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A Study of Oral AT2101 in Treatment-naive Patients With Gaucher Disease

This study has been completed.
Sponsor:
Information provided by:
Amicus Therapeutics
ClinicalTrials.gov Identifier:
NCT00446550
First received: March 9, 2007
Last updated: May 12, 2011
Last verified: May 2011

March 9, 2007
May 12, 2011
December 2007
August 2009   (final data collection date for primary outcome measure)
The primary objective of this study is to evaluate the safety and tolerability of AT2101. [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]
The primary objective of the study is to evaluate the safety of AT2101.
Complete list of historical versions of study NCT00446550 on ClinicalTrials.gov Archive Site
The secondary objective of the study is to evaluate the pharmacodynamic effects of AT2101. [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]
The secondary objective of the study is to evaluate the pharmacodynamic effects of AT2101.
Not Provided
Not Provided
 
A Study of Oral AT2101 in Treatment-naive Patients With Gaucher Disease
A Randomized, Open-label Study To Assess the Safety and Tolerability of AT2101 in Treatment-naive Adult Patients With Type I Gaucher Disease

This study is being conducted to evaluate the safety and effects of AT2101 in patients with type I Gaucher disease who are not receiving ERT or SRT.

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 I Gaucher disease who have not already received enzyme replacement therapy (ERT) or substrate reduction therapy (SRT), or who have not received ERT or SRT in the 12 months before screening. This study will also evaluate the effects of AT2101 on parameters that are commonly abnormal in Gaucher disease. The study will involve 9 visits over 29 weeks.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Gaucher Disease, Type 1
Drug: AT2101

Arm 1: AT2101 oral capsules, dose 1, regimen 1

Arm 2: AT2101 oral capsules, dose 1, regimen 2

  • Experimental: 1
    AT2101 dose regimen 1
    Intervention: Drug: AT2101
  • Experimental: 2
    AT2101 dose regimen 2
    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
19
August 2009
August 2009   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Confirmed diagnosis of type 1 Gaucher disease with a known genotype and a documented missense gene mutation in at least one of the two mutated GBA alleles
  • Clinically stable
  • Treatment naïve to enzyme replacement therapy (ERT) and substrate reduction therapy (SRT) or has not received ERT or SRT in the 12 months before screening
  • Willing not to initiate ERT or SRT treatment during study participation
  • At the screening period visit, subjects must meet at least two of the following criteria:
  • Platelet count of ≤ 150,000 per microliter
  • Hemoglobin ≤ 12 g/dL for females and ≤13 g/dL for males
  • Liver volume ≥ 1.25 multiples of normal
  • Spleen volume ≥ 2 multiples of normal
  • All subjects of reproductive potential are required to practice an acceptable method of contraception
  • Provide written informed consent to participate in the study

Exclusion Criteria:

  • A clinically significant disease, severe complications from Gaucher disease, or serious illness that may preclude participation in the study in the opinion of the Investigator
  • During the screening period, any clinically significant findings, as deemed by the Investigator
  • Partial or total splenectomy
  • Documentation of moderate or severe pulmonary hypertension, defined as pulmonary arterial pressure (PAP) > 35 mmHg or significant Gaucher related lung disease
  • History of allergy or sensitivity to the study drug or any excipients, including any prior serious adverse reaction to iminosugars
  • Pacemaker or other contraindication for MRI scanning
  • Pregnant or breast-feeding
  • Current/recent drug or alcohol abuse
  • Treatment with any investigational product in the last 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 of symptoms of gastrointestinal, liver or kidney disease, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs
Both
18 Years to 74 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Germany,   Israel,   South Africa,   United Kingdom
 
NCT00446550
GAU-CL-202
Yes
Eugene Schneider, MD / Medical Director, Clinical Research, Amicus Therapeutics
Amicus Therapeutics
Not Provided
Study Director: Eugene Schneider, MD Amicus Therapeutics
Amicus Therapeutics
May 2011

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