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Trial Title:
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants with DOK7-Congenital Myasthenic Syndromes (CMS)
NCT ID:
NCT06436742
Condition:
Congenital Myasthenic Syndrome
Conditions: Official terms:
Lambert-Eaton Myasthenic Syndrome
Myasthenic Syndromes, Congenital
Syndrome
Study type:
Interventional
Study phase:
Phase 1
Overall status:
Recruiting
Study design:
Allocation:
Randomized
Intervention model:
Parallel Assignment
Primary purpose:
Treatment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Intervention:
Intervention type:
Biological
Intervention name:
ARGX-119
Description:
Intravenous infusion of ARGX-119
Arm group label:
ARGX-119
Intervention type:
Other
Intervention name:
Placebo
Description:
Intravenous infusion of placebo
Arm group label:
Placebo
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.
Criteria for 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 months and agree to remain on a
same stable dosing regimen of the same medication until the end of the study.
Exclusion Criteria:
- 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.
Gender:
All
Minimum age:
18 Years
Maximum age:
N/A
Healthy volunteers:
No
Locations:
Facility:
Name:
Ottawa Hospital Research Institute - Civic Campus
Address:
City:
Ottawa
Zip:
K1Y 4E9
Country:
Canada
Status:
Recruiting
Contact:
Last name:
Hanns Lochmuller, MD
Phone:
857-350-4834
Email:
clinicaltrials@argenx.com
Facility:
Name:
Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur)
Address:
City:
Valencia
Zip:
46026
Country:
Spain
Status:
Recruiting
Contact:
Last name:
Nuria Muelas Gomez, MD
Phone:
857-350-4834
Email:
clinicaltrials@argenx.com
Start date:
September 24, 2024
Completion date:
October 2025
Lead sponsor:
Agency:
argenx
Agency class:
Industry
Source:
argenx
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06436742
