Safety and Efficacy of P-188 NF in DMD Patients

• Status: Suspended
• Trial end date: 2020-11-01
• Target enrollment: 0
• Participant gender: Male

Summary

This is an open-label study to evaluate the safety, tolerability and efficacy of daily, subcutaneous dosing with P-188 NF (Carmeseal-MD™) in non-ambulatory boys with Duchenne Muscular Dystrophy (DMD). This study will determine if continuous treatment with Carmeseal-MD™ can maintain or improve pulmonary function, and skeletal and cardiac muscle function, compared to baseline, in boys 12-25 years of age.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Phrixus Pharmaceuticals, Inc.
• Collaborator: Charley's Fund

Criteria

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