Condition category
Nervous System Diseases
Date applied
05/07/2007
Date assigned
26/07/2007
Last edited
02/10/2014
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

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 measures

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

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

http://www.actelion.com/

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

Results - basic reporting

Publication summary

2007 results in: http://www.ncbi.nlm.nih.gov/pubmed/17689147

Publication citations

  1. 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.

Additional files

Editorial Notes