A Long-Term Extension Study of AT2101 in Type 1 Gaucher Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics
ClinicalTrials.gov Identifier:
NCT00813865
First received: December 22, 2008
Last updated: September 7, 2012
Last verified: September 2012

December 22, 2008
September 7, 2012
January 2009
May 2012   (final data collection date for primary outcome measure)
The primary objective of the study is to evaluate the long-term safety of orally administered AT2101 in adult patients with type 1 Gaucher disease. [ Time Frame: Every 3 months ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT00813865 on ClinicalTrials.gov Archive Site
  • The secondary objective of the study is to assess the long-term efficacy of orally administered AT2101 in adult patients with type 1 Gaucher disease [ Time Frame: Every 3 months ] [ Designated as safety issue: No ]
  • The tertiary objective of the study is to assess the pharmacodynamics of orally administered AT2101 in adult patients with type 1 Gaucher disease [ Time Frame: Every 3 months ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
A Long-Term Extension Study of AT2101 in Type 1 Gaucher Patients
An Open-label, Multicenter, Long-term Extension Study to Assess the Safety, Efficacy, and Pharmacodynamics of AT2101 in Adult Patients With Type 1 Gaucher Disease

This study will measure the long-term safety of AT2101 and its effects on hemoglobin, platelets, liver and spleen volume, and bone density. This study will also look at the effects of AT2101 on beta-glucocerebrosidase levels, glucosylceramide levels, and other blood markers of Gaucher disease.

This is an open-label, long-term extension study designed to assess the long-term safety, efficacy and pharmacodynamics of AT2101 in patients with type 1 Gaucher disease who successfully complete Study GAU-CL-202. Participants may enter this study immediately upon completion of Study GAU-CL-202, or at any later time point. Each participant will continue to receive AT2101 at the same dose level and regimen as in the previous study.

Study visits will occur every 3 months. At every visit, safety evaluations will be performed and blood samples will be collected for pharmacodynamic tests. At the completion of every year of treatment in this study, or at the early termination visit, participants will undergo a comprehensive physical examination, MRIs of liver, spleen and femoral bones, DEXA scans of femoral bones and lumbar spine, and complete the SF-36 questionnaire. Duration of treatment will depend on the time of each participant's enrollment in the study. Participants will be contacted approximately 1 month after study completion for assessment of adverse events.

Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Gaucher Disease
  • Type 1 Gaucher Disease
  • Gaucher Disease, Type 1
Drug: AT2101
AT2101 oral capsules
Other Name: AT2101, afegostat tartrate
  • Experimental: Arm 1
    AT2101 dose 1, regimen 1 (same as in the lead-in study GAU-CL-202)
    Intervention: Drug: AT2101
  • Experimental: Arm 2
    AT2101, dose 1, regimen 2 (same as in the lead-in study GAU-CL-202)
    Intervention: Drug: AT2101
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
8
May 2012
May 2012   (final data collection date for primary outcome measure)

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
  • Provides written informed consent to participate in the study

Exclusion Criteria:

  • During the screening period (Visit 1), any clinically significant findings 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
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   Paraguay,   United Kingdom
 
NCT00813865
GAU-CL-202X
No
Amicus Therapeutics
Amicus Therapeutics
Not Provided
Study Director: Medical Director Amicus Therapeutics
Amicus Therapeutics
September 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP