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ISRCTN
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ISRCTN26761144
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ClinicalTrials.gov identifier
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Public title
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A phase I/II randomised control study of OGT 918 in patients with Niemann-Pick type C disease
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Scientific title
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Acronym
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N/A
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Serial number at source
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OGT 918-007
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Study hypothesis
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Niemann-Pick type C disease is an inherited neurodegenerative disorder characterised by an intracellular lipid-trafficking defect with secondary accumulation of Glycosphingolipids (GSLs).
The purpose of the study was to evaluate the effects of miglustat (OGT 918) as a treatment for Niemann-Pick Type C Disease in adult, juvenile, and paediatric patients over a 24-month treatment period. We hypothesised that patients in the treatment group would show slower rates of decline or stabilisation in one or more markers of the disease compared to the standard care group.
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Ethics approval
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Approval received from:
1. Centre 1: Salford and Trafford LREC on the 24th December 2001 (ref: 01266)
2. Centre 2: Institutional Review Board of Columbia Presbyterian Medical Centre on the 5th April 2002 (ref: 14413)
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Study design
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Randomised controlled intervention study conducted at two centres.
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Countries of recruitment
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United Kingdom, United States of America
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Disease/condition/study domain
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Niemann-Pick type C disease
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Participants - inclusion criteria
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1. Juveniles and adults (12 years and over) and paediatric patients aged 4 - 11 years
2. Patients with Niemann-Pick type C disease confirmed by reduced cholesterol esterification and abnormal filipin staining in cultured fibroblasts
3. Capable of cooperating with physical examination and other testing
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Participants - exclusion criteria
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1. Clinically significant diarrhoea (greater than three liquid stools per day for more than seven days without definable cause) within three months before enrolment
2. Significant gastrointestinal disorders or other intercurrent illnesses
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Anticipated start date
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01/03/2003
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Anticipated end date
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30/04/2004
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Status of trial
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Completed |
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Patient information material
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Target number of participants
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41
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Interventions
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Patients in the juvenile/adult group (12 years or older) were randomised in a 2:1 ratio to either miglustat 200 mg three times daily orally (p.o.) for 12 months or standard symptomatic care (no study drug) as a control group.
Both miglustat-treated and standard care groups received other concomitant medications for standard indications throughout the study. All children received miglustat in a dose adjusted according to Body Surface Area (BSA).
Patients were assessed one week after commencing miglustat therapy and monthly thereafter with dose modification as clinically indicated.
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Primary outcome measure(s)
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Primary efficacy endpoint: change from baseline in Horizontal Saccadic Eye Movement (HSEM)-alpha (a measure of HSEM velocity) at 12 months or last available value.
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Secondary outcome measure(s)
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Secondary efficacy endpoints:
1. HSEM-beta
2. Assessments of swallowing (at screening and months 6 and 12; the assessor evaluated the patient's swallowing ability with prespecified substances, using a five-degree category scale from "no problems of swallowing" to "could not swallow the substance at all")
3. Auditory acuity (part of neurological examination at screening and months 3, 6, 9 and 12)
4. Ambulatory ability (standard ambulation index; part of neurological examination at screening and months 3, 6, 9 and 12)
5. Cognition (Mini Mental Status Examination (MMSE); at screening and months 3, 6, 9, 12)
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Sources of funding
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Actelion Pharmaceuticals Ltd (Switzerland)
The study was initially supported by Oxford GlycoSciences, the original manufacturer of miglustat (OGT 918). During the study the sponsor changed from Oxford GlycoSciences, a wholly-owned subsidiary of Celltech R&D Ltd, to Actelion Pharmaceuticals Ltd.
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Trial website
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Publications
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Contact name
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Dr
Marc
Patterson
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Address
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Division of Pediatric Neurology
Neurological Institute of New York, and
Columbia University College of Physicians and Surgeons
180 Fort Washington Avenue, HP-542
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City/town
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New York
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Zip/Postcode
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NY 10032
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Country
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United States of America
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Tel
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+1 212 305 6038
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Email
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mcp73@columbia.edu
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Sponsor
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Actelion Pharmaceuticals Ltd (Switzerland)
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Address
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c/o Dr Cynthia Calabresse
Gewerbestrasse 16
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City/town
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Allschwil
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Zip/Postcode
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4123
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Country
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Switzerland
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Sponsor website:
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http://www.actelion.com/
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Date applied
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05/07/2007
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Last edited
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20/09/2007
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Date ISRCTN assigned
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26/07/2007
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