A phase I/II randomised control study of OGT 918 in patients with Niemann-Pick type C disease
| ISRCTN | ISRCTN26761144 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN26761144 |
| ClinicalTrials.gov number | NCT00517153 |
| Secondary identifying numbers | OGT 918-007 |
- Submission date
- 05/07/2007
- Registration date
- 26/07/2007
- Last edited
- 02/10/2014
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nervous System Diseases
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Plain English summary of protocol
Not provided at time of registration
Contact information
Dr Marc Patterson
Scientific
Scientific
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
| Phone | +1 (0)212 305 6038 |
|---|---|
| mcp73@columbia.edu |
Study information
| Study design | Randomised controlled intervention study conducted at two centres |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Not specified |
| Study type | Treatment |
| Scientific title | |
| Study objectives | 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(s) | 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 |
| Health condition(s) or problem(s) studied | 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 |
| Pharmaceutical study type(s) | |
| Phase | Phase I/II |
| Drug / device / biological / vaccine name(s) | 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 study start date | 01/03/2003 |
| Completion date | 30/04/2004 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Other |
| Sex | Both |
| Target number of participants | 41 |
| Key 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 |
| Key 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 |
| Date of first enrolment | 01/03/2003 |
| Date of final enrolment | 30/04/2004 |
Locations
Countries of recruitment
- United Kingdom
- United States of America
Study participating centre
Division of Pediatric Neurology
New York
NY 10032
United States of America
NY 10032
United States of America
Sponsor information
Actelion Pharmaceuticals Ltd (Switzerland)
Industry
Industry
c/o Dr Cynthia Calabresse
Gewerbestrasse 16
Allschwil
4123
Switzerland
| Website | http://www.actelion.com/ |
|---|---|
"ROR" |
https://ror.org/001yedb91 |
Funders
Funder type
Industry
Actelion Pharmaceuticals Ltd (Switzerland)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | results | 01/09/2007 | Yes | No |
