Bethesda, MDNCT03307304Now EnrollingIRB Ready

Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) Clinical Trial in Bethesda, MD

Access cutting-edge juvenile neuronal ceroid lipofuscinosis (cln3) 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

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IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related juvenile neuronal ceroid lipofuscinosis (cln3) treatment provided free

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Check if you qualify for this juvenile neuronal ceroid lipofuscinosis (cln3) clinical trial in Bethesda, MD

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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 Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) Study in Bethesda

Background: CLN3, or Batten disease, is a genetic disorder. This deadly disease leads to decline of brain and nervous system functions. Symptoms of CLN3 typically occur between 4 and 7 years of age. They include changes in how a person sees, thinks, and moves. CLN3 can also cause seizures. No effective treatments for the disease are yet known. There is limited testing of potential therapies. Researchers want to study CLN3 more so they can improve future therapies. Objective: To identify clinical or biochemical markers that can be used as therapeutic outcome measures for CLN3. Eligibility: People with CLN3. It must be based on Two CLN3 mutations OR One CLN3 mutation AND findings seen with a powerful microscope Family members of a person with CLN3. Design: Participants will have already been referred to NIH for CLN3 evaluation. If participants agree to do the study, they will: 1. give spinal fluid, blood, urine, and skin samples. They may provide other samples if they were already collected. These may include cells, surgical specimens, and DNA. 2. will be seen by multiple healthcare specialists. Participants may provide medical records or photos. Participants will sign a release of medical records form.P Researchers may send samples or clinical data to other investigators. For research testing, the samples will not include the participant s name. For a test in a clinical lab, researchers will include the participant s name. These results will become part of the clinical record at NIH.

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

Who Can Participate

Inclusion Criteria

For the Main and Sub-Studies, participants \> 1 week of age, of all genders, demographics, geographic locations, and disease severity will be recruited in order to obtain cross-sectional representation of CLN3-related conditions (Main and Sub-Studies) or all NCLs (Sub-Study B). Participants in the Main study will be followed at approximately yearly intervals to obtain longitudinal data. Participants in Sub-Study A may elect to send in medical records and samples only, or to come to the NIH for evaluations as outlined in Section 4. We anticipate that participants in Sub-Study B will be seen mostly at NCL/CLN3-related family conferences. Main Study: Individuals \> 1 week of age with a diagnosis of CLN3 or a CLN3-related/other NCL-type condition. Diagnosis determined by one of the following:
Two CLN3 or NCL condition-appropriate genetic mutations
One CLN3 mutation AND i) clinical presentation suggestive of CLN3, OR ii) characteristic electron microscopy (EM) findings (such as curvilinear body, fingerprint profile, granular osmiophilic deposits). Sub-Study A: Individuals \> 1 week of age with a diagnosis of CLN3 or CLN3-related/other NCL-type condition. Diagnosis determined by one of the following:
Two CLN3 or condition-appropriate genetic mutations
One CLN3 mutation AND i) clinical presentation suggestive of CLN3, OR ii) characteristic electron microscopy (EM) findings (such as curvilinear body, fingerprint profile, granular osmiophilic deposits). OR Individuals \> 1 month of age who have family member(s) diagnosed with CLN3 or CLN3-related/other NCL-type condition. Sub-Study B: Individuals \> 1 week of age with a clinical diagnosis of CLN3 or NCL. OR Individuals \> 1 month of age who have family member(s) diagnosed with CLN3 or NCL.

Exclusion Criteria

Main Study:
Individuals who cannot travel to the NIH because of their medical condition.
Individuals who, in the opinion of the Investigator, are unable to comply with the protocol or have medical conditions that would potentially increase the risk of participation.
Females who are pregnant. Sub-Studies A and B:
Unaffected individuals \> 18 years of age who have cognitive impairments.
Individuals who, in the opinion of the Investigator, are unable to comply with the protocol or have medical conditions that would potentially increase the risk of participation.

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 (NCT03307304) 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.

Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) Treatment Options in Bethesda, MD

If you're searching for juvenile neuronal ceroid lipofuscinosis (cln3) treatment options in Bethesda, MD, this clinical trial (NCT03307304) 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 juvenile neuronal ceroid lipofuscinosis (cln3) 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 juvenile neuronal ceroid lipofuscinosis (cln3) clinical trials near you to find additional studies recruiting in your area.

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