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(904) 244-0411

Phase 3, Double blind, Randomized, Placebo controlled, Multicenter Study for the Safety, Tolerability, Efficacy of Trappsol® Cyclo™ vs. Placebo in Niemann Pick Disease

  • Status
    Accepting Candidates
  • Age
    4 Years - 100 Years
  • Sexes
    All
  • Healthy Volunteers
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Description

The main purpose of this study is to investigate the effect of the study drug Trappsol Cyclo and the current standard-of-care, compared to placebo and the current standard of care, on the disease progression and symptoms of a rare genetic disorder called Niemann Pick Disease Type C1 (NPC). Those with this condition can experience many symptoms related to a decline in cognitive (thinking) and motor (movement) function, as well as difficulty swallowing, sleep disturbances, and pneumonia. The study drug is provided by the Sponsor. Total participation in this study will be up to 100 weeks (23 months). The patient will need to visit the clinic at least 52 times and will receive treatment every 2 weeks. Patients will have blood work, exams, EKGs, lung function tests, hearing tests, questionnaires, swallowing tests, and urine tests. It is open for patients over 3 years of age.

Details

Protocol number CTD-TCNPC-301

Eligibility

Patients who have NPC with at least one neurological symptom.

Lead researchers

Participate in a study

Here are some general steps to consider when participating in a research study:

  1. Step
    1

    Contact the research team

    Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.

    Primary contact

  2. Step
    2

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step
    3

    Provide your consent to participate

    If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.

  4. Step
    4

    Participate

    If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.