Amyotrophic Lateral SclerosisPhase 2ReALiSeNCT06441682
A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing ARGX-119 in people with Amyotrophic Lateral Sclerosis, run by argenx at 9 sites in 6 countries.
Trial summary
This study aims to evaluate the safety of ARGX-119 in adults with ALS. The study will also assess the impact of ARGX-119 on ALS disease outcomes, including muscle function. The study consists of 2 periods: a treatment period when participants will receive one of three ARGX-119 doses or placebo and an extension period… 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?
- ARGX-119
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 9 sitesin 6 countries
- Study dates
- 23 Oct 2024to 12 Aug 2026
- Phase
- Phase 2
- 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
- The participant is at least 18 and ≤80 years of age
- The participant is diagnosed with familial or sporadic ALS according to Gold Coast criteria
- The participant has a Treatment Research Initiative to Cure ALS (TRICALS) risk profile of ≥ -6.0 to < -2.0
- Slow vital capacity (SVC) of ≥ 60% of the predicted value according to Global Lung Function Initiative 2012
Who cannot join
- Use of noninvasive ventilation more than 10 hours a day or use of a tracheostomy for ventilatory support
- Any history of or current exposure to any gene or cell therapies (off-label use or investigational) for ALS
- Pregnant or lactating state or intention to become pregnant during the study
Trial locations and contacts
9 sites in 6 countries.
9 sites in 6 countries
Canada2 sites
Kaye Edmonton Clinic
Edmonton, 11400
Montreal Neurological Institute and Hospital
Montreal, H3A 2B4
Denmark2 sites
Aarhus Universitets Hospital
Aarhus, 8200
Bispebjerg University Hospital
Copenhagen, 2400
France2 sites
Hôpital La Pitié Salpêtrière
Paris, 75013
CHU Bretonneau
Tours, 37000
Sweden1 site
Akademiskt specialistcentrum Karolinska Institutet
Stockholm, 113 61
About this study
Official title: A Phase 2a, Double-Blinded, Randomized, Placebo-Controlled, and Active-Treatment Extension Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of ARGX-119 in Participants With Amyotrophic Lateral Sclerosis
This study aims to evaluate the safety of ARGX-119 in adults with ALS. The study will also assess the impact of ARGX-119 on ALS disease outcomes, including muscle function. The study consists of 2 periods: a treatment period when participants will receive one of three ARGX-119 doses or placebo and an extension period when all participants will receive the same dose of ARGX-119. Participation in the study will last up to approximately 100 weeks.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- ARGX-119, 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.
ARGX-119Biological
Intravenous infusion of ARGX-119
PlaceboOther
Intravenous infusion of placebo
| Study group | Role | What they receive |
|---|---|---|
| ARGX-119 - Dose 1 | Experimental | Participants will receive first dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period Receives: Biological: ARGX-119 |
| ARGX-119 - Dose 2 | Experimental | Participants will receive second dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period Receives: Biological: ARGX-119 |
| ARGX-119 - Dose 3 | Experimental | Participants will receive third dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period Receives: Biological: ARGX-119 |
| Placebo | Placebo comparator | Participants will receive placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period Receives: Biological: ARGX-119; Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of adverse events (AEs)
Measured: Up to week 96
Secondary outcomes (4)
Rate of change from baseline in electrophysiological muscle scan (MScan)-derived motor unit number (MUN)
Measured: Up to week 24
Maximum observed serum concentration (Cmax) of ARGX-119
Measured: Up to week 96
Incidence of anti-drug antibodies (ADA) against ARGX-119 in serum over time
Measured: Up to week 96
Prevalence of anti-drug antibodies (ADA) against ARGX-119 in serum over time
Measured: Up to week 96
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2024
- Study start23 Oct 2024
- Primary completion12 Aug 2026
- Study completion12 Aug 2026
Why it stopped: The study was terminated following review of interim data indicating that continuation was unlikely to demonstrate a clinically meaningful treatment effect. The decision was made to minimize unnecessary participant burden.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by argenxThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06441682
- Sponsor's protocol ID
- ARGX-119-2303
- Other IDs
- 2024-511318-19-00
- Acronym
- ReALiSe
- Status last verified
- Sep 2026
- First submitted
- 29 May 2024
- Last updated
- 15 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Moss KR, Darvishi FB, Badawi Y, Fish LA, Funke JR, Pedersen TH, Robitaille R, Arnold WD, Burgess RW, Meriney SD, Nishimune H, Saxena S. The Neuromuscular Junction: A Shared Vulnerability in Aging and Disease. J Neurosci. 2025 Nov 12;45(46):e1353252025. doi: 10.1523/JNEUROSCI.1353-25.2025.
PubMed 41224659
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Double-Blinded, Randomized, Placebo-Controlled, and Active-Treatment Extension Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of ARGX-119 in Participants With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT06441682
- Acronym / sponsor ID
- ReALiSe / ARGX-119-2303
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated following review of interim data indicating that continuation was unlikely to demonstrate a clinically meaningful treatment effect. The decision was made to minimize unnecessary participant burden.
- Enrolment
- 60 participants (actual)
- Lead sponsor
- argenxIndustry (pharma, biotech, devices)
- Study start
- 23 Oct 2024 (actual)
- Primary completion
- 12 Aug 2026 (actual)
- Study completion
- 12 Aug 2026 (actual)
- First posted
- 4 Jun 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 study aims to evaluate the safety of ARGX-119 in adults with ALS. The study will also assess the impact of ARGX-119 on ALS disease outcomes, including muscle function. The study consists of 2 periods: a treatment period when participants will receive one of three ARGX-119 doses or placebo and an extension period when all participants will receive the same dose of ARGX-119. Participation in the study will last up to approximately 100 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| ARGX-119 - Dose 1 | Experimental | Biological: ARGX-119Participants will receive first dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period |
| ARGX-119 - Dose 2 | Experimental | Biological: ARGX-119Participants will receive second dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period |
| ARGX-119 - Dose 3 | Experimental | Biological: ARGX-119Participants will receive third dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period |
| Placebo | Placebo comparator | Biological: ARGX-119, Other: PlaceboParticipants will receive placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- The participant is at least 18 and ≤80 years of age
- The participant is diagnosed with familial or sporadic ALS according to Gold Coast criteria
- The participant has a Treatment Research Initiative to Cure ALS (TRICALS) risk profile of ≥ -6.0 to < -2.0
- Slow vital capacity (SVC) of ≥ 60% of the predicted value according to Global Lung Function Initiative 2012
Key exclusion criteria (3 listed)
- Use of noninvasive ventilation more than 10 hours a day or use of a tracheostomy for ventilatory support
- Any history of or current exposure to any gene or cell therapies (off-label use or investigational) for ALS
- Pregnant or lactating state or intention to become pregnant during the study
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 |
|---|---|---|
| ARGX-119Intravenous infusion of ARGX-119 | Biological | ARGX-119 - Dose 1, ARGX-119 - Dose 2, ARGX-119 - Dose 3, Placebo |
| PlaceboIntravenous infusion of placebo | Other | Placebo |
Primary outcome
Assessment of adverse events (AEs)
Time frame: Up to week 96
4 secondary outcomes
Rate of change from baseline in electrophysiological muscle scan (MScan)-derived motor unit number (MUN)
Time frame: Up to week 24
Maximum observed serum concentration (Cmax) of ARGX-119
Time frame: Up to week 96
Incidence of anti-drug antibodies (ADA) against ARGX-119 in serum over time
Time frame: Up to week 96
Prevalence of anti-drug antibodies (ADA) against ARGX-119 in serum over time
Time frame: Up to week 96
Results and safety
The study was stopped early (reason given: The study was terminated following review of interim data indicating that continuation was unlikely to demonstrate a clinically meaningful treatment effect. The decision was made to minimize unnecessary participant burden.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2026.
- Derived from this study: PubMed 41224659Moss KR, Darvishi FB, Badawi Y, Fish LA, Funke JR, Pedersen TH, Robitaille R, Arnold WD, Burgess RW, Meriney SD, Nishimune H, Saxena S. The Neuromuscular Junction: A Shared Vulnerability in Aging and Disease. J Neurosci. 2025 Nov 12;45(46):e1353252025. doi: 10.1523/JNEUROSCI.1353-25.2025.
- Individual participant dataSharing planned: No
Related trials for Amyotrophic Lateral Sclerosis
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Clinical Outcome Assessment for AT & BCI
Spinal Cord Injury, ALS (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.
9 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ARGX-119 in people with Amyotrophic Lateral Sclerosis, run by argenx at 9 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 4 inclusion and 3 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: stopped early, as of the sponsor's last update on 15 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Canada, Denmark, France, Belgium, Netherlands 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was stopped early (reason given: The study was terminated following review of interim data indicating that continuation was unlikely to demonstrate a clinically meaningful treatment effect. The decision was made to minimize unnecessary participant burden.). Results are not available in the source record.
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?
Assessment of adverse events (AEs) (time frame: Up to week 96). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06441682, which also lists 1 linked publication.
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 Sep 2026.