Condition
Niemann-Pick Disease, Type C clinical trials and research
As of November 12, 2026, Find a Trial lists 3 Niemann-Pick Disease, Type C clinical trials that are recruiting, at sites in 8 countries (most in United States (3), Australia (1), Czechia (1)). By phase: Phase 3 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 3Clinical trials
- 8Countries
Newest Niemann-Pick Disease, Type C clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Creating a database for Niemann-Pick Disease, Type C | — | Bethesda, Maryland | Recruiting | Oct 20, 2022 |
| N-acetyl-L-leucine for Niemann-Pick type C | Phase 3 | Rochester, Minnesota and 13 other locations | Recruiting | Dec 20, 2021 |
| Evaluation of markers in Niemann-Pick Disease, Type C | — | Bethesda, Maryland | Recruiting | Jun 26, 2006 |
All recruiting trials (3)
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Creating a database for Niemann-Pick Disease, Type C
This study is collecting blood samples from people with Niemann-Pick Disease, Type C to build a database that helps researchers understand the disease better and find new treatment options.
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N-acetyl-L-leucine for Niemann-Pick type C
This trial tests whether N-acetyl-L-leucine helps people aged 4 and older with Niemann-Pick type C by comparing the medicine to a placebo in a randomized, double-blind crossover design.
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Evaluation of markers in Niemann-Pick Disease, Type C
This study is trying to find signs in the body that show how Niemann-Pick Disease, Type C is getting worse, to help track progress and see how well new treatments might work for people with this condition.
Quick answers
How many Niemann-Pick Disease, Type C clinical trials are recruiting right now?
3, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 8 countries. The most sites are in United States (3 trials), Australia (1 trial), Czechia (1 trial), Germany (1 trial), Netherlands (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 3: 1. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.