A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
Recruiting
Phase 1
Interventional Study
Congenital Myasthenic Syndrome
CMS
At a Glance
- Age
- 18 and older
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 16 (estimated)
- Sponsor
- argenx · Industry
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About This Trial
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…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* 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 m…
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