Safety and Efficacy of P-188 NF in DMD Patients

This is an interventional treatment trial for Duchenne Muscular Dystrophy Read More

Inclusion Criteria:

• Male
• 12 - 25 years of age
• Have phenotypic evidence of DMD
• Have documentation of the presence of a deletion, duplication or point mutation in the dystrophin gene
• Willingness to receive daily subcutaneous (SC) injections of up to 3 mL
• Have LVEDV that is ≥100% of normal corrected for body mass when measured by cardiac MRI
• Have impaired respiratory function (percent predicted PEF ≤80%)
• Have ability to perform PEF within 15% of first assessment
• Have mild to moderate fibrosis of the heart as assessed by MRI
• Have left ventricular ejection fraction fractions of <50%
• Have been non-ambulatory for at least six months
• Be on corticosteroids, with a stable treatment regimen for at least six months
• Have been on a stable treatment regimen for cardiac dysfunction for at least 3 months prior to baseline (ACE inhibitors, beta blockers and/or ARBs)
• Have clinically acceptable screening values, including serum creatinine levels blood urine nitrogen, cystatin C
• Have willingness and ability to comply with scheduled visits, drug administration, drug administrative plan, study procedures, laboratory tests, and treatment restrictions
• Be likely to survive for the duration of the treatment in the investigator's opinion
• Have ability to provide written informed consent (parent/guardian consent if applicable)/assent (if <18 years of age).

Exclusion Criteria:

• Exposure to another investigational drug within 90 days prior to start of study treatment
• Have DMD-related hypoventilation for which daytime assisted ventilation is needed
• Unable to perform pulmonary function testing
• Have respiratory failure
• Unable or unwilling to undergo scan with gadolinium as contrast agent
• Unable or unwilling to undergo echocardiography
• Have severe fibrosis of the heart as assessed by MRI
• Used carnitine, creatine, glutamine, oxatomide, coenzyme Q10 or vitamin E or any herbal medicines with 30 days prior to baseline
• Have a history of major surgical procedure within 30 days prior to start of study treatment
• Have ongoing immunosuppressive therapy (other than corticosteroids)
• Are participating in a therapeutic clinical trial
• Are on any concomitant medication with a depressive or stimulating effect on respiration or the respiratory tract
• Have a diagnosis of chronic lung disease
• Chronic use of beta-2 agonists or any other bronchodilating medication (chronic use is daily intake for more than 14 days within the last 6 months)
• Have moderate or severe hepatic impairment or moderate to severe renal impairment
• Have expectation of major surgical procedure during the conduct of the study
• Have prior or ongoing medical conditions that makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of the treatment results
• Have ever previously received P-188 NF as a therapeutic agent