An open-label, multicentre, long-term extension study to assess the safety, efficacy and pharmacodynamics of AT2101 in adult patients with Type I Gaucher disease.

• Conditions: Type I Gaucher Disease; MedDRA version: 9.1Level: LLTClassification code 10018048Term: Gaucher's disease

• Registration Number: EUCTR2008-007158-36-GB
• Lead Sponsor: Amicus Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-04-29
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

- Male or female subjects, 18 years of age or older
- Completed study GAU-CL-202 with no significant protocol violations or safety concerns
- Clinically stable
- Has not received enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) in the past 12 months and is willing not to initiate ERT or SRT during study participation
- All subjects of reproductive potential are required to practice an acceptable method of contraception as defined in Section 5.5 of the protocol
- Provides written informed consent to participate in the study
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- During the screening period (Visit 1), any clinically significant findings, based on physical and brief neurological examination, ophthalmologic examination, medical history review, laboratory assessment, vital sign assessment and/or other significant finding which would compromise the safety of the subject, or preclude the subject from completing the study as deemed by the investigator
- A clinically significant disease, severe complications from Gaucher disease, or serious intercurrent illness that may preclude participation in the study, in the opinion of the Investigator
- History of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars (e.g., miglustat)
- Pacemaker or other contraindication for MRI scanning
- Pregnant or breast-feeding
- Presence or sequelae of gastrointestinal, liver, or kidney disease, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs
- Subject is otherwise unsuitable for the study in the opinion of the investigator

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified