- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00316498
Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease
A Phase I/II Randomized, Controlled Study of OGT 918 in Adult and Juvenile Patients With Niemann Pick C Disease
This study is done in conjunction with a trial, conducted at Columbia University College of Physicians and Surgeons in New York and the Royal Manchester Children's Hospital in England, to examine the effectiveness of a new drug called OGT 918 for treating Niemann-Pick Type C (NPC) disease. Patients with this genetic disorder do not transport lipids (fatty substances) in their cells, resulting in problems of the liver, spleen and brain. An early sign of NPC is a reduced ability to move the eyes rapidly up and down or from side to side. These voluntary eye movements are called saccades. Patients in the OGT 918 trial who participate in this sub-study will have their saccadic eye movements measured to see if improvement occurs with OGT 918 treatment.
Patients with Niemann-Pick Type C disease 12 years of age and older who are enrolled in the OGT 918 trial described above may be eligible for this study.
Participants will have both vertical (up and down) and horizontal (side to side) saccadic eye movements measured at two time points before starting treatment with OGT 918 and after 12 months of treatment. For the test, patients sit in a chair with their head positioned as for a regular eye examination (steadied by a chin cup and headrest) and follow with their eyes a series of lights or laser spots moving on a screen at a distance of 1 meter (3 feet). During the test, patients wear either special recording glasses, infrared goggles, or special contact lenses for measuring eye movements. A full eye evaluation lasts about 1 hour, and each eye is evaluated twice. The evaluations are separated in time by at least an hour, and possibly a day.
Study Overview
Detailed Description
Study Type
Enrollment
Phase
- Phase 1
Contacts and Locations
Study Locations
-
-
-
Abingdon, United Kingdom
- Oxford GlycoSciences Ltd.
-
-
-
-
Maryland
-
Bethesda, Maryland, United States, 20892
- National Institutes of Health Clinical Center, 9000 Rockville Pike
-
-
New York
-
New York, New York, United States, 10032-3784
- Columbia University
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
- INCLUSION CRITERIA:
Patients with NPC disease confirmed by abnormal cholesterol esterification and abnormal filipin staining.
Patients aged 12 and over. Body weight must not be less than 14 kg.
Patients who can ingest a capsule.
EXCLUSION CRITERIA:
Patients younger than 18 who are unable to give informed assent and/or whose legal guardian is unable to provide informed consent.
Patients aged 18 and over who cannot provide informed consent and/or whose legal guardian is unable to provide witnessed informed consent.
Fertile patients who do not agree to use adequate contraception throughout the study and for three months after cessation of OGT 918 treatment.
Patients who cannot tolerate the study procedures or who are unable to travel to the study center as required by this protocol.
Patients currently undergoing therapy with other investigational agents or patients taking drugs or food supplements which may interfere with gastrointestinal absorption or motility.
Patients suffering from clinically significant diarrhoea (greater than 3 liquid stools per day for greater than 7 days) without definable cause within 3 months of Screening Visit, or who have a history of significant gastrointestinal disorders.
Patients with an intercurrent medical condition that would render them unsuitable for the study e.g. HIV, hepatitis infection.
Patients who in the opinion of the investigator (for whatever reason) are thought to be unsuitable for the study.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
Collaborators and Investigators
Sponsor
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Study Completion
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Mental Disorders
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Lymphatic Diseases
- Neurocognitive Disorders
- Genetic Diseases, Inborn
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Dementia
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Frontotemporal Lobar Degeneration
- Histiocytosis, Non-Langerhans-Cell
- Histiocytosis
- Frontotemporal Dementia
- Pick Disease of the Brain
- Niemann-Pick Diseases
- Niemann-Pick Disease, Type A
- Niemann-Pick Disease, Type C
- Hypoglycemic Agents
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Anti-Infective Agents
- Antiviral Agents
- Enzyme Inhibitors
- Anti-HIV Agents
- Anti-Retroviral Agents
- Glycoside Hydrolase Inhibitors
- Miglustat
Other Study ID Numbers
- 030011
- 03-EI-0011
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Niemann Pick Diseases
-
Cyclo Therapeutics, Inc.RecruitingNiemann-Pick Disease, Type C1United States, Spain, Israel, Taiwan, Brazil, Germany, Australia, Poland, Turkey, Italy, United Kingdom
-
Xinhua Hospital, Shanghai Jiao Tong University...UnknownNiemann-Pick Disease, Type C1China
-
Mandos LLCTerminatedNiemann-Pick Disease, Type CCosta Rica
-
Eunice Kennedy Shriver National Institute of Child...TerminatedNiemann-Pick Disease, Type C1United States
-
Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1Israel, Sweden, United Kingdom
-
Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyCompletedNiemann-Pick Disease, Type C1United States
-
Cyclo Therapeutics, Inc.Active, not recruitingNiemann-Pick Disease, Type C1United States
-
Mandos LLCCompletedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, France, New Zealand, Singapore, Spain, Turkey
-
ActelionCompleted
-
Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyWithdrawnNiemann-Pick Disease, Type C