NCT00001491 is a clinical trial in Fabry's Disease, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).

Who is sponsoring NCT00001491?

NCT00001491 is sponsored by National Institute of Neurological Disorders and Stroke (NINDS).

What condition does NCT00001491 study?

NCT00001491 studies Fabry's Disease.

Is NCT00001491 still recruiting?

NCT00001491 is currently completed. Last updated 2 July 2017.

Last reviewed 2017-07-02 · How we verify

NCT00001491

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

Analysis of the Nervous System in Patients With Fabry's Disease

Completed Last updated 2 July 2017

What this trial tests

trial in Fabry's Disease in 325 participants. Completed in 3 March 2008.

Timeline

11 May 1995

3 March 2008

Quick facts

Lead sponsor	National Institute of Neurological Disorders and Stroke (NINDS)
Status	Completed
Study type	OBSERVATIONAL
Enrollment	325
Start date	11 May 1995
Estimated completion	3 March 2008
Sites	1 location across United States

Conditions studied

Fabry's Disease — all drugs for Fabry's Disease →

Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Who can join

Eligibility, any sex, with Fabry's Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Fabry's disease a genetic disorder (X-linked recessive) due to the absence of the enzyme ceramidetrihexosidase. The disease is characterized by abnormal collections of glycolipids in cells (histiocytes) within blood vessel walls, tumors on the thighs, buttocks, and genitalia, decreased sweating, tingling sensations in the extremities, and cataracts. Patients with Fabry 's disease die from complications of the kidney, heart, or brain. The purpose of this study is to measure levels of a protein marker (PGP 9.5) in the skin, blood, and fluid surrounding the brain and spinal cord (CSF) in patients with Fabry's disease. In addition the study will attempt to determine if levels of the protein are directly related to the severity of disease in the nervous system. PGP 9.5 protein levels will be measured in normal volunteers and patients with other diseases of the nervous system then compared to the levels recorded in patients with Fabry's disease. This research study is designed to improve the understanding of Fabry's disease. Patients participating in it will not directly benefit from it. However, knowledge gained as a result of this study may contribute to the development of effective therapies for Fabry's disease.

Publications & conference data

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

Vitamin C Urinary Loss in Fabry Disease: Clinical and Genomic Characteristics of Vitamin C Renal Leak.
Ebenuwa I, Violet PC, Padayatty SJ, Wang Y, et al · · 2023 · cited 6× · PMID 37229630 · DOI 10.1016/j.tjnut.2022.12.009

Verify or expand the search:

Other National Institute of Neurological Disorders and Stroke (NINDS) trials

Trials by the same sponsor.

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NCT06615973 — Screening for Social Determinants of Health (SDOH) and Cognitive Function in Individuals With History of Stroke · 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 NCT00001491 (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 National Institute of Neurological Disorders and Stroke (NINDS)
Last refreshed: 2 July 2017

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/NCT00001491.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing