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A Phase 2 Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

key information

study id #: NCT04004065

condition: Duchenne Muscular Dystrophy

status: recruiting

purpose:

This study will be comprised of 2 parts: Part A (Multiple Ascending Dose (MAD)) which will be conducted to evaluate the safety and tolerability of SRP-5051 at multiple ascending dose levels to determine the maximum tolerated dose (MTD); Part B (Dose Expansion) will be conducted to evaluate SRP-5051 administered at the MTD in patients who have completed Part A.

intervention: SRP-5051

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT04004065

last updated: July 22, 2019

study details

start date: June 26, 2019

estimated completion: March 2021

phase of development: Phase 2

size / enrollment: 24

primary outcomes:

  • Part A: Incidence of Adverse Events (AEs) [ Time Frame: From signing of informed consent until 28 days after last dose of study drug administered (up to 60 weeks) ]
    Incidence of adverse events includes clinically significant laboratory abnormalities.
  • Part B: Change From Baseline Biopsy in Exon-Skipping Levels [ Time Frame: Baseline, Part B Week 24 ]
  • Part B: Change From Baseline Biopsy in Dystrophin Protein Production Levels [ Time Frame: Baseline, Part B Week 24 ]

secondary outcomes:

  • Part A: Pharmacokinetic (PK) Plasma Concentration of SRP-5051 [ Time Frame: Predose and at multiple timepoints (up to 24 hours) after end of infusion ]
  • Part B: Incidence of Adverse Events (AEs) [ Time Frame: Up to 28 weeks in Part B ]
    Incidence of adverse events includes clinically significant laboratory abnormalities.
  • Part B: Change From Baseline in Forced Vital Capacity (FVC) (percent predicted) [ Time Frame: Baseline, Part B Week 24 ]
  • Part B: Change From Baseline in the North Star Ambulatory Assessment (NSAA) [ Time Frame: Baseline, Part B Week 24 ]
  • Part B: Change From Baseline in the Performance of Upper Limb (PUL) Scores [ Time Frame: Baseline, Part B Week 24 ]
  • Part B: Change From Baseline in the Brooke Upper Extremity Scale score (Brooke score) [ Time Frame: Baseline, Part B Week 24 ]
  • Part B: PK Plasma Concentration of SRP-5051 [ Time Frame: Part B predose and at multiple timepoints (up to 12 hours) after end of infusion ]

inclusion criteria:
• Has a genetic diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.
• Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration, or has not received corticosteroids for at least 12 weeks prior to study drug administration.

exclusion criteria:
• Has a left ventricular ejection fraction (LVEF) less than (<) 40.0 percent (%) based on an echocardiogram (ECHO) performed within 12 weeks prior to Screening or at the Screening visit.
• Has a FVC < 40.0% of predicted value within 12 weeks prior to Screening or at Screening.
• Initiation or change of dosing (except for modifications to accommodate changes in weight) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, β-blockers, or potassium.
• Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations.
• Treatment with any exon 51-skipping therapy within 24 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time.

study contacts

sponsor: Sarepta Therapeutics

contacts: Medical Information, +1 888-727-3782, clinicaltrials@sarepta.com

investigators: Medical Director, Sarepta Therapeutics

locations: United States

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