What side effects are associated with this type of intervention?

Common treatments for ALS include riluzole, edaravone, and investigational drugs like masitinib and neurotrophic factor-secreting mesenchymal stromal cells (MSC-NTF cells). Riluzole can cause side effects such as nausea, weakness, and liver function abnormalities. Edaravone may lead to bruising, gait disturbances, and allergic reactions. Masitinib has been associated with gastrointestinal issues and skin reactions. MSC-NTF cells are generally well-tolerated but can cause transient fever and headache. Dalfampridine, a potassium channel blocker, may cause seizures, urinary tract infections, and insomnia.

What prior FDA approvals exist?

The FDA has approved several treatments for Amyotrophic Lateral Sclerosis (ALS). Riluzole, approved in 1995, is the first drug shown to prolong survival. Edaravone, approved in 2017, helps slow the decline in daily functioning. While Dalfampridine, a potassium channel blocker, is primarily used for multiple sclerosis, it is being studied for its potential benefits in ALS. Other investigational treatments include masitinib, a tyrosine kinase inhibitor, and neurotrophic factor-secreting mesenchymal stromal cells, which are still under clinical evaluation.

What other types of research exist?

Promising novel alternatives to Dalfampridine for ALS patients include: <ol> <li>Zilucoplan: An investigational drug that was part of the HEALEY ALS Platform Trial, although it was stopped early for futility.</li> <li>Verdiperstat: Another investigational drug from the HEALEY ALS Platform Trial, recently reported as negative.</li> <li>CNM-Au8: A gold nanocrystal suspension under investigation for its potential neuroprotective effects.</li> <li>Pridopidine: A sigma-1 receptor agonist being studied for its neuroprotective properties.</li> <li>SLS-005 (Trehalose): A sugar molecule with potential autophagy-enhancing effects.</li> <li>Levosimendan: A calcium sensitizer that has shown some promise in improving respiratory muscle function in ALS patients.</li> <li>Reldesemtiv: A fast skeletal muscle troponin activator that has been evaluated for its impact on muscle function in ALS patients.</li> </ol>

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Potassium Channel Blocker

Dalfampridine for ALS

Phase 1

Waitlist Available

Led By Dale Lange, MD

Research Sponsored by Weill Medical College of Cornell University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

No current or exposure to any therapeutic agent targeting PLS or ALS within 30 days of enrollment

Impaired walking as measured by a Hauser Index of greater than 1 and less than 7 (2 to 6, inclusive)

Must not have

Active cancer within the previous 2 years, except treated basal cell carcinoma of the skin

Patient has been administered botulinum toxin in the lower extremities within 6 months prior to the screening visit and/or is expected to receive botulinum toxin in the lower extremities during the course of the study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up over the course of study at weeks 2, 4, 6, 10, 14, 18

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new medication, ropinirole hydrochloride, to see if it is safe and can be tolerated by patients. It focuses on people with specific motor neuron diseases, PLS or upper motor neuron predominant ALS. The effectiveness of the medication will be measured by checking if patients can walk faster over a short distance.

Who is the study for?

This trial is for adults aged 18-99 with primary lateral sclerosis (PLS) or upper motor neuron predominant ALS. Participants must have stable walking impairment, no severe allergies to dalfampridine, and a forced vital capacity over 60%. Women must use effective birth control. Exclusions include pregnancy, recent experimental drug use, certain medical conditions like liver disease or seizures, and metal implants above the neck.

What is being tested?

The study tests the safety and effectiveness of dalfampridine in improving walking speed in PLS/ALS patients over an 18-week period. It involves repeated timed walks to measure any improvement while on medication compared to baseline performance without medication.

What are the potential side effects?

Dalfampridine can cause side effects such as difficulty sleeping, dizziness, nausea, headache, weakness, back pain and balance problems. In rare cases it may increase seizure risk; hence it's not given to those with a history of seizures.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I haven't taken any drugs for PLS or ALS in the last 30 days.

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I have some difficulty walking but can still walk.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had active cancer in the last 2 years, except for treated skin cancer.

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I have received or will receive botulinum toxin in my legs within the last 6 months.

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I am not pregnant, breastfeeding, or trying to conceive.

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I do not have serious liver, kidney diseases, nerve issues, or certain genetic conditions.

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I am allergic to pyridine or ingredients in dalfampridine tablets.

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My kidney function is reduced with a creatinine clearance of ≤50 mL/min.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ over the course of study at weeks 2, 4, 6, 10, 14, 18

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~over the course of study at weeks 2, 4, 6, 10, 14, 18

This trial's timeline: 3 weeks for screening, Varies for treatment, and over the course of study at weeks 2, 4, 6, 10, 14, 18 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

consistent improvement in the Timed 25 Foot Walk test

Secondary study objectives

Effect of Dalfampridine on quality of life

Effects of Dalfampridine on functional status

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: AmpyraExperimental Treatment1 Intervention

Ampyra open label

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

dalfampridine

2013

Completed Phase 4

~90

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Motor Neuron Disease (MND) or Amyotrophic Lateral Sclerosis (ALS) include riluzole, edaravone, and sodium phenylbutyrate-taurursodiol. Riluzole works by inhibiting glutamate release, which helps reduce excitotoxicity, a process that damages neurons. Edaravone acts as a free radical scavenger, reducing oxidative stress that contributes to neuronal damage. Sodium phenylbutyrate-taurursodiol combines two drugs that reduce neuronal cell death by targeting different cellular pathways. These treatments are crucial as they aim to slow disease progression and improve quality of life. Dalfampridine, a potassium channel blocker, enhances signal conduction in damaged nerves, which may improve motor function. Understanding these mechanisms helps tailor treatment plans to manage symptoms and potentially slow disease progression in ALS patients.