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Open ALS Clinical Trials

Our program offers patients two types of clinical trials: ones that explore new ways to treat ALS, and ones that aim to learn more about the disease. Both types of studies are equally important to our mission to ultimately uncover pathways to prevent ALS in the first place.

Dr. Maragakis speaking with a patient

Therapeutic studies for ALS

Bringing treatment to ALS patients is the main mission of this program’s efforts. Our goal is to enroll all potential ALS patients into clinical trials that could help slow, halt, and eventually improve ALS. These therapies can take the form of traditional medications taken in pill form or by injection, but have also included gene therapy studies and the study of medical devices.


REViVALS-1A/HELIXMITH

A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety of Engensis in Participants with Amyotrophic Lateral Sclerosis

Status: Active, Enrolling
Principal Investigator: Nicholas Maragakis, M.D.
Contact: Betsy Mosmiller | 410-502-0495
Route of Administration: Intramuscular Injection
Length of Study: ~6 Months

Hepatocyte growth factor (HGF) is a protein your body naturally produces in small amounts that can protect nerves and cause the growth of new blood vessels. Unfortunately, your body only makes a small amount of this protein and not always in the areas where you need it. Researchers have isolated the genes responsible for directing the production of HGF and have designed a product called Engensis, containing these HGF genes that will be injected into your legs, hands and arms. Engensis is a special plasmid containing 2 pieces of DNA for 2 different types of HGF, making it more effective. Once Engensis is absorbed into your muscles, your cells use their natural mechanisms to read the DNA and produce HGF. This process begins almost immediately after injection and lasts for approximately 30 days. Important to note, once Engensis is in your body, the DNA is not mixed or incorporated into your own genes (DNA). There is a 2:1 ratio of study drug to placebo.


MERIDIAN

A Phase 2, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of pegcetacoplan in subjects with amyotrophic lateral sclerosis (ALS)

Status: Active, Enrolling
Principal Investigator: Nicholas Maragakis, M.D.
Contact: Betsy Mosmiller | 410-502-0495
Route of Administration: Subcutaneous infusion + open label extension (OLE)
Length of Study: ~52 Weeks + 52 Weeks OLE

This study is being done to assess the safety and effectiveness of pegcetacoplan (also know as APL-2) in people with ALS. Pegcetacoplan acts by turning down part of your immune system that helps get rid of pathogens that cause disease; this system is called the complement system. Under normal conditions, the complement system will help remove pathogens and damaged cells without hurting your body. However, sometimes uncontrolled or improper activation of the complement system can damage your body. Activation of the complement system is believed to play a role in the development and progression of ALS based on research in people with ALS and ALS mouse models. You will have twice weekly, at home, subcutaneous (under the skin) infusions.


HEALEY

Platform Trial for the Treatment of Amyotrophic Lateral Sclerosis (ALS): A Perpetual Multi-Center, Multi-Regimen Clinical Trial Evaluating the Safety and Efficacy of Investigational Products for the Treatment of ALS

Status: Active, Enrolling
Principal Investigator: Nicholas Maragakis, M.D.
Contact: Betsy Mosmiller | 410-502-0495
Route of Administration: Varies with each regimen/drug group
Length of Study: ~30 Weeks

The HEALEY ALS Platform Trial is a research trial that tests the safety and effectiveness of multiple treatments in ALS. A regimen is a specific course of treatment, each with a different study drug.

Multiple different regimens may be active within the platform trial at the same time. If you qualify for the platform trial, you will be randomly assigned to a regimen (one treatment). You will have an equal chance of being assigned to any active regimen. However, if you complete a regimen and then choose to participate in another regimen, you will not be randomized to your first regimen again.

Within a regimen, you will have a 3 in 4 chance of being assigned to the active drug group, and a 1 in 4 chance of being assigned to the placebo group.


ORAL EDARAVONE

A Phase 3b Multicenter, Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Oral Edaravone Administered for a Period of 48 Weeks in Subjects with Amyotrophic Lateral Sclerosis (ALS) (MT-1186-A02)

Status: Active, Enrolling
Principal Investigator: Jeffrey Rothstein, M.D., Ph.D.
Contact: Betsy Mosmiller | 410-502-0495
Route of Administration: Oral/liquid solution
Length of Study: ~58 Weeks

The intravenous form of edaravone has been approved as a treatment for ALS in 2017 by the FDA. Its commercial name is Radicava. This study is to test if the oral version (a liquid solution you will swallow) is safe and well tolerated in patients with ALS. Its effects will be compared in 2 different ways. One group will be given the study drug every day during the trial and the other will be given the drug and a placebo alternating period of the study cycles. (1st cycle 14 days of drug and 14 days of placebo, the 10 days of drug and 18 days of placebo for cycles 2-12). Neither you nor the study team will know to which group you will be assigned.


SUMMIT

Brain-Computer Interface (BCI) Implant for Severe Communication Disability

Status: Active, Enrolling
Principal Investigator:Nathan Crone, M.D.
Route of Administration: Implanted device
Length of Study: 1 year
Contact:410-955-6772

The Summit Study will test a specific, fully-implanted brain-computer interface (BCI), the Medtronic Summit System, for patients with chronic locked-in syndrome, a disorder in which communication is impaired by paralysis. The goal of the study is to assess this particular BCI’s ability to safely assist with communication. Participants must undergo surgery to implant electrodes onto the surface of the brain and a wireless transmitter under the skin of the chest. Participants will train and test (for up to 4 hours each day, two days per week for a full year) with the BCI to control assistive typing technology and trigger a care-giver calling alert at their own home. Participants must be able to travel to the Johns Hopkins Hospital several times a week. More about this study.


VALOR

A Phase I, Placebo-Controlled Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of BIIB067 Administered to Adult Subjects with Amyotrophic Lateral Sclerosis (Protocol 233AS101)

Status: Currently enrolling, "fast progressors" only
Principal Investigator: Nicholas J. Maragakis, M.D.
Contact: Betsy Mosmiller | 410-502-0495
Route of Administration: Intrathecal
Length of Study: ~36 weeks + optional open label extension

This research is being done to find out about the safety, tolerability and pharmacokinetics (PK) of a range of single (Part A) and repeated doses (Part B) of the study drug BIIB067, administered intrathecally (into the spine) in adult subjects with ALS. PK looks at the level of the study drug in your body and what happens to this level over time from the moment that it is given to you up to the point at which it is completely eliminated from your body. It is studied by measuring the amount of study drug that gets into your blood, urine and cerebrospinal fluid after you take the study drug. Cerebrospinal fluid (CSF) is the fluid around the spinal cord.

ALS is a disease that causes motor nerve cells to gradually break down and die. In most patients, the cause of ALS is not understood and doctors describe patients in this group as sporadic ALS patients. In a separate, small group of ALS patients (SOD1 ALS patients) the disease is caused by a genetic mutation in the SOD1 gene. The mutation of the SOD1 gene leads to the production of an abnormal SOD1 protein that is likely to be toxic to cells and could possibly lead to the nerve cell death seen in patients with ALS.

While sporadic ALS patients have two normal copies of the SOD1 gene, most SOD1 ALS patients have one mutated copy of the SOD1 gene and one normal copy of the SOD1 gene. The study drug BIIB067 is thought to bind to both the normal and mutated copies of the SOD1 gene. This binding may reduce the amount of both normal and toxic SOD1 protein. The main goal of this study is to determine if the study drug is tolerated by ALS subjects. A secondary goal is to determine if the study drug can reduce total SOD1 protein.


COMBAT

A Phase 2b/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, 12 Month Clinical Trial to Evaluate the Efficacy and Safety of MN-166 (Ibudiast) followed by an Open-Label Extension Phase in Subjects with Amyotrophic Lateral Sclerosis

Status: Currently enrolling
Principal Investigator: Nicholas J. Maragakis, M.D.
Contact: Betsy Mosmiller | 410-502-0495
Route of Administration: Intrathecal
Length of Study: ~13 months + ~6 months optional label extension

Ibudilast is being investigated as a potential new therapy to slow the progression of ALS. Ibudilast is thought to have neuroprotective effects by working against nervous system inflammation. Ibudilast is a capsule, taken orally with a 1:1 ratio of study drug to placebo. This study has a 12 month double-blind phase, followed by a 6 month open label extension period. Patients who enroll in this study must also be taking the approved ALS drug Riluzole (Rilutek®). Patients taking the approved ALS drug Edaravone/Radicava® are not eligible for this study.

Ibudilast (developed by MediciNova) acts as a nonselective phosphodiesterase inhibitor and has been used as an anti-asthmatic medication as it reduces release of leukotrienes, cytokines and other molecules involved in bronchospasm. It also modulates survival and activation of immune cells in the CNS by preventing the production of pro-inflammatory agents from microglia. It also modulates release of migration inhibitory factor (MIF).


A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB105 Administered Intrathecally to Adults With Amyotrophic Lateral Sclerosis With or Without Poly-CAG Expansion in the Ataxin-2 Gene

Status: Currently Enrolling
Principal Investigator: Jeffrey Rothstein, M.D., Ph.D.
Contact: Betsy Mosmiller | 410-502-0495
Route of Administration: Intrathecal (Lumbar Puncture)
Length of Study: ~6 months

This research is being done to find out about the safety, tolerability and pharmacokinetics (PK) of the study drug BIIB105, administered intrathecally (into your spinal fluid) in adult subjects with ALS with or without a mutation in the Ataxin-2 Gene. PK looks at the level of the study drug in your body and what happens to this level over time from the moment that it is given to you up to the point at which it is completely eliminated from your body. It is studied by measuring the amount of study drug that gets into your blood, urine and cerebrospinal fluid after you take the study drug. Cerebrospinal fluid (CSF) is the fluid around the spinal cord. Subjects will be assigned to one of 7 cohorts, depending on their ALS genetic status.


A Phase 2A Open Label, Multi-center Study to Evaluate the Safety and Tolerability of Multiple Doses of AT-1501 in Adults with ALS

Status: Currently Enrolling
Principal Investigator: Nicholas Maragakis, M.D.
Contact: Betsy Mosmiller | 410-502-0495
Route of Administration: IV Infusion
Length of Study: ~19 weeks

This study is being done to determine the safety and tolerability of multiple doses of AT-1501 in adults with ALS. AT-1501 (Developed by Anelixis) is an investigational antibody that targets the CD40 ligand (CD40LG), a protein present at the surface of some white blood cells that is involved in inflammation. In ALS, the CD40LG protein is produced in excess and is thought to be involved in neurodegeneration. This compound demonstrated preclinical results in a mouse model of ALS and in other preclinical studies and has a good safety profile. As an open label study, all participants will receive the study drug.


MASITINIB

A Prospective, Multicenter, Randomised, Double-blind, Placebo-controlled, Parallel Groups, Phase 3 Study to Compare the Efficacy and Safety of Masitinib in Combination With Riluzole Versus Placebo in Combination With Riluzole in the Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)

Status: Currently Enrolling
Principal Investigator: Jeffrey Rothstein, M.D., Ph.D.
Contact: Betsy Mosmiller | 410-502-0495
Route of Administration: Oral
Length of Study: ~19 weeks

Masitinib (developed by ABSciences) is a selective, oral tyrosine kinase inhibitor with neuroprotective capability, demonstrated via numerous preclinical studies. It is a twice daily, oral study drug that aims to be neuroprotective and to reduce inflammation in specialized brain cells called microglia and mast cells that are affected in people with ALS. This is a phase 3 study. It includes 3 groups: 2 groups of different dose strengths and one placebo group. Participants must have had symptoms of ALS for less than 2 years and must be taking Riluzole.

Betsy Mosmiller using a lung function test

ALS Biomarker Studies

Our greatest limitation to treating Amyotrophic Lateral Sclerosis (ALS) is the lack of understanding what causes or enables sporadic ALS. However, Johns Hopkins researchers are uncovering powerful discoveries for this progressive disease. These discoveries start in research laboratories across the institution as well as collaborations with investigators across the globe.

Study of [11C]CPPC to Assess the Safety and Tolerability in Patients With ALS

Status: Active, Enrolling
Principal Investigator: Nicholas Maragakis, M.D.
Contact: Betsy Mosmiller | 410-502-0495
Route of Administration: Intravenous single dose injection
Length of Study: 6 weeks

This is a Phase 1 study of safety and tolerability of an investigational radiotracer drug called [5-cyano-N-(4-(4-[11C]Methylpiperazin-1-yl)-2-(Piperidin-1-yl)Phenyl)Furan-2-carboxamide] ([11C]CPPC). A radiotracer is a substance that chemically marks certain structures in the body. In this case, [11C]CPPC highlights structures expressing colony stimulating factor receptor (CSF1R), a receptor that is expressed on microglial cells. A safety and tolerability study is looking to see if there are any unanticipated, possibly harmful, effects of the use of the radiotracer in humans. However, ultimately, the investigators would like to know if this drug can be used to make better images of the brain for patients with amyotrophic lateral sclerosis (ALS), which could help doctors better understand the disease and help take care of patients with ALS. This study will use a radiotracer to look for a chemical receptor which ALS patients have more of in the brain. After receiving the radiotracer, participants' brains will be scanned with a positron emission tomography (PET) imaging machine.


REFINE

Radicava (Edaravone) Biomarker Study in Participants with Amyotrophic Lateral Sclerosis

Status: Currently Enrolling
Principal Investigator: Nicholas J. Maragakis, M.D.
Contact: Betsy Mosmiller | 410-502-0495
Length of Study: ~8 months

This is an observational study in which participants will be followed from prior to starting Radicava through 6 months of treatment. Biomarkers from blood and urine will be collected and analyzed to help better understand Edaravone’s effect on disease progression and its mechanism of action.

Browse other clinical trials

For a complete list of open clinical trials across Johns Hopkins, visit the database at the Institute for Clinical and Translational Research. You can search by condition, researcher or doctor’s name.

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