64 Participants Needed

QRL-201 for ALS

Recruiting at 15 trial locations
QC
Overseen ByQurAlis Corporation
Approved in 4 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but if you are on approved ALS treatments, you must be on a stable dose during the study.

How is the drug QRL-201 different from other ALS drugs?

QRL-201 is unique because it is a new treatment being tested for ALS, while existing drugs like riluzole and edaravone only extend survival by a few months. The research does not provide specific details about QRL-201's mechanism or effects, but it is part of ongoing efforts to find more effective treatments for ALS.12345

Who Is on the Research Team?

AG

Angela Genge, MD

Principal Investigator

QurAlis Corporation

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

My condition shows signs of nerve damage affecting muscle movement.
I can undergo a lumbar puncture procedure.
My ALS symptoms started less than 2 years ago.
See 4 more

Exclusion Criteria

Currently enrolled in any other clinical study involving either an investigational product (IP) or off-label use of a drug or device
I have previously received stem cell or gene therapy.
Your lab test results show a significant health problem, as determined by the doctor in charge of the study.
See 3 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple-ascending doses of QRL-201 or placebo intrathecally

Approximately 60 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • QRL-201
Trial Overview 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.
How Is the Trial Designed?
4Treatment groups
Experimental Treatment
Placebo Group
Group I: QRL-201: Sporadic ALSExperimental Treatment1 Intervention
Group II: QRL-201: C9orf72-ALSExperimental Treatment1 Intervention
Group III: Placebo: Sporadic ALSPlacebo Group1 Intervention
Group IV: Placebo: C9orf72-ALSPlacebo Group1 Intervention

QRL-201 is already approved in European Union, United States, Canada, United Kingdom for the following indications:

🇪🇺
Approved in European Union as QRL-201 for:
🇺🇸
Approved in United States as QRL-201 for:
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Approved in Canada as QRL-201 for:
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Approved in United Kingdom as QRL-201 for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

QurAlis Corporation

Lead Sponsor

Trials
6
Recruited
270+

Published Research Related to This Trial

In the CENTAUR trial, the combination of oral sodium phenylbutyrate and taurursodiol significantly reduced levels of neuroinflammatory biomarkers YKL-40 and C-reactive protein (CRP) by approximately 20% and 30%, respectively, after 24 weeks compared to placebo.
The reductions in YKL-40 and CRP levels correlated with improved scores on the ALS Functional Rating Scale-Revised (ALSFRS-R), suggesting these biomarkers may serve as treatment-sensitive indicators of disease progression in ALS.
Effect of sodium phenylbutyrate and taurursodiol on plasma concentrations of neuroinflammatory biomarkers in amyotrophic lateral sclerosis: results from the CENTAUR trial.Bowser, R., An, J., Mehta, L., et al.[2023]
A Delphi panel of 24 ALS healthcare providers in France reached a consensus to adopt the new Gold Coast criteria for diagnosing ALS, replacing the more complex El Escorial criteria, which will streamline clinical practice.
AMX0035, a treatment targeting endoplasmic reticulum stress and mitochondrial dysfunction, is viewed as a promising new option for ALS management, especially when used alongside riluzole, pending confirmation from ongoing phase III trials.
Management of amyotrophic lateral sclerosis in clinical practice: Results of the expert consensus using the Delphi methodology.Cassereau, J., Bernard, E., Genestet, S., et al.[2023]
In a phase 2 trial (CENTAUR) involving adults with ALS, the coformulation of sodium phenylbutyrate-taurursodiol (PB-TURSO) significantly improved survival, with a median overall survival of 25.0 months compared to 18.5 months for the placebo group.
Participants who started treatment with PB-TURSO at baseline experienced a 6.5-month longer median survival, indicating that PB-TURSO not only slows functional decline but also offers survival benefits for ALS patients.
Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis.Paganoni, S., Hendrix, S., Dickson, SP., et al.[2022]

Citations

Effect of sodium phenylbutyrate and taurursodiol on plasma concentrations of neuroinflammatory biomarkers in amyotrophic lateral sclerosis: results from the CENTAUR trial. [2023]
Management of amyotrophic lateral sclerosis in clinical practice: Results of the expert consensus using the Delphi methodology. [2023]
Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis. [2022]
MN-166 (ibudilast) in amyotrophic lateral sclerosis in a Phase IIb/III study: COMBAT-ALS study design. [2022]
New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022. [2022]
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