Niemann-Pick Type C Disease
Conditions
Keywords
Zavesca, miglustat, Niemann-Pick Type C, Actelion
Brief summary
This is a phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2:1 ratio to either treatment with miglustat or to a non-treatment group. Both groups will follow an identical visit schedule.
Interventions
DRUGmiglustat
Patients aged 12 years or more randomised to the treatment group will receive 200 mg OGT 918 three times daily initially for twelve months. An additional sub-study of up to 12 patients aged under 12 years will receive a lower dose of OGT 918 according to their BSA
Sponsors
Actelion
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
4 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Patients with Niemann-Pick Type C disease confirmed by abnormal cholesterol esterification and abnormal filipin staining. 2. Patients who can ingest a capsule. 3. Patients who are above the age of four (4) years of age.
Exclusion criteria
1. Patients younger than 18 years who are unable to give informed assent and/or whose legal guardian is unable to provide informed consent. 2. Patients aged 18 and over who cannot provide informed consent and/or whose legal guardian is unable to provide witnessed informed consent. 3. Sexually active and fertile patients who do not agree to use adequate contraception throughout the study and for three months after cessation of OGT 918 treatment. 4. Patients who cannot tolerate the study procedures or who are unable to travel to the study center as required by this protocol. 5. Patients currently undergoing therapy with other investigational agents or patients taking drugs or food supplements which may interfere with gastrointestinal absorption or motility. 6. Patients suffering from clinically significant diarrhea (\>3 liquid stools per day for \>7 days) without definable cause within 3 months of Screening Visit, or who have a history of significant gastrointestinal disorders. 7. Patients with an intercurrent medical condition that would render them unsuitable for the study e.g. HIV, hepatitis infection. 8. Patients who in the opinion of the Investigator (for whatever reason) are thought to be unsuitable for the study. 9. Patients with an adjusted Creatinine Clearance of less than 70 ml/min/1.73m2 (CrCl \<70). 10. Patients younger than four (4) years of age.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoints will be two parameters - Horizontal Saccadic α and Horizontal Saccadic β - which are estimated for each patient from their saccadic eye movement data | Baseline to Month 12 |
Secondary
| Measure | Time frame |
|---|---|
| Highest amplitude for which a velocity measurement is obtained | Baseline to Month 12 |
Outcome results
None listed