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

Sponsored by:	Amicus Therapeutics
Information provided by:	Amicus Therapeutics
ClinicalTrials.gov Identifier:	NCT00813865

Purpose

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.

Condition	Intervention	Phase
Gaucher Disease Type 1 Gaucher Disease Gaucher Disease, Type 1	Drug: AT2101	Phase II

Genetics Home Reference related topics: cholesteryl ester storage disease Farber lipogranulomatosis Gaucher disease long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency mitochondrial trifunctional protein deficiency primary carnitine deficiency

MedlinePlus related topics: Gaucher's Disease

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety/Efficacy Study
Official Title:	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

Further study details as provided by Amicus Therapeutics:

Primary Outcome Measures:

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 ]

Secondary Outcome Measures:

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 ]

Estimated Enrollment:	16
Study Start Date:	January 2009
Estimated Study Completion Date:	December 2011
Estimated Primary Completion Date:	December 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Arm 1: Experimental AT2101 dose 1, regimen 1 (same as in the lead-in study GAU-CL-202)	Drug: AT2101 AT2101 oral capsules
Arm 2: Experimental AT2101, dose 1, regimen 2 (same as in the lead-in study GAU-CL-202)	Drug: AT2101 AT2101 oral capsules

Detailed Description:

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, and at the end of study (EOS) visit, participants will undergo a comprehensive physical examination, MRIs of liver, spleen and femoral bones, DEXA scans of femoral bones and lumbar spine (if performed in Study GAU-CL-202), and 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.

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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
Willing not to initiate enzyme replacement therapy (ERT) or substrate reduction therapy (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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00813865

Locations

United States, Florida
University Research Foundation for Lysosomal Storage Diseases, Inc.
Coral Springs, Florida, United States, 33065
United Kingdom
Royal Free Hospital
London, United Kingdom, WC1N 3BG

Sponsors and Collaborators

Amicus Therapeutics

Investigators

Study Director:

Eugene Schneider, MD

Amicus Therapeutics

More Information

Responsible Party:	Amicus Therapeutics ( Eugene Schneider, MD, Medical Director, Clinical Research )
Study ID Numbers:	GAU-CL-202X
Study First Received:	December 22, 2008
Last Updated:	December 22, 2008
ClinicalTrials.gov Identifier:	NCT00813865
Health Authority:	United States: Food and Drug Administration; United Kingdom: Medicines and Healthcare Products Regulatory Agency

Study placed in the following topic categories:

Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Lysosomal Storage Diseases
Sphingolipidosis
Central Nervous System Diseases
Brain Diseases
Lymphatic Diseases

Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Lipidoses
Metabolic disorder
Gaucher Disease
Lipid Metabolism Disorders
Brain Diseases, Metabolic

Additional relevant MeSH terms:

Reticuloendotheliosis
Lysosomal Storage Diseases, Nervous System
Nervous System Diseases

ClinicalTrials.gov processed this record on January 13, 2009