Bethesda, MDNCT05588167Now EnrollingIRB Ready

Niemann-Pick Disease, Type C Clinical Trial in Bethesda, MD

Access cutting-edge niemann-pick disease, type c treatment through this clinical trial at a research site in Bethesda. Study-provided care at no cost to qualified participants.

Sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

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Expert Care in Bethesda

Access niemann-pick disease, type c specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related niemann-pick disease, type c treatment provided free

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Why Participate?

  • No-Cost Study Care

  • Local to Bethesda

    Convenient for MD 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 Bethesda site if eligible
  4. 4Begin participation

About This Niemann-Pick Disease, Type C Study in Bethesda

Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other parts of the body. There is no cure for NPC, and symptoms only get worse over time. Symptoms can include seizures, difficulty moving or talking, or dementia. But symptoms can vary among different people with the disease. Some may have seizures, while others do not, for example. Some people begin showing symptoms in childhood; in others, symptoms may not appear until they are adults. Researchers want to learn more about why NPC affects people differently. This natural history study will gather data from people with NPC in order to understand more about the disease and how it affects the body. Objective: This study will create the first and largest database about NPC. Eligibility: People of any age who have NPC. Design: Participants will have blood drawn from a vein. This will happen only once. The blood will be used to analyze the participants DNA. The participants medical records will be reviewed. The study team will collect data on participants NPC diagnosis and symptoms; they will record how long participants have had each symptom. The study team will also collect data on each participants age, sex, race, height, weight, medications, and other test results. The study team will communicate with participants. They will discuss the study and answer any questions. Participants will receive up to $190. ...

Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Who Can Participate

Inclusion Criteria

; In order to be eligible to participate in this study, an individual must meet all of the following criteria:
Provision of signed and dated informed consent form
Stated willingness to comply with all study procedures and availability for the duration of the study
Male or female, any age, demographic or ethnic background will be eligible for this study
Diagnosis of NPC will be based on clinical, biochemical or molecular testing.

Exclusion Criteria

An individual who meets any of the following criteria will be excluded from participation in this study:
Unwilling to provide consent
Unable to provide biospecimen to obtain DNA
Unable to provide medical records or clinical data

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 Bethesda?

Yes, this clinical trial (NCT05588167) has an active research site in Bethesda, MD 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.

Niemann-Pick Disease, Type C Treatment Options in Bethesda, MD

If you're searching for niemann-pick disease, type c treatment options in Bethesda, MD, this clinical trial (NCT05588167) 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 Bethesda research site is actively enrolling participants for this clinical trial. You'll receive care from experienced niemann-pick disease, type c 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 niemann-pick disease, type c clinical trials near you to find additional studies recruiting in your area.

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