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.

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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

Must not have

Prior exposure to stem cell or gene therapy products

Pathogenic variant, likely pathogenic variant, or variant of uncertain significance in the superoxide dismutase 1 (SOD1) and/or fused in sarcoma (FUS) genes

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline through day 421 [end of study visit

Awards & highlights

Summary

This trial tests the safety and tolerability of QRL-201, a new drug, in people with ALS. The drug is given directly into the spinal fluid to better reach the nervous system.

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.

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.

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

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I can undergo a lumbar puncture procedure.

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My condition shows signs of nerve damage affecting muscle movement.

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

Exclusion Criteria

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

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I have previously received stem cell or gene therapy.

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I have a genetic variant in the SOD1 or FUS genes.

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I have a significant infection or an ongoing inflammatory condition.

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I cannot receive medications directly into my spinal canal.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline through day 421 [end of study visit

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline through day 421 [end of study visit

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through day 421 [end of study visit for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

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

Secondary study objectives

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

Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: QRL-201: Sporadic ALSExperimental Treatment1 Intervention

Multiple-ascending doses of QRL-201 will be intrathecally administered to individuals with ALS.

Group II: QRL-201: C9orf72-ALSExperimental Treatment1 Intervention

QRL-201 will be intrathecally administered to individuals with C9orf72-ALS.

Group III: Placebo: Sporadic ALSPlacebo Group1 Intervention

Multiple-ascending doses of placebo comparator will be intrathecally administered to individuals with ALS.

Group IV: Placebo: C9orf72-ALSPlacebo Group1 Intervention

Placebo comparator will be intrathecally administered to individuals with C9orf72-ALS.

0 / 7Eligibility 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

Common treatments for Amyotrophic Lateral Sclerosis (ALS) include riluzole, edaravone, and sodium phenylbutyrate-taurursodiol. Riluzole works by inhibiting glutamate release, which is thought to reduce excitotoxicity that leads to motor neuron death. Edaravone acts as an antioxidant, reducing oxidative stress and slowing functional decline. Sodium phenylbutyrate-taurursodiol combines two compounds that reduce neuronal cell death by targeting cellular stress pathways. These treatments are crucial for ALS patients as they aim to slow disease progression, preserve motor function, and improve quality of life.

Translating biological findings into new treatment strategies for amyotrophic lateral sclerosis (ALS).