Amyotrophic Lateral SclerosisPhase 2NCT06315608
MRG-001 in Patients With Amyotrophic Lateral Sclerosis
A Phase 2 study testing MRG-001 in people with Amyotrophic Lateral Sclerosis and Lou Gehrig Disease, run by MedRegen LLC.
Trial summary
The proposed study is an Open-Label, Single-Dose Study to Assess the Safety, and Pharmacodynamics (PD) signals of MRG-001 in Patients with Amyotrophic Lateral Sclerosis (ALS). MRG-001 will be administered subcutaneously 3 times per week for 2 weeks. This cycle will be repeated for 3 months. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Motor Neuron Disease
- What is being tested?
- MRG-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- No sites listed yet
- Study dates
- 1 Jul 2025to 1 Mar 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Able to provide written informed consent (either from patient or patient's legally acceptable representative and complying with study procedures, in the PI's opinion.
- Male or female patients between 18-75 years.
- Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.
- Time since onset of weakness due to ALS ≤ 48 months at the time of the Screening Visit
- Vital Capacity ≥ 50% of predicted capacity for age, height, and sex at the time of the Screening Visit measured by Slow Vital Capacity (SVC), or Forced Vital Capacity (FVC).
- Patients must either not take Riluzole or be on a stable dose of Riluzole for ≥ 30 days prior to the Master Protocol Screening Visit. Riluzole-naïve participants are permitted in the study.
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- Participants must either not take Edaravone or have completed at least one cycle of edaravone prior to the Master Protocol Screening Visit. Edaravone-naïve participants are permitted in the study.
- Participants must either not take Relyvrio (AMX0035) or be on a stable dose of Relyvrio for ≥ 30 days prior to the Master Protocol Screening Visit. Relyvrio-naïve participants are permitted in the study.
- Women of child-bearing potential (defined as females who are not surgically sterile or who are not over the age of 52 and amenorrhoeic for at least 12 months) must utilize appropriate birth control throughout the study duration.
- Male patients must agree to use a medically acceptable method of contraception /birth control throughout the study duration.
Who cannot join
- Subjects who meet one or more of the following criteria will not be considered eligible to participate in the clinical study:
- Participation in another interventional clinical trial (drug or device) within 30 days of Screening and at any time during the study.
- Significant pre-existing organ dysfunction prior to randomization:
- Lung: Receiving supplemental home oxygen therapy at baseline for pre-existing medical condition (other than COVID-19), as documented in medical record.
- Heart: Pre-existing congestive heart failure defined as an ejection fraction <20% as documented in the medical record. Clinically significant ventricular arrhythmias (ventricular tachycardia, ventricular fibrillation), unstable angina, myocardial infarction (past 3 months), heart and coronary vessel surgery (past 3 months), significant valvular heart disease, uncontrolled arterial hypertension with systolic blood pressure >180 mm Hg and diastolic blood pressure >110 mm Hg.
- Renal: End-stage renal disease requiring renal replacement therapy or creatinine clearance <50 mL/min.
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- Hematologic: Baseline platelet count <30,000/mm3 or hemoglobin levels <6.0 g/dL.
- Neurological: Stage ≥3 hepatic encephalopathy by West Haven criteria.
- History of splenectomy or splenomegaly (spleen weighing > 750 g).
- Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.
- Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the SI's opinion.
- Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational) including tofersen (Qalsody).
- History of splenectomy or splenomegaly (spleen weighing >750 g).
- Co-infection with human immunodeficiency virus (HIV).
- History of organ or bone marrow transplantation, other than a corneal transplant.
- or recent (within 3 months) chronic use of immunosuppressive drugs (tacrolimus, mycofenolate mofetil, cyclosporine, rapamycine, hydrochloroquine, azathiopurine, methotrexate), e.g., biologicals, JAK1/2 inhibitors, interferons, interleukins or (prednisone or related corticosteroids are allowed).
- Hypersensitivity to either of the components of MRG-001.
- If female, known pregnancy, or has a positive serum pregnancy test, or lactating/breastfeeding.
- Underlying diseases that, in the opinion of the site investigator, might be complicated or exacerbated by proposed treatments or might confound assessment of study drug.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: An Open-Label, Proof of Concept Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of MRG-001 in Patients With Amyotrophic Lateral Sclerosis
The proposed study is an Open-Label, Single-Dose Study to Assess the Safety, and Pharmacodynamics (PD) signals of MRG-001 in Patients with Amyotrophic Lateral Sclerosis (ALS). MRG-001 will be administered subcutaneously 3 times per week for 2 weeks. This cycle will be repeated for 3 months. In total, patients are expected to receive 18 injections over the span of 3 months.
- Conditions
- Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Motor Neuron Disease, Motor Neuron Atrophy
- Being tested
- MRG-001
- Keywords
- MRG-001, Stem Cells, Regulatory T-cells, Fixed-Dose Combination
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRG-001Drug
MRG-001 is a clear liquid solution for subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| MRG-001 (0.01 mL/kg) | Experimental | MRG-001 will be subcutaneously administered at 0.01 mL/kg 3 times per week every other day for two weeks per month (Day 0, 2, 4, 7, 9, 11). This cycle will be repeated 3 months in total. Receives: Drug: MRG-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of Treatment-Emergent Adverse Events
Assess the safety and tolerability of MRG-001 in patients with Amyotrophic Lateral Sclerosis (ALS) as determined by the absence of serious adverse events (SAE)
Measured: 3 Months
Secondary outcomes (3)
Stem Cell Mobilization
The absolute change in numbers of CD34+ hematopoetic stem cells as measured by flow cytometry
Measured: 24 hours
Regulatory T-cell Mobilization
The absolute change in numbers of FOXP3+ regulatory T-cells as measured by flow cytometry
Measured: 24 hours
Change in Disease Progression
Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) at Baseline, 1, 2 and 3 months. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function. Positive data means the reversal of ALS disease, negative data means disease progression, zero means stabilization of disease.
Measured: 3 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2024
- Study start1 Jul 2025 (estimated)
- Primary completion1 Jan 2026 (estimated)
- Study completion1 Mar 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- MedRegen LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ali R Ahmadi, MD PhD, Study director, MedRegen LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06315608
- Sponsor's protocol ID
- MRG24ALS
- Status last verified
- Aug 2024
- First submitted
- 12 Mar 2024
- Last updated
- 23 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Ahmadi AR, Atiee G, Chapman B, Reynolds L, Sun J, Cameron AM, Wesson RN, Burdick JF, Sun Z. A phase I, first-in-human study to evaluate the safety and tolerability, pharmacokinetics, and pharmacodynamics of MRG-001 in healthy subjects. Cell Rep Med. 2023 Sep 19;4(9):101169. doi: 10.1016/j.xcrm.2023.101169. Epub 2023 Aug 25.
PubMed 37633275
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Proof of Concept Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of MRG-001 in Patients With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT06315608
- Acronym / sponsor ID
- MRG24ALS
- Conditions
- Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Motor Neuron Disease, Motor Neuron Atrophy
- Keywords
- MRG-001, Stem Cells, Regulatory T-cells, Fixed-Dose Combination
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- MedRegen LLCIndustry (pharma, biotech, devices)
- Study start
- 1 Jul 2025 (estimated)
- Primary completion
- 1 Jan 2026 (estimated)
- Study completion
- 1 Mar 2026 (estimated)
- First posted
- 18 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The proposed study is an Open-Label, Single-Dose Study to Assess the Safety, and Pharmacodynamics (PD) signals of MRG-001 in Patients with Amyotrophic Lateral Sclerosis (ALS). MRG-001 will be administered subcutaneously 3 times per week for 2 weeks. This cycle will be repeated for 3 months. In total, patients are expected to receive 18 injections over the span of 3 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MRG-001 (0.01 mL/kg) | Experimental | Drug: MRG-001MRG-001 will be subcutaneously administered at 0.01 mL/kg 3 times per week every other day for two weeks per month (Day 0, 2, 4, 7, 9, 11). This cycle will be repeated 3 months in total. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Able to provide written informed consent (either from patient or patient's legally acceptable representative and complying with study procedures, in the PI's opinion.
- Male or female patients between 18-75 years.
- Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.
- Time since onset of weakness due to ALS ≤ 48 months at the time of the Screening Visit
- Vital Capacity ≥ 50% of predicted capacity for age, height, and sex at the time of the Screening Visit measured by Slow Vital Capacity (SVC), or Forced Vital Capacity (FVC).
Key exclusion criteria (19 listed)
- Subjects who meet one or more of the following criteria will not be considered eligible to participate in the clinical study:
- Participation in another interventional clinical trial (drug or device) within 30 days of Screening and at any time during the study.
- Significant pre-existing organ dysfunction prior to randomization:
- Lung: Receiving supplemental home oxygen therapy at baseline for pre-existing medical condition (other than COVID-19), as documented in medical record.
- Heart: Pre-existing congestive heart failure defined as an ejection fraction <20% as documented in the medical record. Clinically significant ventricular arrhythmias (ventricular tachycardia, ventricular fibrillation), unstable angina, myocardial infarction (past 3 months), heart and coronary vessel surgery (past 3 months), significant valvular heart disease, uncontrolled arterial hypertension with systolic blood pressure >180 mm Hg and diastolic blood pressure >110 mm Hg.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MRG-001MRG-001 is a clear liquid solution for subcutaneous injection | Drug | MRG-001 (0.01 mL/kg) |
Primary outcome
Assessment of Treatment-Emergent Adverse Events
Time frame: 3 Months
Assess the safety and tolerability of MRG-001 in patients with Amyotrophic Lateral Sclerosis (ALS) as determined by the absence of serious adverse events (SAE)
3 secondary outcomes
Stem Cell Mobilization
Time frame: 24 hours
Regulatory T-cell Mobilization
Time frame: 24 hours
Change in Disease Progression
Time frame: 3 Months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2024.
- Results publication: PubMed 37633275Ahmadi AR, Atiee G, Chapman B, Reynolds L, Sun J, Cameron AM, Wesson RN, Burdick JF, Sun Z. A phase I, first-in-human study to evaluate the safety and tolerability, pharmacokinetics, and pharmacodynamics of MRG-001 in healthy subjects. Cell Rep Med. 2023 Sep 19;4(9):101169. doi: 10.1016/j.xcrm.2023.101169. Epub 2023 Aug 25.
- Company ProfileLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ali R Ahmadi, MD PhD study director
MedRegen LLC
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MRG-001 in people with Amyotrophic Lateral Sclerosis and Lou Gehrig Disease, run by MedRegen LLC.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ali R Ahmadi, MD PhD (443-759-8563, info@medregenco.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessment of Treatment-Emergent Adverse Events (time frame: 3 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06315608, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Aug 2024.