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

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 2


Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment
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

Resource links provided by NLM:

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Gaucher disease Schindler disease succinic semialdehyde dehydrogenase deficiency

MedlinePlus related topics: Gaucher's Disease

Genetic and Rare Diseases Information Center resources: Gaucher Disease Gaucher Disease Type 1 Sphingolipidosis

U.S. FDA Resources

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 ]


Enrollment:	8
Study Start Date:	January 2009
Study Completion Date:	May 2012
Primary Completion Date:	May 2012 (Final data collection date for primary outcome measure)

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

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, 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.

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
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

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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
Paraguay
Sociedad Espanola de Soccorros Mutuos
Asuncion, Paraguay
United Kingdom
Royal Free Hospital
London, United Kingdom, WC1N 3BG

Sponsors and Collaborators

Amicus Therapeutics

Investigators


Study Director:	Medical Director	Amicus Therapeutics

More Information

No publications provided


Responsible Party:	Amicus Therapeutics
ClinicalTrials.gov Identifier:	NCT00813865 History of Changes
Other Study ID Numbers:	GAU-CL-202X
Study First Received:	December 22, 2008
Last Updated:	September 7, 2012
Health Authority:	United States: Food and Drug Administration United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:


Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors	Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Sphingolipidoses

ClinicalTrials.gov processed this record on March 03, 2015