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Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease

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ClinicalTrials.gov Identifier: NCT00316498
Recruitment Status : Completed
First Posted : April 20, 2006
Last Update Posted : July 2, 2017
Sponsor:
Information provided by:
National Institutes of Health Clinical Center (CC)

Tracking Information
First Submitted Date  ICMJE April 19, 2006
First Posted Date  ICMJE April 20, 2006
Last Update Posted Date July 2, 2017
Study 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 Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
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
Brief Summary

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  ICMJE Phase 1
Study Design  ICMJE Primary Purpose: Treatment
Condition  ICMJE Niemann Pick Diseases
Intervention  ICMJE Drug: OGT918
Study Arms  ICMJE 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 Information
Recruitment Status  ICMJE Completed
Enrollment  ICMJE
 (submitted: April¬†19,¬†2006)
30
Original Enrollment  ICMJE Same as current
Study Completion Date  ICMJE August 15, 2007
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE
  • 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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE 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  
 
Administrative Information
NCT Number  ICMJE NCT00316498
Other Study ID Numbers  ICMJE 030011
03-EI-0011
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE 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