Amyotrophic Lateral SclerosisPhase 1NCT06714396
A Study Exploring the PK/PD Relationship of QRL-101 in Adults With ALS
A Phase 1 study testing QRL-101 in people with Amyotrophic Lateral Sclerosis, run by QurAlis Corporation at 1 site in Netherlands.
Trial summary
This Phase 1 randomized, placebo-controlled, double-blind study will evaluate the PK/PD relationship of single doses of QRL-101 in 12 ALS participants. The study will also assess safety and tolerability in participants receiving either QRL-101 or placebo. 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?
- QRL-101
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 1 siteNetherlands
- Study dates
- 11 Nov 2024to 22 Aug 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Age 18 to 80 years of age inclusive at the time of signing the informed consent.
- Diagnosis of ALS, by either Gold Coast Criteria or El Escorial Criteria (possible, probable laboratory supported, probable, or definite)
- Clinical chemistry laboratory values within acceptable range for the population, as per investigator judgment.
- Body mass index of 18 to 32 kg/m2 (inclusive).
- Willing and able to practice effective contraception.
Who cannot join
- Currently enrolled in any other clinical trial involving a study drug, off-label drug or device use, or any other type of medical research judged not scientifically or medically compatible with this study.
- History or presence of medical illness including, but not limited to, any cardiovascular, hepatic, respiratory, hematological, endocrine, psychiatric, or neurological disease, convulsions, or any clinically significant laboratory abnormality that, in the judgment of the investigator, indicate a medical problem that would preclude study participation.
- Off-label use of drugs or devices that are being used or investigated 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 planned randomization date.
- Taking any prescription or nonprescription medicines that are cytochrome P450 3A4 inducers, inhibitors, or substrates within 30 days or 5 half-lives, whichever is greater, prior to the planned randomization date
Trial locations and contacts
1 site in 1 country.
Netherlands1 site
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands, 9728 NZ
About this study
Official title: A Randomized, Placebo-Controlled, Double-Blind, Single-Dose, Phase 1 Study to Explore the PK/PD Relationship of QRL-101 in People Living With ALS
This Phase 1 randomized, placebo-controlled, double-blind study will evaluate the PK/PD relationship of single doses of QRL-101 in 12 ALS participants. The study will also assess safety and tolerability in participants receiving either QRL-101 or placebo.
Read the detailed description
This is a randomized, placebo-controlled, double-blind, single-dose, single-site, Phase 1 study to explore the pharmacokinetics (PK) and pharmacodynamics (PD) of single doses of QRL-101 in participants with ALS. In addition, the safety and tolerability of QRL-101 will be evaluated. Single doses of QRL-101 or placebo will be administered as an oral liquid to approximately 12 participants with ALS. The study will explore the PK, PD, and PK/PD relationship among three dose levels of QRL-101 and placebo. The randomization will be 1:1:1:1 for the four study groups and will be conducted in 3 blocks (cohorts) of 4, where participants will be randomized 3:1 (QRL-101:placebo) for increasing dose levels of QRL-101.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- QRL-101, Placebo
- 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.
QRL-101Drug
Single doses of QRL-101 will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.
PlaceboOther
Single doses of compartor placebo will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.
| Study group | Role | What they receive |
|---|---|---|
| QRL-101 | Experimental | Single doses of QRL-101 will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data. Receives: Drug: QRL-101 |
| Placebo | Experimental | Single doses of comparator placebo will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
QRL-101 plasma concentration
Measured: Baseline through Follow up (Day 10)
PK/PD Relationship: Strength-Duration Time Constant (SDTC)
Measured: Baseline through Follow up (Day 10)
Secondary outcomes (5)
Safety and Tolerability: Adverse Events (AEs)
Measured: Baseline through Follow up (Day 10)
Safety and Tolerability: Serious Adverse Events (SAEs)
Measured: Baseline through Follow up (Day 10)
Plasma PK profile: Maximum concentration observed (Cmax)
Measured: Baseline through Follow up (Day 10)
Plasma PK profile: Time of maximum drug concentration (Tmax)
Measured: Baseline through Follow up (Day 10)
Plasma PK profile: Area under the concentration time curve from time 0 to 24 hours (AUC0-24hr)
Measured: Baseline through Follow up (Day 10)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2024
- Study start11 Nov 2024
- Primary completion22 Aug 2025
- Study completion22 Aug 2025
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
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- QurAlis Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06714396
- Sponsor's protocol ID
- QRL-101-04
- Status last verified
- Aug 2025
- First submitted
- 27 Nov 2024
- Last updated
- 2 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Placebo-Controlled, Double-Blind, Single-Dose, Phase 1 Study to Explore the PK/PD Relationship of QRL-101 in People Living With ALS
- ClinicalTrials.gov ID
- NCT06714396
- Acronym / sponsor ID
- QRL-101-04
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- QurAlis CorporationIndustry (pharma, biotech, devices)
- Study start
- 11 Nov 2024 (actual)
- Primary completion
- 22 Aug 2025 (actual)
- Study completion
- 22 Aug 2025 (actual)
- First posted
- 3 Dec 2024 (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.
This Phase 1 randomized, placebo-controlled, double-blind study will evaluate the PK/PD relationship of single doses of QRL-101 in 12 ALS participants. The study will also assess safety and tolerability in participants receiving either QRL-101 or placebo.
Detailed description
This is a randomized, placebo-controlled, double-blind, single-dose, single-site, Phase 1 study to explore the pharmacokinetics (PK) and pharmacodynamics (PD) of single doses of QRL-101 in participants with ALS. In addition, the safety and tolerability of QRL-101 will be evaluated. Single doses of QRL-101 or placebo will be administered as an oral liquid to approximately 12 participants with ALS. The study will explore the PK, PD, and PK/PD relationship among three dose levels of QRL-101 and placebo. The randomization will be 1:1:1:1 for the four study groups and will be conducted in 3 blocks (cohorts) of 4, where participants will be randomized 3:1 (QRL-101:placebo) for increasing dose levels of QRL-101.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| QRL-101 | Experimental | Drug: QRL-101Single doses of QRL-101 will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data. |
| Placebo | Experimental | Other: PlaceboSingle doses of comparator placebo will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age 18 to 80 years of age inclusive at the time of signing the informed consent.
- Diagnosis of ALS, by either Gold Coast Criteria or El Escorial Criteria (possible, probable laboratory supported, probable, or definite)
- Clinical chemistry laboratory values within acceptable range for the population, as per investigator judgment.
- Body mass index of 18 to 32 kg/m2 (inclusive).
- Willing and able to practice effective contraception.
Key exclusion criteria (5 listed)
- Currently enrolled in any other clinical trial involving a study drug, off-label drug or device use, or any other type of medical research judged not scientifically or medically compatible with this study.
- History or presence of medical illness including, but not limited to, any cardiovascular, hepatic, respiratory, hematological, endocrine, psychiatric, or neurological disease, convulsions, or any clinically significant laboratory abnormality that, in the judgment of the investigator, indicate a medical problem that would preclude study participation.
- Off-label use of drugs or devices that are being used or investigated 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 planned randomization date.
- Taking any prescription or nonprescription medicines that are cytochrome P450 3A4 inducers, inhibitors, or substrates within 30 days or 5 half-lives, whichever is greater, prior to the planned randomization date
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 |
|---|---|---|
| QRL-101Single doses of QRL-101 will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data. | Drug | QRL-101 |
| PlaceboSingle doses of compartor placebo will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data. | Other | Placebo |
Primary outcome
QRL-101 plasma concentration
Time frame: Baseline through Follow up (Day 10)
PK/PD Relationship: Strength-Duration Time Constant (SDTC)
Time frame: Baseline through Follow up (Day 10)
5 secondary outcomes
Safety and Tolerability: Adverse Events (AEs)
Time frame: Baseline through Follow up (Day 10)
Safety and Tolerability: Serious Adverse Events (SAEs)
Time frame: Baseline through Follow up (Day 10)
Plasma PK profile: Maximum concentration observed (Cmax)
Time frame: Baseline through Follow up (Day 10)
Plasma PK profile: Time of maximum drug concentration (Tmax)
Time frame: Baseline through Follow up (Day 10)
Plasma PK profile: Area under the concentration time curve from time 0 to 24 hours (AUC0-24hr)
Time frame: Baseline through Follow up (Day 10)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2025.
Related trials for Amyotrophic Lateral Sclerosis
Recruiting
MindEx: A Novel, Multifocal, Cognitive Brain-Machine Interface System
Paralysis; Quadriplegic, Quadraplegia
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruitingPhase 1
A Study of Single Ascending Doses of NI112 in Adults With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
RecruitingObservational study
Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
Frontotemporal Dementia, Amyotrophic Lateral Sclerosis
Recruiting
Anticipated Patient and Caregiver Burden
Amyotrophic Lateral Sclerosis
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing QRL-101 in people with Amyotrophic Lateral Sclerosis, run by QurAlis Corporation at 1 site in Netherlands.
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 5 inclusion and 5 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: finished, as of the sponsor's last update on 2 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Netherlands. 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. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
QRL-101 plasma concentration (time frame: Baseline through Follow up (Day 10)). The study lists 2 primary outcomes in all. 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 2 Sep 2025; AzTrials last refreshed its copy on 1 Nov 2026.