Contact information
Type
Scientific
Primary contact
Dr Marc Patterson
ORCID ID
Contact details
Division of Pediatric Neurology
Neurological Institute of New York
and
Columbia University College of Physicians and Surgeons
180 Fort Washington Avenue
HP-542
New York
NY 10032
United States of America
+1 (0)212 305 6038
mcp73@columbia.edu
Additional identifiers
EudraCT number
ClinicalTrials.gov number
NCT00517153
Protocol/serial number
OGT 918-007
Study information
Scientific title
Acronym
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.
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.
Ethics approval
1. Centre 1: Salford and Trafford LREC, 24/12/2001, ref: 01266
2. Centre 2: Institutional Review Board of Columbia Presbyterian Medical Centre, 05/04/2002, ref: 14413
Study design
Randomised controlled intervention study conducted at two centres
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Not specified
Trial type
Treatment
Patient information sheet
Condition
Niemann-Pick type C disease
Intervention
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.
Intervention type
Drug
Phase
Phase I/II
Drug names
Miglustat (OGT 918)
Primary outcome measure
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 measures
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)
Overall trial start date
01/03/2003
Overall trial end date
30/04/2004
Reason abandoned (if study stopped)
Eligibility
Participant 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
Participant type
Patient
Age group
Other
Gender
Both
Target number of participants
41
Participant exclusion criteria
1. Clinically significant diarrhoea (greater than three liquid stools per day for more than 7 days without definable cause) within 3 months before enrolment
2. Significant gastrointestinal disorders or other intercurrent illnesses
Recruitment start date
01/03/2003
Recruitment end date
30/04/2004
Locations
Countries of recruitment
United Kingdom, United States of America
Trial participating centre
Division of Pediatric Neurology
New York
NY 10032
United States of America
Sponsor information
Organisation
Actelion Pharmaceuticals Ltd (Switzerland)
Sponsor details
c/o Dr Cynthia Calabresse
Gewerbestrasse 16
Allschwil
4123
Switzerland
Sponsor type
Industry
Website
Funders
Funder type
Industry
Funder name
Actelion Pharmaceuticals Ltd (Switzerland)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Basic results (scientific)
Publication list
2007 results in: http://www.ncbi.nlm.nih.gov/pubmed/17689147
Publication citations
-
Results
Patterson MC, Vecchio D, Prady H, Abel L, Wraith JE, Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study., Lancet Neurol, 2007, 6, 9, 765-772, doi: 10.1016/S1474-4422(07)70194-1.