Sacramento, CANCT06436742Now EnrollingIRB Ready

Congenital Myasthenic Syndrome Clinical Trial in Sacramento, CA

Access cutting-edge congenital myasthenic syndrome treatment through this clinical trial at a research site in Sacramento. Study-provided care at no cost to qualified participants.

Sponsored by argenx

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Expert Care in Sacramento

Access congenital myasthenic syndrome specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related congenital myasthenic syndrome treatment provided free

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Check if you qualify for this congenital myasthenic syndrome clinical trial in Sacramento, CA

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Why Participate?

  • No-Cost Study Care

  • Local to Sacramento

    Convenient for CA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Sacramento site if eligible
  4. 4Begin participation

About This Congenital Myasthenic Syndrome Study in Sacramento

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.

Sponsor: argenx

Who Can Participate

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.
Pregnant or lactating state or intention to become pregnant during the study.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Sacramento?

Yes, this clinical trial (NCT06436742) has an active research site in Sacramento, CA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Congenital Myasthenic Syndrome Treatment Options in Sacramento, CA

If you're searching for congenital myasthenic syndrome treatment options in Sacramento, CA, this clinical trial (NCT06436742) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Sacramento research site is actively enrolling participants for this clinical trial. You'll receive care from experienced congenital myasthenic syndrome specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all congenital myasthenic syndrome clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Sacramento, CA