Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT06436742 · ClinicalTrials.gov registry record · Phase 1
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
A Phase 1 study of Congenital Myasthenic Syndrome and CMS, sponsored by argenx.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 16
- Enrollment target
- 9
- Study locations
NCT06436742: Recruiting Phase 1 study of Congenital Myasthenic Syndrome and CMS, sponsored by argenx.
NCT06436742 is a Phase 1 study of Congenital Myasthenic Syndrome and CMS that is actively recruiting participants, run by argenx. The registered enrollment target is 16 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (73% lower). The trial reports 9 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06436742, a Phase 1 study of Congenital Myasthenic Syndrome and CMS, is actively recruiting participants, sponsored by argenx.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 16 participants
- Enrollment target
- 9
- Study locations
Study Summary
The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will also assess how ARGX-119 is processed by the body (pharmacokinetics), how the immune system reacts to it (immunogenicity), and how it may improve the way patients feel and function. After the screening period, eligible participants will be randomized in a 4:1 ratio to receive intravenous infusions of ARGX-119 or placebo during the double-blinded treatment period. Participants will then enter the follow-up period. After the follow-up period, participants may enrol in the active-treatment period, where they will receive open-label ARGX-119. The full duration of the study is approximately 38 months.
Conditions Studied
Interventions
- OTHER Placebo
- BIOLOGICAL ARGX-119
Study Locations (9)
Other
- Ottawa Hospital Research Institute - Civic Campus - Ottawa
- CHU - Hospital de la Timone - Marseille
- Group Hospitalier Pitie-Salpetriere - Paris
- Fondazione IRCCS Istituto Neurologico Carlo Besta - Milan
- Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur) - Valencia
- Clinical Trials Centre - South Eastern Health and Social Care Trust - The Ulster Hospital - Belfast
- John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust - Oxford
California
- UC Davis Medical Center - Sacramento
Illinois
- Ann and Robert H Lurie Childrens Hospital of Chicago - Chicago
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2024-09-24 |
| Est. Completion | 2028-01-24 |
| Phase | Phase 1 |
What NCT06436742 shows while recruiting
NCT06436742 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (73% lower).
The record links to 2 conditions, with Congenital Myasthenic Syndrome appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT06436742 lists 9 locations in 3 states (Other, California, Illinois).
Frequently Asked Questions
What is clinical trial NCT06436742 about?
NCT06436742 is a clinical study titled "A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)". The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will also assess how ARGX-119 is processed by the body (pharmacokinetics), how the immune system reacts to it (immunogenicity), and how it may im...
What is the current status of trial NCT06436742?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 16 participants. The study started on 2024-09-24. Estimated completion is 2028-01-24.
What conditions does trial NCT06436742 study?
This clinical trial studies the following conditions: Congenital Myasthenic Syndrome, CMS.
What interventions are being tested in trial NCT06436742?
The interventions under investigation include: Placebo (OTHER), ARGX-119 (BIOLOGICAL).
Who is sponsoring clinical trial NCT06436742?
This trial is sponsored by argenx, which has 49 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06436742 being conducted?
This trial has 9 study locations across California, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Congenital Myasthenic Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06436742's enrollment target sits among peer trials
16 1787th of 2000 higher than 203 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02422641 · 16 participants · Phase 2
Prospective Evaluation Of High-Dose Systemic Methotrexate In Patients With Breast Cancer And Leptomeningeal Metastasis
- NCT02851758 · 16 participants · NA
Transplantation of Autologously Derived Mitochondria Following Ischemia
- NCT03291028 · 16 participants
Analysis of Primary and Metastatic Tumors in Patients With Renal Cell Carcinoma and Urothelial Carcinoma
- NCT03425526 · 16 participants · Phase 1
Donor T Cell Therapy in Treating Immunocompromised Patients With Adenovirus-Related Disease
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI