Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04708314
Recruitment Status : Terminated (The Sponsor has decided to focus their resources on other areas of therapy.)
First Posted : January 13, 2021
Last Update Posted : May 17, 2021
Sponsor:
Collaborator:
Sarepta Therapeutics, Inc.
Information provided by (Responsible Party):
Rare Disease Research, LLC

Tracking Information
First Submitted Date  ICMJE September 21, 2020
First Posted Date  ICMJE January 13, 2021
Last Update Posted Date May 17, 2021
Actual Study Start Date  ICMJE October 31, 2020
Actual Primary Completion Date May 13, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 12, 2021)
Explore the safety and tolerability of Golodirsen in number of participants with Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs [ Time Frame: Baseline up to 96 weeks ]
Adverse Event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment emergent adverse events were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug [up to 148 weeks]) that were absent before treatment or that worsened relative to pre-treatment state. AEs included both serious and non-serious adverse events.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures
 (submitted: January 12, 2021)
  • Change from Baseline to Week 96 in Forced Vital Capacity Percent (FVC%) Predicted [ Time Frame: Baseline up to 96 weeks ]
    FVC is the total amount of air exhaled during the forced expiratory volume test that is measured during spirometry; and is the most important measurement of lung function. This test requires participant to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which is used to dilate participant's bronchial (breathing) tubes. Percent of predicted FVC = (observed value) / (predicted value) * 100%.
  • Change from Baseline to Week 96 in Performance of Upper Limb (PUL) Score [ Time Frame: Baseline up to 96 weeks ]
    The PUL was specifically designed for DMD patients to evaluate the progression of weakness and natural history of functional decline in DMD. Psychometric methods were employed to create a viable scale to enable a clinician-reported outcome assessment tool that can establish clinical meaningfulness and relevance of activities of daily living (ADL).
  • Change from Baseline to Week 96 in Brooke scale for upper extremity [ Time Frame: Baseline up to 96 weeks ]
    The Brooke Score for Arms and Shoulders was developed specifically for use in this population to assess arm and shoulder function over 6 functional grades.
  • Change from Baseline to Week 96 in 9-Hole Peg Test [ Time Frame: Baseline up to 96 weeks ]
    In this timed test, the patient is instructed to take 9 pegs from a shallow bowl in the testing apparatus and put each peg into a hole, 1 at a time. When all 9 pegs are in place, the patient takes them out 1 at a time and puts them back in the shallow bowl. This test is repeated twice with each hand and the best time for each hand is recorded.
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
Official Title  ICMJE An Open-Label Study to Evaluate the Safety of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
Brief Summary

This is an open-label study to evaluate the safety and tolerability of golodirsen injection in Non-ambulant DMD patients with confirmed genetic mutations amenable to treatment by exon 53 skipping (Golodirsen).

Golodirsen 30 mg/kg will be administered as an intravenous (IV) infusion over approximately 35 to 60 minutes once a week during the treatment period (up to 96 weeks). After the treatment period, patients can go into a safety extension period (not to exceed 48 weeks) until the patient is able to transition to commercially available drug or a separate golodirsen study.

Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations. Exploratory assessments, including pulmonary function tests (PFTs), upper extremity testing, and other measurements of functional status, will occur at functional assessment visits every 12 weeks over the first year of treatment and approximately every 24 weeks over the second year of treatment.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Drug: Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]
Vyondys 53
Study Arms  ICMJE 30 mg/kg
Golodirsen 30 mg/kg will be administered as an intravenous (IV) infusion over approximately 35 to 60 minutes once a week during the treatment period (up to 96 weeks). After the treatment period, patients can go into a safety extension period (not to exceed 48 weeks) until the patient is able to transition to commercially available drug or a separate golodirsen study.
Intervention: Drug: Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: March 1, 2021)
2
Original Estimated Enrollment  ICMJE
 (submitted: January 12, 2021)
12
Actual Study Completion Date  ICMJE May 13, 2021
Actual Primary Completion Date May 13, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Be a male with DMD with a mutation that may be amenable to exon 53 skipping as documented by a genetic report from an accredited laboratory confirming mutation endpoints by multiplex ligation-dependent probe amplification.
  2. Be 7 years of age or older.
  3. Has been on a stable dose of oral corticosteroids for at least 24 weeks prior to study drug administration and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the study or has not received corticosteroids for at least 24 weeks prior to study drug administration and does not expect to start corticosteroids throughout the study.
  4. Be unable to ambulate ("non-ambulatory"). By definition, loss of ambulation means patient or caregiver reported continuous wheelchair use that has been verified by a clinical evaluator. The following conditions should be met:

    1. Condition is not secondary to acute worsening of mobility due to orthopedic morbidity (eg, fracture, sprain, or injury) or surgical procedure.
    2. Unable to perform 10-meter walk run test.
  5. Has stable pulmonary function that, in the opinion of the Investigator, is unlikely to decompensate over the study period.
  6. Patients who are post-pubertal and sexually active must agree to use, for the entire duration of the study and for 90 days post last dose, a male condom and the female sexual partner must also use a medically acceptable form of birth control (eg, oral contraceptives).
  7. Able to understand and comply with all study requirements, in the Investigator's opinion, or if under the age of 18 years, must have a parent(s) or legal guardian(s) who is able to understand.
  8. Willing to provide informed consent to participate in the study, or if under the age of 18 years, be willing to provide informed assent, if applicable, and have a parent(s) or legal guardian(s) who is willing to provide informed consent for the patient to participate in the study.

Exclusion Criteria:

  1. Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks of study drug administration that in the opinion of the Investigator might have an effect on skeletal muscle strength or function (eg, growth hormone, anabolic steroids).
  2. Previous treatment with any investigational drug or exon skipping therapy within the last 3 months.
  3. Major change in physiotherapy regimen within the past 3 months or expected change over the study period.
  4. Major surgery within 3 months of study drug administration or planned major surgery for any time during this study.
  5. Presence of other clinically significant illness that cannot be attributed to classic Duchenne disease course including significant cardiac, pulmonary, hepatic, renal, hematologic, immunologic, behavioral disease, or malignancy.
  6. Systemic use of any aminoglycoside antibiotic within 12 weeks of study drug administration or anticipated need for use of an aminoglycoside antibiotic or statin during the study.
  7. Must not require antiarrhythmic and/or antidiuretic therapy for heart failure. Patients are allowed to take other medication including angiotensin-converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents, β blockers or potassium, provided they have been on a stable dose for 24 weeks prior to study drug administration and the dose is expected to remain constant throughout the study.
  8. If the patient is asymptomatic but has a LVEF < 40% at Screening or clinically significant at the discretion of Investigator, the Investigator should discuss inclusion of patient in the study with the appropriate institutional safety committee or medical monitor.
  9. Prior or ongoing medical condition that could, in the Investigator's opinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Gender Eligibility Description: Only male patients are affected by this condition
Ages  ICMJE 7 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04708314
Other Study ID Numbers  ICMJE 19-06-001
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Rare Disease Research, LLC
Study Sponsor  ICMJE Rare Disease Research, LLC
Collaborators  ICMJE Sarepta Therapeutics, Inc.
Investigators  ICMJE Not Provided
PRS Account Rare Disease Research, LLC
Verification Date May 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP