MRG-001 in Patients With Amyotrophic Lateral Sclerosis

Phase 2

Not yet recruiting

• Conditions: Amyotrophic Lateral Sclerosis; Lou Gehrig Disease; Motor Neuron Disease; Motor Neuron Atrophy

• Registration Number: NCT06315608
• Lead Sponsor: MedRegen LLC

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-3-12
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: Not yet recruiting
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Inclusion Criteria:<br><br> - Able to provide written informed consent (either from patient or patient's legally<br> acceptable representative and complying with study procedures, in the PI's opinion.<br><br> - Male or female patients between 18-75 years.<br><br> - Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported<br> probable, or definite ALS defined by revised El Escorial criteria.<br><br> - Time since onset of weakness due to ALS = 48 months at the time of the Screening<br> Visit<br><br> - Vital Capacity = 50% of predicted capacity for age, height, and sex at the time of<br> the Screening Visit measured by Slow Vital Capacity (SVC), or Forced Vital Capacity<br> (FVC).<br><br> - Patients must either not take Riluzole or be on a stable dose of Riluzole for = 30<br> days prior to the Master Protocol Screening Visit. Riluzole-naïve participants are<br> permitted in the study.<br><br> - Participants must either not take Edaravone or have completed at least one cycle of<br> edaravone prior to the Master Protocol Screening Visit. Edaravone-naïve participants<br> are permitted in the study.<br><br> - Participants must either not take Relyvrio (AMX0035) or be on a stable dose of<br> Relyvrio for = 30 days prior to the Master Protocol Screening Visit. Relyvrio-naïve<br> participants are permitted in the study.<br><br> - Women of child-bearing potential (defined as females who are not surgically sterile<br> or who are not over the age of 52 and amenorrhoeic for at least 12 months) must<br> utilize appropriate birth control throughout the study duration.<br><br> - Male patients must agree to use a medically acceptable method of contraception<br> /birth control throughout the study duration.<br><br>Exclusion Criteria:<br><br> - Subjects who meet one or more of the following criteria will not be considered<br> eligible to participate in the clinical study:<br><br> - Participation in another interventional clinical trial (drug or device) within 30<br> days of Screening and at any time during the study.<br><br> - Significant pre-existing organ dysfunction prior to randomization:<br><br> - Lung: Receiving supplemental home oxygen therapy at baseline for pre-existing<br> medical condition (other than COVID-19), as documented in medical record.<br><br> - Heart: Pre-existing congestive heart failure defined as an ejection fraction <20% as<br> documented in the medical record. Clinically significant ventricular arrhythmias<br> (ventricular tachycardia, ventricular fibrillation), unstable angina, myocardial<br> infarction (past 3 months), heart and coronary vessel surgery (past 3 months),<br> significant valvular heart disease, uncontrolled arterial hypertension with systolic<br> blood pressure >180 mm Hg and diastolic blood pressure >110 mm Hg.<br><br> - Renal: End-stage renal disease requiring renal replacement therapy or creatinine<br> clearance <50 mL/min.<br><br> - Hematologic: Baseline platelet count <30,000/mm3 or hemoglobin levels <6.0 g/dL.<br><br> - Neurological: Stage =3 hepatic encephalopathy by West Haven criteria.<br><br> - History of splenectomy or splenomegaly (spleen weighing > 750 g).<br><br> - Active cancer or history of cancer, except for the following: basal cell carcinoma<br> or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in<br> situ, prostatic carcinoma in situ, or other malignancies curatively treated and with<br> no evidence of disease recurrence for at least 3 years.<br><br> - Presence of unstable psychiatric disease, cognitive impairment, dementia or<br> substance abuse that would impair ability of the participant to provide informed<br> consent, in the SI's opinion.<br><br> - Exposure at any time to any gene therapies under investigation for the treatment of<br> ALS (off-label use or investigational) including tofersen (Qalsody).<br><br> - History of splenectomy or splenomegaly (spleen weighing >750 g).<br><br> - Co-infection with human immunodeficiency virus (HIV).<br><br> - History of organ or bone marrow transplantation, other than a corneal transplant.<br><br>or recent (within 3 months) chronic use of immunosuppressive drugs (tacrolimus,<br>mycofenolate mofetil, cyclosporine, rapamycine, hydrochloroquine, azathiopurine,<br>methotrexate), e.g., biologicals, JAK1/2 inhibitors, interferons, interleukins or<br>(prednisone or related corticosteroids are allowed).<br><br> - Hypersensitivity to either of the components of MRG-001.<br><br> - If female, known pregnancy, or has a positive serum pregnancy test, or<br> lactating/breastfeeding.<br><br> - Underlying diseases that, in the opinion of the site investigator, might be<br> complicated or exacerbated by proposed treatments or might confound assessment of<br> study drug.

Exclusion Criteria

Not provided

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Assessment of Treatment-Emergent Adverse Events

Secondary Outcome Measures

Name	Time	Method
Stem Cell Mobilization;Regulatory T-cell Mobilization;Change in Disease Progression