Ignite Creation Date:
2025-12-25 @ 12:21 AM
Ignite Modification Date:
2025-12-25 @ 10:24 PM
Study NCT ID:
NCT03558958
Status:
TERMINATED
Last Update Posted:
2023-01-11
First Post:
2018-06-05
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Safety and Efficacy of P-188 NF in DMD Patients
Sponsor:
Phrixus Pharmaceuticals, Inc.
Organization:
Study Overview
Official Title:
An Exploratory, Open-label Study to Assess the Effect of P-188 NF (Carmeseal-MD) on Safety, on Respiratory and Cardiac Dysfunction and on Upper Limb Strength in Non-ambulatory Patients With Duchenne Muscular Dystrophy (DMD)
Status:
TERMINATED
Status Verified Date:
2021-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Terminated due to lack of funding.
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Detailed Description:
Based on a large number of studies conducted in pre-clinical models of muscular dystrophy and heart failure, this study is being undertaken to explore the safety and efficacy of Carmeseal-MD™ (P-188 NF) on endpoints associated with cardiovascular, pulmonary and musculoskeletal function. These preclinical studies indicate that Carmeseal-MD™ acts to stabilize fragile cell membranes thus maintaining cell function and preventing fibrosis, necrosis and apoptosis in animal models of muscular dystrophy.
This is a single arm, open label trial that is designed to provide a first evaluation of Carmeseal-MD™ in non-ambulatory patients with DMD. It assigns up to ten (10) patients to receive a fixed dose of 5 mg of P-188 NF per Kg patient body weight (adjusted individually for each patient at baseline visit) injected subcutaneously once-a-day for 52 weeks. The first 3 enrolled subjects (Group 1) will be at least 18 years of age and up to 25 years of age. Enrollment of Group 2 will begin after a review of Group 1 safety data through 28 days of dosing of Carmeseal-MD™. Group 2 will include subjects that are at least 12 years of age and up to 25 years old. Evaluations will be for Carmeseal-MD™ administered in addition to the current standard of care therapies and interventions such as corticosteroids, ACE inhibitors, ARBs, beta blockers, bronchodilator medications and airway clearance, cough assist and non-invasive ventilation devices.
The major hypothesis for the trial is that measures of function of skeletal and cardiac muscle that decline over the course of the disease will either remain stable or improve with P-188 NF treatment when a decline would be expected. To assess these possible beneficial effects, comparisons are planned between pre- and post-treatment on measures of function for the various body systems affected by DMD.
This is a single arm, open label trial that is designed to provide a first evaluation of Carmeseal-MD™ in non-ambulatory patients with DMD. It assigns up to ten (10) patients to receive a fixed dose of 5 mg of P-188 NF per Kg patient body weight (adjusted individually for each patient at baseline visit) injected subcutaneously once-a-day for 52 weeks. The first 3 enrolled subjects (Group 1) will be at least 18 years of age and up to 25 years of age. Enrollment of Group 2 will begin after a review of Group 1 safety data through 28 days of dosing of Carmeseal-MD™. Group 2 will include subjects that are at least 12 years of age and up to 25 years old. Evaluations will be for Carmeseal-MD™ administered in addition to the current standard of care therapies and interventions such as corticosteroids, ACE inhibitors, ARBs, beta blockers, bronchodilator medications and airway clearance, cough assist and non-invasive ventilation devices.
The major hypothesis for the trial is that measures of function of skeletal and cardiac muscle that decline over the course of the disease will either remain stable or improve with P-188 NF treatment when a decline would be expected. To assess these possible beneficial effects, comparisons are planned between pre- and post-treatment on measures of function for the various body systems affected by DMD.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: