Evaluation of Efficacy, Tolerability, and Pharmacokinetics of MYMD1 for Chronic Inflammation and Sarcopenia/Frailty

Recruiting at 2 trial locations

Overseen By Director of Regulatory Affairs

Age: 65+

Sex: Any

Trial Phase: Phase 2

Sponsor: TNF Pharmaceuticals, Inc.

Must not be taking: Anti-inflammatories, Anxiolytics, Cannabis, Opioids

Disqualifiers: Dementia, Diabetes, Cancer, COPD, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 30

Duration: 4 weeks

40 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests MYMD1, a drug aimed at reducing inflammation, in older adults with muscle weakness and frailty. The drug works by lowering the chemicals that cause inflammation, potentially improving muscle strength.

Will I have to stop taking my current medications?

The trial requires that you stop taking anti-inflammatory drugs on a daily basis. If you are on a stable antidepressant regimen for at least 3 months and agree not to increase the dose during the trial, you may continue taking it. Other medications, especially those with narrow therapeutic ranges, systemic steroids, and certain other drugs, may also need to be stopped or adjusted.

How does the drug MYMD-1 differ from other treatments for Duchenne muscular dystrophy?

The drug MYMD-1 is unique because it is part of a novel trial design that uses a small-sample, sequential, multiple assignment approach, which increases the efficiency of treatment effect estimates by enriching the placebo arm with external control data and using data from all stages. This method aims to improve the accuracy of drug development in rare diseases like Duchenne muscular dystrophy. ^{1 2 3 4 5}

Research Team

Leonard Dunn, MD

Principal Investigator

Clinical Research of West Florida

Lon Lynn, DO

Principal Investigator

Clinical Research of West Florida

Jeremy Walston, MD

Principal Investigator

Johns Hopkins University

Eligibility Criteria

Inclusion Criteria

Weight

You are 65 years or older, at the time of signing the ICF.

Elevated biomarkers of inflammation (serum IL-6 level ≥2.

Exclusion Criteria

Known history or presence of severe active acute or chronic liver disease (eg, cirrhosis)

Medical conditions that would impact mobility testing or handgrip strength including Rheumatoid arthritis, any autoimmune condition, Parkinson's disease, muscular dystrophy, cerebral vascular accident, lower or upper extremity neuropathy, major skeletal joint deformity, upper extremity joint dysfunction, partial or complete upper extremity amputation or missing anatomy impacting grip, history of pain with walking, gout, chronic obstructive pulmonary disease, congestive heart failure, exercise induced angina, lower extremity amputation (partial or complete) or missing anatomy impacting walking, recent surgery or hospitalization (past 3 months); lower or upper extremity fracture in the past 6 months, lower or upper extremity tendinitis, diagnosis of cancer other than basal cell carcinoma, dialysis dependent renal disease, Meniere's disease, spinal cord fracture or compression, paraplegia or quadriplegia or any other medical condition that in the opinion of the Investigator would impair measurement of a 6-minute walk or handgrip strength

Requires regular assistance from another person for general activities of daily living (eg, bathing, dressing, toileting)

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Treatment

Participants receive MYMD1 or placebo in a blinded fashion until the end-of-study visit on Day 28

4 weeks

Multiple visits for PK sampling and safety assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Weekly phone calls for 30-day follow-up

Treatment Details

Interventions

• MYMD-1
• Placebo

Participant Groups

Group I: Cohort 3: MYMD1 900mg Experimental Treatment 1 Intervention

Subjects randomly assigned to the MYMD1 900mg cohort

Group II: Cohort 2: MYMD1 750mg Experimental Treatment 1 Intervention

Subjects randomly assigned to the MYMD1 750 cohort

Group III: Cohort 1: MYMD1 600mg Experimental Treatment 1 Intervention

Subjects randomly assigned to the MYMD1 600mg cohort

Group IV: Cohort 4: MYMD1 1050mg Active Control 1 Intervention

Subjects randomly assigned to the MYMD1 1050mg cohort

Group V: Cohort 3: Placebo 900mg Placebo Group 1 Intervention

Subjects assigned to the 900mg placebo group

Group VI: Cohort 4: Placebo group 1050mg Placebo Group 1 Intervention

Subjects assigned to the 1050mg placebo group

Group VII: Cohort 2: Placebo 750mg Placebo Group 1 Intervention

Subjects assigned to the 750mg placebo group

Group VIII: Cohort 1: Placebo 600mg Placebo Group 1 Intervention

Subjects assigned to the 600mg placebo group

Who Is Running the Clinical Trial?

TNF Pharmaceuticals, Inc.

Lead Sponsor

MyMD Pharmaceuticals, Inc.

Lead Sponsor

Trials

3

Recruited

110+

Findings from Research

In a Phase 2 study involving 51 ambulant Duchenne muscular dystrophy (DMD) patients, drisapersen at a dose of 6 mg/kg/week showed a potential benefit in improving the 6-minute walking distance (6MWD) compared to placebo, with a mean difference of 27.1 meters at week 24.

Drisapersen was generally well-tolerated, with the most common side effects being injection site reactions and subclinical proteinuria, indicating a manageable safety profile for this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy. McDonald, CM., Wong, B., Flanigan, KM., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Dynamic enrichment of Bayesian small-sample, sequential, multiple assignment randomized trial design using natural history data: a case study from Duchenne muscular dystrophy. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Molecular treatments in Duchenne muscular dystrophy. [2021]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Update on Standard Operating Procedures in Preclinical Research for DMD and SMA Report of TREAT-NMD Alliance Workshop, Schiphol Airport, 26 April 2015, The Netherlands. [2021]