MDA For Strength Independence & Life

Hope Through Research

At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently-needed answers to our families.

Amyotrophic Lateral Sclerosis (ALS) Clinical Trials

A clinical trial is a test in humans to evaluate biomedical or health-related outcomes, including experimental medications or therapies. Clinical trials are experiments, not treatments, and participation requires careful consideration.

Although it's possible to benefit from participating in a clinical trial, it's also possible that no benefit — or even harm — may occur. Keep your MDA clinic doctor informed about any clinical trial participation. (Note that MDA has no ability to influence who is chosen to participate in a clinical trial.)

Clinical trials are conducted in a series of successive steps, called phases. Each phase is designed to answer a separate research question.

  • Observational: Tests health or biomedical outcomes to understand the disease course. These studies are conducted without an intervention (e.g., an investigational new drug).
  • Phase 0: Tests how a new drug distributes in the body at a very low dose (below what is expected to have a therapeutic benefit).
  • Phase 1: Tests whether a new drug is safe in a small group of people, and determines the best way to dose it.
  • Phase 2: Tests whether a new drug is effective in a medium-sized group of people, and confirms its safety.
  • Phase 3: Tests whether a new drug is effective in a large group of people, and monitors its side effects and safety.
  • Phase 4: Studies the drug after it has been marketed to understand the benefits and side effects associated with long-term use.

*Note: Some clinical trials are not assigned a phase by the U.S. Food and Drug Administration (FDA). These include trials that test devices or behavioral interventions.

Amyotrophic Lateral Sclerosis (ALS) Clinical Trials

Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Status: 
Not Yet Recruiting
Phase: 
Phase 2

Mexiletine in Sporadic Amyotrophic Lateral Sclerosis

Status: 
Recruiting
Phase: 
Phase 2

Clinical Trial of Ezogabine (Retigabine) in ALS Subjects

Status: 
Recruiting
Phase: 
Phase 2

MRI Biomarkers in ALS

Status: 
Recruiting

Spinal Interneuron Excitability in ALS

Status: 
Not Yet Recruiting

PRE-Symptomatic Studies in ALS

Status: 
Recruiting

Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS)

Status: 
Not Yet Recruiting
Phase: 
Phase 2

MIROCALS: Modifying Immune Response and OutComes in ALS

Status: 
Not Yet Recruiting
Phase: 
Phase 2

Clinical Research in ALS Study

Status: 
Recruiting

A Trial of Tocilizumab in ALS Subjects

Status: 
Recruiting
Phase: 
Phase 2

The Use of Airway Clearance Devices in ALS

Status: 
Not Yet Recruiting

Genomic Translation for ALS Care

Status: 
Recruiting

CNS10-NPC-GDNF for the Treatment of ALS

Status: 
Not Yet Recruiting
Phase: 
Phase 1

Find MDA
in your Community