A Phase 2 interventional study of ARGX-119 IV and Placebo IV in Spinal Muscular Atrophy (SMA), sponsored by argenx. Recruiting at 28 sites in 4 countries. Open to participants aged 5 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-10-08.
Sponsored by argenx · Phase 2, Interventional, and Treatment
Among 121 studies in Muscular Atrophy, Spinal with a site in United States — 21 open to participants now, 39 under way, 7 of them phase 3. Muscular Atrophy, Spinal · United States.
Among the 121 studies in Muscular Atrophy, Spinal with a site in United States, this one is the 21st largest by planned enrollment of the 76 interventional ones that give a count, is one of 8 that started in 2025, is one of 18 in phase 2, reaches primary completion later than 26 of the 38 other live ones, and was the 112th registered.
282 studies on the registry are indexed under Muscular Atrophy, Spinal; 73 are open to participants now.
Browse Muscular Atrophy, Spinal studies →21 of the 121 studies in Muscular Atrophy, Spinal with a site in United States are open now. Recruiting first, the newest first:
Johns Hopkins University · Sites in United States
Ages 18–80
St. Jude Children's Research Hospital · Sites in United States
Biogen · Sites in United States
Ages 0 days–7 mo
Biogen · Sites in United States, China
Ages 15–60
Novartis Pharmaceuticals · Sites in United States
Ages 2–100
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 58 Muscular Atrophy, Spinal studies recruiting now, 28 (48%) set no upper age limit and 27 (47%) stop at 75 or younger; 46 take children under 18; 11 take healthy volunteers.
This study: 5 Years to 17 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
121 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 18 · Phase 2 27 · Phase 3 20 · Phase 4 6
First in 1986; this study’s year is marked. 8 started in 2025.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 60 is above the median of 29 across 156 interventional studies indexed under Muscular Atrophy, Spinal.
Median enrollment 33 across the 76 interventional studies, 75 across the 43 observational. Of the 76 that completed or were terminated, 33 (43%) have results posted.
This tab as a report to print or save as PDF → With an account.
61 of 121 have an industry lead sponsor. The 10 most frequent:
argenx is the lead sponsor of 87 studies on the registry; 29 are open to participants now.
Of its 22 completed or terminated interventional studies of FDA-regulated products, 16 (73%) have results posted.
argenx has 1 study filed under Muscular Atrophy, Spinal — fewer than ten, so there is no comparison to draw.
7 of the studies in Muscular Atrophy, Spinal with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05067790Phase 3BiogenA Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) | 2027-09-24 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
| NCT07223320Teachers College, Columbia UniversityHABIT-ILE + FST in Children With SMA | 2026-12-31 |
| NCT07400198Jacqueline MontesGait and Bone Health in SMA | 2027-06-30 |
| NCT06532474St. Jude Children's Research HospitalExploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies | 2027-09 |
Add all 7 to your calendar (.ics) → With an account.
Among the 121 studies in Muscular Atrophy, Spinal with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it moves through the body, and how the immune system responds to it. The study consists of a double-blinded treatment period (DBTP) where participants will either receive ARGX-119 IV or placebo IV, in addition to disease-modifying therapy (DMT) for 24 weeks. Participants who complete the DBTP will enter the open-label active-treatment extension period (ATEP) during which all participants will receive ARGX-119 IV up to 100 weeks (approximately 2 years).
This phase 2 study aims to establish proof of concept with the age-appropriate dose of ARGX-119 in ambulant pediatric patients with spinal muscular atrophy (SMA). Despite available treatments, there remains an unmet medical need for patients with SMA. Neuromuscular junction (NMJ) dysfunction contributes to the pathophysiology of SMA, including muscle weakness and fatigability. Activation of muscle-specific kinase (MuSK) by ARGX-119 may stabilize and improve NMJ function in patients with SMA, reducing muscle weakness and fatigability, and improving quality of life.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants receive ARGX-119 IV during the DBTP
Biological: ARGX-119 IV
Participants receive placebo IV during the DBTP
Other: Placebo IV
Participants receive ARGX-119 IV during the ATEP. Participants from ARGX-119 IV arm in the DBTP will receive placebo once to maintain the DBTP blinding
Biological: ARGX-119 IV · Other: Placebo IV
Drug pages: Adimanebart · Anti-MuSK Agonistic Monoclonal Antibody ARGX-119
Intravenous infusion of ARGX-119
Intravenous infusion of placebo
Incidence of AEs
Adverse Events
Time frame: Up to 124 weeks
Incidence of SAEs
Serious Adverse Events
Time frame: Up to 124 weeks
Change in RHS total score from baseline to week 24 of the double blinded treatment period (DBTP)
The RHS (Revised Hammersmith Scale) is a validated 36-item scale developed to evaluate the spectrum of gross motor function. Maximum total score; 69 (optimal motor function)
Time frame: Up to 24 weeks
Change from baseline over time for the 6MWT - distance and fatigue index
In the 6MWT (6-minute walk test) participants will be instructed to walk as fast as possible without jogging or running along a 25- meter linearly marked course for 6 minutes without using walking devices or assistance. Distance walked over the entire 6-minute period, and other parameters, will be determined
Time frame: Up to 124 weeks
ARGX-119 serum concentrations over time
ARGX-119 serum concentrations over time
Time frame: Up to 124 weeks
Incidence of antidrug antibodies (ADA) against ARGX-119
Incidence of antidrug antibodies (ADA) against ARGX-119
Time frame: Up to 124 weeks
Not enrolling at this site — choose another above.
Plan to share: No
No publications or documents are linked to this record.
Started Dec 2025 (actual). Primary completion is estimated for May 2029, 41 months after the start.
Of 15 phase 2 interventional studies under Muscular Atrophy, Spinal, half reached primary completion within 34 months of starting (the median), counting those still running; a quarter within 25 and three quarters within 42. This study's planned 41 months is above that.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drugs tested here: Adimanebart · Anti-MuSK Agonistic Monoclonal Antibody ARGX-119