Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 12:21 AM
Ignite Modification Date:
2025-12-25 @ 12:21 AM
NCT ID:
NCT03558958
Eligibility 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
Healthy Volunteers:
False
Sex:
MALE
Minimum Age:
12 Years
Maximum Age:
25 Years
Study:
NCT03558958
Study Brief:
Protocol Section:
NCT03558958