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A phase I/II randomised control study of OGT 918 in patients with Niemann-Pick type C disease
ISRCTN ISRCTN26761144
DOI 10.1186/ISRCTN26761144
ClinicalTrials.gov identifier NCT00517153
EudraCT number
Public title A phase I/II randomised control study of OGT 918 in patients with Niemann-Pick type C disease
Scientific title
Acronym N/A
Serial number at source OGT 918-007
Study hypothesis 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.
Lay summary Not provided at time of registration
Ethics approval Approval received from:
1. Centre 1: Salford and Trafford LREC on the 24/12/2001 (ref: 01266)
2. Centre 2: Institutional Review Board of Columbia Presbyterian Medical Centre on the 05/04/2002 (ref: 14413)
Study design Randomised controlled intervention study conducted at two centres.
Countries of recruitment United Kingdom, United States of America
Disease/condition/study domain Niemann-Pick type C disease
Participants - inclusion criteria 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
Participants - exclusion criteria 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
Anticipated start date 01/03/2003
Anticipated end date 30/04/2004
Status of trial Completed
Patient information material
Target number of participants 41
Interventions 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.
Primary outcome measure(s) 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.
Secondary outcome measure(s) 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)
Sources of funding 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.
Trial website
Publications 2007 results in http://www.ncbi.nlm.nih.gov/pubmed/17689147
Contact name Dr  Marc  Patterson
  Address Division of Pediatric Neurology
Neurological Institute of New York, and
Columbia University College of Physicians and Surgeons
180 Fort Washington Avenue, HP-542
  City/town New York
  Zip/Postcode NY 10032
  Country United States of America
  Tel +1 212 305 6038
  Email mcp73@columbia.edu
Sponsor Actelion Pharmaceuticals Ltd (Switzerland)
  Address c/o Dr Cynthia Calabresse
Gewerbestrasse 16
  City/town Allschwil
  Zip/Postcode 4123
  Country Switzerland
  Sponsor website: http://www.actelion.com/
Date applied 05/07/2007
Last edited 26/09/2012
Date ISRCTN assigned 26/07/2007
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