|First Submitted Date ICMJE||April 19, 2006|
|First Posted Date ICMJE||April 20, 2006|
|Last Update Posted Date||July 2, 2017|
|Start Date ICMJE||October 17, 2002|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures ICMJE||Not Provided|
|Original Primary Outcome Measures ICMJE||Not Provided|
|Change History||Complete list of historical versions of study NCT00316498 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures ICMJE||Not Provided|
|Original Secondary Outcome Measures ICMJE||Not Provided|
|Current Other Outcome Measures ICMJE||Not Provided|
|Original Other Outcome Measures ICMJE||Not Provided|
|Brief Title ICMJE||Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease|
|Official Title ICMJE||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.
|Detailed Description||None given, verified by PI.|
|Study Type ICMJE||Interventional|
|Study Phase||Phase 1|
|Study Design ICMJE||Primary Purpose: Treatment|
|Condition ICMJE||Niemann Pick Diseases|
|Intervention ICMJE||Drug: OGT918|
|Study Arms||Not Provided|
|Publications *||Platt FM, Neises GR, Reinkensmeier G, Townsend MJ, Perry VH, Proia RL, Winchester B, Dwek RA, Butters TD. Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin. Science. 1997 Apr 18;276(5311):428-31.|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Completed|
|Estimated Completion Date||August 15, 2007|
|Primary Completion Date||Not Provided|
|Eligibility Criteria ICMJE||
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.
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.
|Ages||12 Years and older (Child, Adult, Senior)|
|Accepts Healthy Volunteers||No|
|Contacts ICMJE||Contact information is only displayed when the study is recruiting subjects|
|Listed Location Countries ICMJE||United Kingdom, United States|
|Removed Location Countries|
|NCT Number ICMJE||NCT00316498|
|Other Study ID Numbers ICMJE||030011
|Has Data Monitoring Committee||Not Provided|
|U.S. FDA-regulated Product||Not Provided|
|IPD Sharing Statement||Not Provided|
|Responsible Party||Not Provided|
|Study Sponsor ICMJE||National Eye Institute (NEI)|
|Collaborators ICMJE||Not Provided|
|Investigators ICMJE||Not Provided|
|PRS Account||National Institutes of Health Clinical Center (CC)|
|Verification Date||August 15, 2007|
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP