Patient Guide: A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

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✓ Get matched to the right study location for you
✓ Connect directly with study coordinators at 14 participating sites
✓ Understand eligibility with our simplified screening tool
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Trial ID: NCTNCT05163288

Status: 🟢 Enrolling Now

Condition: Niemann-Pick Disease, Type C

Phase: PHASE3

Find a Study Location Near You

This study is available at 14 research sites. We'll help you connect with the location that's right for you.

Participating sites include:

• Rochester, Minnesota
• Parkville, Victoria
• Praha,
• And 11 more locations - let us help you find the closest one

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A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C - Join Clinical Trial NCTNCT05163288

How to Join This Clinical Trial - NCTNCT05163288

Learn how to participate in this PHASE3 trial studying an investigational therapy for Niemann-Pick Disease, Type C. This study is currently enrolling participants.

Am I Eligible for This Clinical Trial?

This clinical research study is looking for participants with Niemann-Pick Disease, Type C. Eligibility requirements include specific age ranges, health status, and medical history. Contact the study team to learn if you qualify.

Condition Being Studied: Niemann-Pick Disease, Type C
Treatment Being Tested: Investigational treatment
Study Phase: PHASE3 - Large-scale efficacy study
Enrollment Status: Currently enrolling participants
Study Identifier: NCTNCT05163288 - ClinicalTrials.gov Identifier
Sponsored By: Study sponsor

What to Expect as a Participant

Participating in this clinical trial involves:

Initial screening to determine eligibility
Regular study visits and health assessments
Receiving the study treatment or placebo
Medical monitoring and follow-up care
Contributing to medical research that may help others
Potential access to new treatments before they're widely available

Clinical Trial Benefits and Compensation

Participants in this clinical research study may receive:

Close medical monitoring by healthcare professionals
Access to potential new treatments
Compensation for time and travel (varies by study)
No-cost study-related medical care
The opportunity to help advance medical knowledge

Bratislava, 833 40 - Slovakia

Status: ACTIVE_NOT_RECRUITING

And 4 more locations available. Contact us to find the nearest participating site.

How to Enroll in This Study

To learn more about participating in this PHASE3 clinical trial for Niemann-Pick Disease, Type C:

Review the eligibility criteria with your healthcare provider
Contact the study team for a pre-screening interview
Schedule an in-person screening visit if eligible
Review and sign the informed consent form
Begin participation in the clinical trial

Why Choose Quri.ai as Your Clinical Trial Matching Service

Quri.ai is a free clinical trial matching service that helps patients connect with research sites. We make it easier to:

Find clinical trials that match your condition
Get matched to the right study location near you
Connect directly with study coordinators
Understand if you qualify with simplified eligibility screening
Navigate the enrollment process with personalized support
Access new treatments through clinical research participation