OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00041535
• Date of registration: 09/07/2002
• Prospective Registration: No
• Primary sponsor: National Institute of Neurological Disorders and Stroke (NINDS)
• Public title: OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease
• Scientific title: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease
• Date of first enrolment: July 5, 2002
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00041535
• Study type: Interventional
• Study design: Primary purpose: Treatment.
• Phase: Phase 2

Countries of recruitment

United Kingdom, United States

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

Patients with neuronopathic Gaucher disease, confirmed by clinical diagnosis, who if
receiving enzyme replacement therapy have been stable on enzyme replacement therapy for at
least 6 months or have successfully undergone a bone marrow transplant at least one year
prior to study entry.

Patients who are at least 4 years old and can swallow a capsule.

EXCLUSION CRITERIA:

Patients younger than 18 years who are unable to give informed assent and/or whose legal
guardian is unable to provide informed consent.

Patients aged 18 and over who cannot provide informed consent and/or whose legal guardian
is unable to provide witnessed informed consent.

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.

Patients who cannot tolerate the study procedures or who are unable to travel to the study
center as required by this protocol.

Patients currently undergoing therapy with other investigational agents or patients taking
drugs or food supplements which may interfere with gastrointestinal absorption or motility.

Patients suffering from clinically significant diarrhea (greater than 3 liquid stools per
day for greater than 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.

Patients who in the opinion of the investigator (for whatever reason) are thought to be
unsuitable for the study.

Patients with an adjusted Creatinine Clearance of less than 70 ml/min/1.73m(2) (CrCl less
than 70).



Age minimum: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Gaucher Disease

Intervention(s)

Drug: OGT 918

Secondary ID(s)

02-N-0243

020243

Source(s) of Monetary Support

Please refer to primary and secondary sponsors