Safety Study of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
Information source: Amicus Therapeutics
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Gaucher Disease, Type 1
Intervention: AT2101 (Drug)
Phase: Phase 2
Status: Completed
Sponsored by: Amicus Therapeutics Official(s) and/or principal investigator(s):
Eugene Schneider, MD, Study Director, Affiliation: Amicus Therapeutics
Summary
Gaucher disease is a lysosomal storage disorder resulting from a deficiency in the key enzyme
beta-glucocerebrosidase (GCase). The enzyme deficiency is caused by genetic mutations, which
can result in the production of misfolded GCase. AT2101 is designed to act as a
pharmacological chaperone by selectively binding to the misfolded GCase and helping it fold
correctly, which may restore GCase activity.
This study is being conducted to test the safety of AT2101 in patients with type 1 Gaucher
disease already receiving enzyme replacement therapy (ERT). The study will involve six visits
over a period of 7 weeks and will evaluate the safety of AT2101.
Clinical Details
Official title: A Randomized, Open-Label Study to Assess the Safety and Tolerability of Multiple Dose Levels and Multiple Dosing Regimens of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving ERT
Study design: Treatment, Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety Study
Primary outcome: The primary objective of the study is to evaluate the safety of AT2101.
Secondary outcome: The secondary objective of the study is to assess pharmacodynamic effects of AT2101, including beta-glucocerebrosidase (GCase), glucocerebroside (GlcCer), chitotriosidase, and pulmonary and activation regulated chemokine (PARC).
Eligibility
Minimum age: 18 Years.
Maximum age: 65 Years.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Have a confirmed diagnosis of type 1 Gaucher disease with a documented gene mutation
- Clinically stable
- Male or female subjects, 18 to 65 years old
- All subjects of childbearing potential must be using adequate birth control
- Body mass index (BMI) between 18 and 30 kg/m2 and weighing at least 60 kg for males
and 52 kg for females
- Provide written informed consent to participate in the study
Exclusion Criteria:
- Clinically significant disease, severe complications from Gaucher disease, or serious
illness that may preclude participation in the study in the opinion of the
Investigator that would compromise the safety of the subject or preclude the subject
from completing the study
- During the screening period, any clinically significant findings, as deemed by the
investigator
- Partial or total splenectomy (removal of spleen)
- History of pulmonary hypertension or Gaucher related lung disease
- History of allergy or sensitivity to the study drug or any excipients, including any
prior serious adverse reaction to iminosugars (e. g., N-butyldeoxynojirimycin or
miglustat)
- Pregnant or breast-feeding
- Current/recent drug or alcohol abuse
- Treatment with any investigational product in the 90 days before study entry
- Treatment in the previous 90 days with any drug known to have a well defined potential
for toxicity to a major organ
- Presence or symptoms of gastrointestinal, liver or kidney disease, or other conditions
known to interfere with the absorption, distribution, metabolism, or excretion of
drugs
Locations and Contacts
University of California San Francisco, San Francisco, California 94143-0748, United States
University Research Foundation for Lysosomal Storage Diseases, Inc, Coral Springs, Florida 33065, United States
Emory University Lysosomal Storage Disease Center, Decatur, Georgia 30033, United States
University of Iowa Health Center, Iowa City, Iowa 52242, United States
University of Minnesota, Minneapolis, Minnesota 55455, United States
New York University School of Medicine, New York, New York 10016, United States
Lysosomal Disease Center, Cincinnati, Ohio 45229, United States
Oregon Health and Science University, Portland, Oregon 97239, United States
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States
Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania 15213, United States
Baylor College of Medicine, Houston, Texas 77030, United States
Additional Information
Starting date: February 2007
Ending date: March 2008
Last updated: June 4, 2008
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