A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

Part of paid clinical trials in Sacramento, California.

Sponsor
argenx
Study ID
NCT06436742
Phase
PHASE1
Status
Recruiting
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Conditions

  • CMS
  • Congenital Myasthenic Syndrome

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • ARGX-119 — BIOLOGICAL
    Intravenous infusion of ARGX-119
  • Placebo — OTHER
    Intravenous infusion of placebo

Study Details

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.

Key Dates

Start date
Sep 24, 2024
Status verified
Feb 2026
Primary completion
Aug 24, 2027
Completion
Jan 24, 2028

Study Design

Enrollment
16 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Double-blinded treatment period - ARGX-119 IV
    Participants receive ARGX-119 during the double-blinded treatment period
  • Placebo Comparator: Double-blinded treatment period - Placebo IV
    Participants receive placebo during the double-blinded treatment period
  • Experimental: Active-treatment period - ARGX-119 IV
    Participants receive ARGX-119 during the active-treatment period

Primary Outcome Measure

Assessment of adverse events (AEs) [ Time Frame: Up to week 42 ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
UC Davis Medical CenterSacramentoCalifornia95817-
Ann and Robert H Lurie Childrens Hospital of ChicagoChicagoIllinois60611
Nancy Kuntz, MD
[email protected]
857-350-4834

Find similar trials in Sacramento, CA

By research site
UC Davis Medical Center· Sacramento, CAAnn and Robert H Lurie Childrens Hospital of Chicago· Chicago, IL

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