Dettagli per singolo studio

DEC-NET Serial number GB487

Published online	26/09/2005 13.38.00
Last updated	26/09/2005 13.40.27

Other protocol ID number	OGT 918 -006
This trial has been approved by an ethics committee
Current trial status	Closed to recruitment of participants: follow-up continuing
First subject enrolment Target N. of subjects	08/2003 N/A
Major Disease (ICD9 class)	LIPIDOSES
Experimental drug
Miglustat (OGT 918) Treatment regimen (dosage and duration) Recommended dose reductions of OGT 918 BSA (m2) recommended dose 1.8 (adult) 200mg TID 1.25 200mg BID 0.88 200mg in morning, 100mg in afternoon 0.62-0.73 100mg BID 0.47 100mg OD
Gender	Both
Age (range)	2 - 19 years
Eligibility criteria
Inclusion criteria
Children with neuronopathic Gaucher Disease, confirmed by clinical diagnosis, who if receiving enzyme replacement therapy have been on a stable dose for at least 6 months or have sucessfully undergone a bone marrow transplant at leastone year prior to study entry. Patients who can swallow a capsule
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 years and over who cannot provide informed consent and/or whose legal guardian is unable to provide witnessed informed consent 3. Fertile patients, who at the time of the study could be sexually active, and 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 centre as required by the 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 diarrhoea (>3 liquid stools per day for > 7 days) without definable cause within 3 months of the screening visit, or who have a history of significant gastrointestinal disorders. 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 70ml/min/1.73m2 (CrCl<70).
Trial design/methodology
Phase	2
Kind of study	Efficacy Safety Pharmacokinetics
Design	Controlled Randomised
Purpose of study
To see if OGT 918 ameliorates the outcome in patients with Neuronopathic Gaucher Disease
Primary outcomes
Measurement of the vertical component of the eye movement
Secondary outcomes
Biomarkers, Organ Volume measurements, lung function, somatosensory evoked potentials, brainstem auditory evoked potentials. Safety -temor assessment, nerve conduction velocity
Summary of study design, objectives, and ongoing research findings
Randomised controlled trial - random allocation (A) substrate deprivation (B) no substrate deprivation