NCT01963650 is a clinical trial in Lipid Metabolism Disorders, sponsored by Shire.

Who is sponsoring NCT01963650?

NCT01963650 is sponsored by Shire.

What condition does NCT01963650 study?

NCT01963650 studies Lipid Metabolism Disorders, Metachromatic Leukodystrophy (MLD), Nervous System Diseases, Brain Diseases, Central Nervous System Diseases, Demyelinating Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Sphingolipidoses, Hereditary Central Nervous System Demyelinating Diseases, Metabolic Inborn Brain Diseases, Lysosomal Storage Diseases, Metabolic Diseases, Sulfatidosis.

Is NCT01963650 still recruiting?

NCT01963650 is currently terminated. Last updated 17 March 2021.

Last reviewed 2021-03-17 · How we verify

NCT01963650

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

Natural History Study of Children With Metachromatic Leukodystrophy

Terminated Last updated 17 March 2021

What this trial tests

trial in Lipid Metabolism Disorders in 1 participant. Terminated before completion.

Timeline

2 November 2015

Primary endpoint
8 April 2016

8 April 2016

Quick facts

Lead sponsor	Shire
Status	Terminated
Study type	OBSERVATIONAL
Enrollment	1
Start date	2 November 2015
Primary completion	8 April 2016
Estimated completion	8 April 2016
Sites	14 locations across Denmark, France, Japan, Belgium, Germany, Argentina, Canada, United States

Conditions studied

Lipid Metabolism Disorders — all drugs for Lipid Metabolism Disorders →
Metachromatic Leukodystrophy (MLD) — all drugs for Metachromatic Leukodystrophy (MLD) →
Nervous System Diseases — all drugs for Nervous System Diseases →
Brain Diseases — all drugs for Brain Diseases →

Sponsor

Shire — full company profile →

Who can join

Under 12, any sex, with Lipid Metabolism Disorders or Metachromatic Leukodystrophy (MLD). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this study is evaluate the natural course of disease progression related to gross motor function in children with metachromatic leukodystrophy (MLD).

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

Disease specific therapies in leukodystrophies and leukoencephalopathies.
Helman G, Van Haren K, Bonkowsky JL, Bernard G, et al · · 2015 · cited 40× · PMID 25684057 · DOI 10.1016/j.ymgme.2015.01.014
Emerging treatments for pediatric leukodystrophies.
Helman G, Van Haren K, Escolar ML, Vanderver A. · · 2015 · cited 14× · PMID 26022168 · DOI 10.1016/j.pcl.2015.03.006
Exploration of Gross Motor Function in Aicardi-Goutières Syndrome.
Gavazzi F, Glanzman AM, Woidill S, Formanowski B, et al · · 2023 · cited 7× · PMID 37499181 · DOI 10.1177/08830738231188753

Verify or expand the search:

Other recruiting trials for Lipid Metabolism Disorders

Currently open trials in the same condition.

NCT06896175 — Effect of Ilex Paraguariensis Harvest Time and Consumption Method of Processed Yerba Mate on the Lipid Profile · NA · recruiting
NCT06494488 — Differential Thrombogenesis by EPA and DHA Mediated by HDL · EARLY_PHASE1 · recruiting
NCT07048158 — "Perfect Heartio" Drink and Cardiovascular Health · NA · recruiting
NCT06383208 — Cardiovascular-Renal Adverse Prognosis Assessment System for Coronary Heart Disease With Chronic Kidney Disease Based on · recruiting
NCT06878781 — Postprandial Metabolome and Metabolic Flexibility · NA · recruiting

Other Shire trials

Trials by the same sponsor.

NCT05067868 — A Study of Replagal in Children and Adults With Fabry Disease in India · Phase 4 · recruiting
NCT03878953 — A Clinical Study of rhPTH(1-84) Treatment in Japanese Participants With Chronic Hypoparathyroidism · Phase 3 · withdrawn
NCT04840667 — A Study of Replagal in Treatment-naïve Adults With Fabry Disease · Phase 3 · terminated
NCT04429984 — Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India · completed
NCT04440488 — ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study · Phase 4 · withdrawn

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 NCT01963650 (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 Shire
Last refreshed: 17 March 2021

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/NCT01963650.

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