the patient who can keep both his arms by his sides in the starting position and is then able to abduct the arms fully so that both the arms are touching over the head. Grade 6 is given when the patient is unable to raise his hands to his mouth, and the hands show no functional usefulness.

  6. Vignos scale [ Time Frame: baseline to 48 weeks of treatment ]
    The Vignos scale for lower limb has grades ranging from 1 to 10; 1 means that the patient is able to walk and climb stairs without assistance, while 10 means that the patient is bed bound. Ambulant patients score 1 to 6 and non-ambulant patients score 7 to 10 on the Vignos scale.

  7. Muscle Strength Measured by Hand-Held Dynamometer [ Time Frame: baseline to 48 weeks of treatment ]
  8. North Star Ambulatory Assessment (NSAA) [ Time Frame: baseline to 48 weeks of treatment ]
    The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.



Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patient (if age 18 years or older) or patient's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act authorization, where applicable, prior to any study-related procedures; patients younger than age 18 years will be asked to give written or verbal assent according to local requirements;
  2. Patient has a confirmed diagnosis of DMD defined as:

    1. Patient is male with clinical signs compatible with DMD; and
    2. Patient has a confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin messenger ribonucleic acid reading frame including determination of unambiguously defined exon boundaries (using techniques such as multiplex ligation-dependent probe amplification, comparative genomic hybridization array, or other techniques with similar capability);
  3. Patient is more than 8 years of age at time of first infusion in the study;
  4. Patient has a Brooke scale rating of 3 or better OR an upright FVC 30% or greater at Screening;
  5. Patient, if sexually active, is willing to abstain from sexual intercourse or employ a barrier or medical method of contraception during and for 3 months following completion of IP administration;
  6. Patient and patient's parent(s)/guardian(s) (if patient is <18 years of age) and/or caregiver(s) are willing and able to comply with scheduled visits, IP administration plan, and study procedures;
  7. Patient must be on a stable dose of glucocorticoid (GC) or not treated with GC for at least 3 months prior to the first dose of IP and is expected to remain on stable dose of GC treatment or off GC for the duration of the study.

Other inclusion criteria may apply

Exclusion Criteria:

  1. Patient has had an acute illness within 4 weeks prior to the first dose of IP;
  2. Patient has evidence of symptomatic cardiomyopathy (New York Heart Association Class III or higher);
  3. Patient requires ventilation support while awake during the day;
  4. Patient has an allergy or hypersensitivity to IP or any of its constituents;
  5. Patient has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator;
  6. Patient has a previous or ongoing medical condition, medical history, physical findings, or laboratory abnormalities that could affect patient 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;
  7. Patient has had surgery within 3 months prior to the first anticipated administration of IP or has known plans to have surgery during the Treatment Period;
  8. Patient has positive test results for hepatitis B antigen, hepatitis C antibody, or human immunodeficiency virus antibody at Screening;
  9. Patient has been diagnosed with asthma that requires chronic treatment with a long-acting beta agonist;
  10. Patient has relevant history of or current drug or alcohol abuse or use of any tobacco/marijuana products by smoking or vaping within 3 months prior to treatment with IP;
  11. Patient is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of IP or within 5 times the half-life of a medication, whichever is longer;
  12. Patient has taken any gene therapy;
  13. Patient is currently taking any other exon skipping agent or has taken any other exon skipping agent within 3 months prior to the first dose of IP;
  14. Patient has hydronephrosis, hydroureter, renal or urinary tract calculi, or ureteral stenosis by renal ultrasound;
  15. Patient was previously enrolled in an interventional study of viltolarsen.

Other exclusion criteria may apply


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04956289


Contacts
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Contact: Trial Info 1-866-677-4276 trialinfo@nspharma.com

Locations
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United States, Oregon
Oregon Health & Science University Withdrawn
Portland, Oregon, United States, 97239
United States, Virginia
Children's Hospital of Richmond at VCU Recruiting
Richmond, Virginia, United States, 23219
China
The Third Medical Center of PLA General Hospital Recruiting
Beijing, China, 1000053
Hunan Children's Hospital Recruiting
Changsha, China, 410021
Italy
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore Recruiting
Roma, Italy, 00168
Russian Federation
Russian National Research Medical University Active, not recruiting
Moscow, Russian Federation, 117997
Saint Petersburg State Paediatric Medical University Active, not recruiting
Saint Petersburg, Russian Federation, 194100
Spain
Hospital Sant Joan de Deu Recruiting
Barcelona, Spain, 08950
Turkey
Yeditepe University Kosuyolu Hospital Recruiting
Istanbul, Turkey, 34718
Sponsors and Collaborators
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.
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Responsible Party: NS Pharma, Inc.
ClinicalTrials.gov Identifier: NCT04956289    
Other Study ID Numbers: NS-065/NCNP-01-211
First Posted: July 9, 2021    Key Record Dates
Last Update Posted: April 1, 2022
Last Verified: July 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked