NCT05588167 is a clinical trial in Niemann-Pick Disease, Type C, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).

What condition does NCT05588167 study?

NCT05588167 studies Niemann-Pick Disease, Type C.

Is NCT05588167 still recruiting?

NCT05588167 is currently recruiting now. Last updated 16 April 2026.

Last reviewed 2026-04-16 · How we verify

NCT05588167

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Recruiting now Last updated 16 April 2026

What this trial tests

trial in Niemann-Pick Disease, Type C in 100 participants. Currently enrolling.

Timeline

28 November 2022

Primary endpoint
30 September 2026

30 September 2026

Quick facts

Lead sponsor	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Status	Recruiting now
Study type	OBSERVATIONAL
Enrollment	100
Start date	28 November 2022
Primary completion	30 September 2026
Estimated completion	30 September 2026
Sites	1 location across United States

Conditions studied

Niemann-Pick Disease, Type C — all drugs for Niemann-Pick Disease, Type C →

Sponsor

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

Who can join

3 Months and older, any sex, with Niemann-Pick Disease, Type C. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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. ...

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

Sterol O-Acyltransferase 1 (<i>SOAT1</i>): A Genetic Modifier of Niemann-Pick Disease, Type C1.
Farhat NY, Alexander D, McKee K, Iben J, et al · · 2024 · cited 7× · PMID 38673803 · DOI 10.3390/ijms25084217

Verify or expand the search:

Other recruiting trials for Niemann-Pick Disease, Type C

Currently open trials in the same condition.

NCT00344331 — Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C · recruiting

Other Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) trials

Trials by the same sponsor.

NCT07502586 — Turner Syndrome: Genetic Considerations · recruiting
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NCT05548881 — Implications of Maternal 45,X Mosaicism as a Secondary Genomic Finding Following Cell-Free DNA Sequencing During Pregnan · withdrawn
NCT06749366 — Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT05588167 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Last refreshed: 16 April 2026

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05588167.

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