Amyotrophic Lateral SclerosisPhase 1NCT05633459
A Study Evaluating the Safety and Tolerability of QRL-201 in ALS
A Phase 1 study testing Multiple ascending doses of QRL-201 and QRL-201 in people with Amyotrophic Lateral Sclerosis, run by QurAlis Corporation at 15 sites in 6 countries.
Trial summary
The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Multiple ascending doses of QRL-201 and QRL-201
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 69 participants
- Where
- 15 sitesin 6 countries
- Study dates
- 16 Dec 2022to 30 Nov 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female participants aged 18 to 80 years diagnosed with ALS
- ALS symptom onset within 24 months of Screening
- Slow vital capacity >50%
- Clinical or electrodiagnostic evidence of lower motor neuron involvement
- Not pregnant and not nursing
- Willing and able to practice effective contraception
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- Able to tolerate lumbar puncture
- If on approved therapies for the treatment of ALS during the course of the study, must be on a stable dose (at the Sponsor's discretion)
- Male or female participants in the sporadic ALS and C9orf72 ALS Cohorts of QRL-201-01 who have completed through at least Day 253 of safety follow up in the double-blind study period OR have completed Cohort 1 of Cohort 2
- Not pregnant and not nursing
- Willing and able to practice effective contraception
- Can visit study site for required visits
- Able to tolerate lumbar puncture
- If on approved therapies for the treatment of ALS during the course of the study, must be on a stable dose (at the Sponsor's discretion)
Who cannot join
- Pathogenic variant, likely pathogenic variant, or variant of uncertain significance in the superoxide dismutase 1 (SOD1) and/or fused in sarcoma (FUS) genes
- Currently enrolled in any other clinical study involving either an investigational product (IP) or off-label use of a drug or device
- Prior exposure to stem cell or gene therapy products
- Any contraindication to intrathecal drug administration
- Abnormal laboratory values deemed clinically significant by the Investigator
- Significant infection or known inflammatory process
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- Any sign and/or history of neurological conditions and other neuromuscular disorders that could affect the electrophysiological recordings.
- An EEG that shows signs of abnormal electrical activity (e.g., epilepsy)
- Currently enrolled in any other clinical study involving either an investigational product (IP) or off label use of a drug or device
- Off-label use of drugs or devices that are being used as disease-modifying therapies in ALS
- Participated in another clinical study within 30 days, or 5 half-lives of the previous investigational product, whichever is greater, prior to the first dose
- Prior exposure to stem cell or gene therapy products
- Is not suitable for inclusion in the Open-Label Extended Dosing period of the study, in the opinion of the Investigator
- An EEG that shows signs of abnormal electrical activity (NOTE: Cohort 1 and Cohort 2 participants only)
Trial locations and contacts
15 sites in 6 countries.
15 sites in 6 countries
Canada5 sites
University of Calgary
Calgary, Alberta, T2N 1N4
University of Alberta
Edmonton, Alberta, T6G 2G3
CHUM - Hopital Notre-Dame
Montreal, Quebec, H2L 4M1
Montreal Neurological Institute-Hospital
Montreal, Quebec, H3A 2B4
Sunnybrook Health Science Centre
Toronto, Ontario, M4N 3M5
Germany4 sites
Charité Research Organisation
Berlin, 10117
Deutsches Zentrum für Neurodegenerative Erkrankungen e. V. (DZNE)
Bonn, North Rhine-Westphalia, 53127
University Hospital Schleswig-Holstein (UKSH) Campus Lübeck, Department for Neurology/ Precision Neurology
Lübeck
Universitätsklinikum Ulm
Ulm, 89081
United Kingdom3 sites
Kings College Hospital NHS Foundation Trust
London, SE5 9RS
National Hospital for Neurology and Neurosurgery
London, WC1N 3BG
The University of Sheffield, Royal Hallamshire Hospital
Sheffield, United Kingdom, S10 2JF
About this study
Official title: A Multi-Center, Randomized, Double-Blind Placebo Controlled Multiple-Ascending Dose Study to Evaluate the Safety and Tolerability of QRL-201 in Amyotrophic Lateral Sclerosis, Followed by an Open-Label Extended Dosing Period
The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS
Read the detailed description
This first-in-human, Phase 1 study will evaluate the safety, tolerability, and pharmacokinetics (PK) of QRL-201 administered intrathecal (IT) to participants with Amyotrophic Lateral Sclerosis. Two dose escalation cohorts of 8 participants each, followed by an additional 48 participants, receiving the study drug in a 6:2 ratio of QRL-201 to placebo.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Multiple ascending doses of QRL-201, Multiple ascending doses of Placebo, QRL-201, Placebo
- Keywords
- ALS, Stathmin-2, STMN2, ASO, Antisense Oligonucleotide
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Multiple ascending doses of QRL-201Drug
Multiple ascending doses of QRL-201 will be intrathecally administered to individuals with sporadic ALS.
Multiple ascending doses of PlaceboDrug
Multiple ascending doses of placebo comparator will be intrathecally administered to individuals with sporadic ALS.
QRL-201Drug
QRL-201 will be intrathecally administered to individuals with C9orf72 ALS.
PlaceboDrug
Placebo comparator will be intrathecally administered to individuals with C9orf72 ALS.
QRL-201Drug
QRL-201 will be intrathecally administered to all participants who enter the Open-Label Extended Dosing Period.
| Study group | Role | What they receive |
|---|---|---|
| QRL-201: Sporadic ALS | Experimental | Multiple-ascending doses of QRL-201 will be intrathecally administered to individuals with ALS. Receives: Drug: Multiple ascending doses of QRL-201 |
| Placebo: Sporadic ALS | Placebo comparator | Multiple-ascending doses of placebo comparator will be intrathecally administered to individuals with ALS. Receives: Drug: Multiple ascending doses of Placebo |
| QRL-201: C9orf72-ALS | Experimental | QRL-201 will be intrathecally administered to individuals with C9orf72-ALS. Receives: Drug: QRL-201 |
| Placebo: C9orf72-ALS | Placebo comparator | Placebo comparator will be intrathecally administered to individuals with C9orf72-ALS. Receives: Drug: Placebo |
| QRL-201: Open-label Extended Dosing Period | Experimental | Open-label QRL-201 will be intrathecally administered to individuals with ALS who qualify for and enter the open-label extended dosing period. Receives: Drug: QRL-201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with one or more treatment emergent adverse events and serious adverse events
Endpoints: A summary of treatment emergent adverse events, serious adverse events, and other non-serious adverse events, regardless of causality, will be reported in the Reported Adverse Events module.
Measured: Baseline through Day 421 [End of Study Visit
Secondary outcomes (3)
Pharmacokinetics (plasma): Maximum observed concentration of QRL-201 (Cmax)
Endpoints: PK: Cmax of QRL-201
Measured: Predose up to 24 hours post dose
Pharmacokinetics (plasma): Area under the concentration time curve from zero to infinity (AUCinf) of QRL-201
Endpoints: PK: AUC (0-inf) of QRL-201
Measured: Predose up to 24 hours post dose
Pharmacokinetics (plasma): Time of maximum concentration (Tmax) of QRL-201
Endpoints: PK: Tmax of QRL-201
Measured: Predose up to 24 hours postdose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Dec 2022
- Study start16 Dec 2022
- Primary completion29 May 2026 (estimated)
- Study completion30 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Parallel Assignment
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 69 participants (actual)
Sponsor and investigators
- Lead sponsor
- QurAlis Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Manoj Malhotra, MD, Study director, QurAlis Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05633459
- Sponsor's protocol ID
- QRL-201-01
- Status last verified
- May 2026
- First submitted
- 21 Nov 2022
- Last updated
- 15 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Randomized, Double-Blind Placebo Controlled Multiple-Ascending Dose Study to Evaluate the Safety and Tolerability of QRL-201 in Amyotrophic Lateral Sclerosis, Followed by an Open-Label Extended Dosing Period
- ClinicalTrials.gov ID
- NCT05633459
- Acronym / sponsor ID
- QRL-201-01
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Stathmin-2, STMN2, ASO, Antisense Oligonucleotide
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 69 participants (actual)
- Lead sponsor
- QurAlis CorporationIndustry (pharma, biotech, devices)
- Study start
- 16 Dec 2022 (actual)
- Primary completion
- 29 May 2026 (estimated)
- Study completion
- 30 Nov 2027 (estimated)
- First posted
- 1 Dec 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS
Detailed description
This first-in-human, Phase 1 study will evaluate the safety, tolerability, and pharmacokinetics (PK) of QRL-201 administered intrathecal (IT) to participants with Amyotrophic Lateral Sclerosis. Two dose escalation cohorts of 8 participants each, followed by an additional 48 participants, receiving the study drug in a 6:2 ratio of QRL-201 to placebo.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
- Model description
- Parallel Assignment
| Arm | Type | Receives |
|---|---|---|
| QRL-201: Sporadic ALS | Experimental | Drug: Multiple ascending doses of QRL-201Multiple-ascending doses of QRL-201 will be intrathecally administered to individuals with ALS. |
| Placebo: Sporadic ALS | Placebo comparator | Drug: Multiple ascending doses of PlaceboMultiple-ascending doses of placebo comparator will be intrathecally administered to individuals with ALS. |
| QRL-201: C9orf72-ALS | Experimental | Drug: QRL-201QRL-201 will be intrathecally administered to individuals with C9orf72-ALS. |
| Placebo: C9orf72-ALS | Placebo comparator | Drug: PlaceboPlacebo comparator will be intrathecally administered to individuals with C9orf72-ALS. |
| QRL-201: Open-label Extended Dosing Period | Experimental | Drug: QRL-201Open-label QRL-201 will be intrathecally administered to individuals with ALS who qualify for and enter the open-label extended dosing period. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Male or female participants aged 18 to 80 years diagnosed with ALS
- ALS symptom onset within 24 months of Screening
- Slow vital capacity >50%
- Clinical or electrodiagnostic evidence of lower motor neuron involvement
- Not pregnant and not nursing
Key exclusion criteria (14 listed)
- Pathogenic variant, likely pathogenic variant, or variant of uncertain significance in the superoxide dismutase 1 (SOD1) and/or fused in sarcoma (FUS) genes
- Currently enrolled in any other clinical study involving either an investigational product (IP) or off-label use of a drug or device
- Prior exposure to stem cell or gene therapy products
- Any contraindication to intrathecal drug administration
- Abnormal laboratory values deemed clinically significant by the Investigator
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Multiple ascending doses of QRL-201Multiple ascending doses of QRL-201 will be intrathecally administered to individuals with sporadic ALS. | Drug | QRL-201: Sporadic ALS |
| Multiple ascending doses of PlaceboMultiple ascending doses of placebo comparator will be intrathecally administered to individuals with sporadic ALS. | Drug | Placebo: Sporadic ALS |
| QRL-201QRL-201 will be intrathecally administered to individuals with C9orf72 ALS. | Drug | QRL-201: C9orf72-ALS |
| PlaceboPlacebo comparator will be intrathecally administered to individuals with C9orf72 ALS. | Drug | Placebo: C9orf72-ALS |
| QRL-201QRL-201 will be intrathecally administered to all participants who enter the Open-Label Extended Dosing Period. | Drug | QRL-201: Open-label Extended Dosing Period |
Primary outcome
Number of participants with one or more treatment emergent adverse events and serious adverse events
Time frame: Baseline through Day 421 [End of Study Visit
Endpoints: A summary of treatment emergent adverse events, serious adverse events, and other non-serious adverse events, regardless of causality, will be reported in the Reported Adverse Events module.
3 secondary outcomes
Pharmacokinetics (plasma): Maximum observed concentration of QRL-201 (Cmax)
Time frame: Predose up to 24 hours post dose
Pharmacokinetics (plasma): Area under the concentration time curve from zero to infinity (AUCinf) of QRL-201
Time frame: Predose up to 24 hours post dose
Pharmacokinetics (plasma): Time of maximum concentration (Tmax) of QRL-201
Time frame: Predose up to 24 hours postdose
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Manoj Malhotra, MD study director
QurAlis Corporation
15 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Multiple ascending doses of QRL-201 and QRL-201 in people with Amyotrophic Lateral Sclerosis, run by QurAlis Corporation at 15 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 15 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in Canada, Germany, United Kingdom, Belgium, Ireland and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with one or more treatment emergent adverse events and serious adverse events (time frame: Baseline through Day 421 [End of Study Visit). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 May 2026.