A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants with DOK7-Congenital Myasthenic Syndromes (CMS)
- Conditions
- Congenital Myasthenic Syndrome
- Interventions
- Other: Placebo
- Registration Number
- NCT06436742
- Lead Sponsor
- argenx
- Brief 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 treatment period. Participants will then enter the follow-up period. The full duration of the study is approximately 11 months.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 16
- At least 18 years of age.
- Has genetically confirmed congenital myasthenic syndromes due to mutation of downstream of kinase 7 (DOK7-CMS).
- Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) must have been receiving the medication for more than 3 months and agree to remain on a same stable dosing regimen of the same medication until the end of the study.
- Diagnosis of CMS due to mutation of any gene other than DOK7.
- Known medical condition that would interfere with an accurate assessment of CMS, confound the results of the study, or put the patient at undue risk, as assessed by the investigator.
- History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for more than 5 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer.
- Different study drug received in another clinical study within 12 weeks or 5 half-lives before screening.
- Current participation in another interventional clinical study or prior participation in any gene therapy or cell therapy study.
- Pregnant or lactating state or intention to become pregnant during the study.
The complete list of exclusion criteria can be found in the protocol.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Participants receiving intravenous infusion of placebo once every other week ARGX-119 ARGX-119 Participants receiving intravenous infusion of ARGX-119 once every other week
- Primary Outcome Measures
Name Time Method Assessment of adverse events (AEs) Up to week 42
- Secondary Outcome Measures
Name Time Method Maximum observed serum concentration (Cmax) of ARGX-119 Up to week 42 Incidence of anti-drug antibodies (ADA) against ARGX-119 Up to week 42 Prevalence of anti-drug antibodies (ADA) against ARGX-119 Up to week 42 Change from baseline over time for key components of the Quantitative Myasthenia Gravis (QMG) scale Up to week 42 Minimum value: 0 (no disease severity); Maximum value: 39 (highest disease severity)
Change from baseline over time for Myasthenia Gravis Activities of Daily Living (MG-ADL) Up to week 42 Minimum value: 0 (normal symptoms); Maximum value: 24 (most severe symptoms)
Change from baseline over time for Patient-Reported Outcomes Measurement Information System Global Health (PROMIS-GH) scale Up to week 42 The participant records their response to each question on a 5-point Likert scale, with lower scores indicating poorer health (Minimum value: 0, Maximum value: 20)
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Trial Locations
- Locations (8)
UC Davis Medical Center
🇺🇸Sacramento, California, United States
Ann and Robert H Lurie Childrens Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Ottawa Hospital Research Institute - Civic Campus
🇨🇦Ottawa, Canada
CHU - Hospital de la Timone
🇫🇷Marseille, France
Group Hospitalier Pitie-Salpetriere
🇫🇷Paris, France
Fondazione IRCCS Istituto Neurologico Carlo Besta
🇮🇹Milan, Italy
Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur)
🇪🇸Valencia, Spain
John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust
🇬🇧Oxford, United Kingdom