What side effects are associated with this type of intervention?

Common treatments for ALS, such as edaravone and riluzole, have known side effects. Edaravone can cause bruising, gait disturbance, headache, and respiratory issues. Riluzole is associated with side effects like nausea, weakness, decreased lung function, and liver enzyme abnormalities. Other treatments under investigation, such as masitinib and skeletal muscle activators, have reported side effects including gastrointestinal issues, fatigue, and potential neuroinflammatory reactions. Overall, while these treatments aim to slow disease progression, they come with a range of mild to moderate side effects that should be monitored by healthcare providers.

What prior FDA approvals exist?

The FDA has approved several treatments for ALS, including riluzole and edaravone. Riluzole works by inhibiting glutamate release, which is thought to reduce the damage to motor neurons. Edaravone is an antioxidant that helps to reduce oxidative stress, a contributing factor in ALS progression. Other investigational treatments, such as masitinib, a tyrosine kinase inhibitor, and neurotrophic factor-secreting mesenchymal stromal cells, are being studied for their potential neuroprotective and anti-inflammatory effects. These treatments aim to slow disease progression and improve quality of life for ALS patients.

What other types of research exist?

Promising alternatives to QRL-201 for ALS include Edaravone and Sigma-1 receptor (S1R) agonist PRE-084. Edaravone has shown efficacy in slowing functional decline in early-stage ALS patients, with a clinically significant slowing of approximately 33% in functional decline over 24 weeks. It is administered either intravenously or orally. PRE-084, a Sigma-1 receptor agonist, has demonstrated neuroprotective effects and improved motor function in preclinical models of ALS.

← Back to Search

Other

QRL-201 for ALS

Phase 1

Recruiting

Research Sponsored by QurAlis Corporation

Eligibility Criteria Checklist

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

Must have

Able to tolerate lumbar puncture

Clinical evidence of lower motor neuron involvement

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline through 253 days

Awards & highlights

Study Summary

This trial tests a new drug to see if it's safe and effective in treating ALS.

Who is the study for?

This trial is for adults aged 18-80 with ALS, who have had symptoms start within the last 2 years. They must be able to perform a breathing test and not be on varying doses of ALS therapies. Participants should not be pregnant or nursing, can undergo spinal taps, and must use contraception.Check my eligibility

What is being tested?

The study tests the safety of different doses of QRL-201 in people with ALS. It involves multiple levels of dosing to find out which is safest and most tolerable for patients.See study design

What are the potential side effects?

While specific side effects are not listed, they may include reactions related to various dosages of QRL-201 or procedures like lumbar puncture. Safety and tolerability are key focuses, so monitoring for any adverse effects will be part of the trial.

Eligibility Criteria

Inclusion Criteria

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

Select...

I can undergo a lumbar puncture procedure.

Select...

My condition shows signs of nerve damage affecting muscle movement.

Select...

I am between 18 and 80 years old and have been diagnosed with ALS.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline through 253 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline through 253 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through 253 days for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of participants with one or more treatment emergent adverse events and serious adverse events

Secondary outcome measures

Pharmacokinetics (plasma): Area under the concentration time curve from zero to infinity (AUCinf) of QRL-201

Pharmacokinetics (plasma): Maximum observed concentration of QRL-201 (Cmax)

Pharmacokinetics (plasma): Time of maximum concentration (Tmax) of QRL-201