Study to Evaluate the Safety of AT2220 in Pompe Disease - Full Text View

Sponsor:	Amicus Therapeutics
Information provided by:	Amicus Therapeutics
ClinicalTrials.gov Identifier:	NCT00688597

Purpose

The main purpose of this study is to determine the safety and tolerability of three different doses of AT2220 in people affected by Pompe disease. The study will also evaluate the effects of AT2220 on functional parameters in Pompe disease.

Condition	Intervention	Phase
Pompe Disease	Drug: AT2220	Phase II

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety Study
Official Title:	An Open-Label, Multicenter, Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Three Dosing Regimens of Oral AT2220 in Patients With Pompe Disease

Resource links provided by NLM:

Genetics Home Reference related topics: Pompe disease succinic semialdehyde dehydrogenase deficiency

U.S. FDA Resources

Further study details as provided by Amicus Therapeutics:

Primary Outcome Measures:

Treatment-emergent Adverse Events [ Time Frame: 11 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Change in functional parameters from Baseline to End of Study [ Time Frame: 11 weeks ] [ Designated as safety issue: No ]

Enrollment:	3
Study Start Date:	November 2008
Estimated Study Completion Date:	November 2009
Estimated Primary Completion Date:	March 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Cohort 1: Experimental AT2220 low dose, regimen 1, for 11 weeks	Drug: AT2220 low dose regimen 1 powder in a bottle for dissolution in water for oral administration
Cohort 2: Experimental AT2220 high dose, regimen 1, for 11 weeks	Drug: AT2220 high dose, regimen 1
Cohort 3: Experimental AT2220 high dose, regimen 2, for 11 weeks	Drug: AT2220 high dose, regimen 2

Detailed Description:

Subjects meeting all eligibility criteria will undergo physical examination, electrocardiogram (ECG), spirometry, muscular strength test, functional muscle test, 6-minute walk test (6MWT) (when appropriate), laboratory tests, MRI and muscle (needle) biopsy. Quality of life will be assessed via SF-36 questionnaire. Functional ability and level of handicap will be assessed by Rotterdam handicap scale.

Eligibility

Ages Eligible for Study:	18 Years to 74 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Male or female, 18 to 74 years of age inclusive
Diagnosis of Pompe disease based on clinical assessment, enzyme assay, and/or genotyping. Confirmatory genotyping will be performed on all subjects who are screened for the study.
Naïve to ERT or has not received ERT in the 3 months prior to screening
Willing not to initiate ERT or other prohibited treatment during study participation
Functional grade for arms and/or legs ≥2 OR sitting FVC ≥30% and <80% of predicted value, reproducible between visits 1 and 2 (± 15%)
Subjects of reproductive potential agree to use reliable methods of contraception during the study
Subject or legal representative is willing and able to provide written informed consent

Exclusion Criteria:

Any intercurrent condition that may preclude accurate interpretation of study data
Obstructive pulmonary disease
Invasive ventilatory support
Use of noninvasive ventilatory support >8 hours/day while awake
History of QTc prolongation >450 msec for males and >470 msec for females
History of allergy or sensitivity to the study drug, including any prior serious adverse reaction to iminosugars (e.g., miglustat, miglitol)
Pregnancy or breast-feeding
Current or recent drug or alcohol abuse
Treatment with another investigational drug within 30 days of study start
Use of prohibited medications ≤3 months prior to screening
Otherwise unsuitable for the study in the opinion of investigator

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00688597

Hide Study Locations

Locations

United States, Arizona
Phoenix Neurological Associates, LTD
Phoenix, Arizona, United States, 85006
United States, California
Stanford University
Stanford, California, United States, 94305
University of California Irvine
Orange, California, United States, 92868
United States, Florida
University of Florida
Gainsville, Florida, United States, 32610-0296
United States, Georgia
Emory University
Decatur, Georgia, United States, 30033
United States, Minnesota
University of Minnesota, Gene Therapy Center
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Washington University of St. Louis
St. Louis, Missouri, United States, 63110
United States, New York
Center for Metabolic Disorders
Lake Success, New York, United States, 11042
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15238
United States, Texas
Methodist Neurological Institute
Houston, Texas, United States, 77030
United States, Washington
Providence St. Peter's Hospital
Olympia, Washington, United States, 98506
Australia
Women's and Children's Hospital
North Adelaide, Australia, 5006
Canada, Ontario
McMaster University Medical Center
Hamilton, Ontario, Canada, L8N3Z5
France
Hopital la Salpetriere
Paris, France, 75013
Germany
Neurologic Rehabilitation Centre Friedehorst
Bremen, Germany, D-28717
Ludwig Maximilians Universtiy Munich
Munchen, Germany, D-80336
Netherlands
Erasmus MC Sophia Children's Hospital
Rotterdam, Netherlands, 3015
United Kingdom
Salford Royal NHS Trust Hope Hospital
Salford, United Kingdom, M68HD

Sponsors and Collaborators

Amicus Therapeutics

Investigators

Study Director:

Mathews Adera, MD

Amicus Therapeutics

More Information

No publications provided

Responsible Party:	Amicus Therapeutics ( Mathews Adera, MD, Medical Director, Clinical Research )
Study ID Numbers:	POM-CL-201
Study First Received:	May 30, 2008
Last Updated:	November 9, 2009
ClinicalTrials.gov Identifier:	NCT00688597 History of Changes
Health Authority:	United States: Food and Drug Administration

Keywords provided by Amicus Therapeutics:

Pompe Disease

Additional relevant MeSH terms:

Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Glycogen Storage Disease
Lysosomal Storage Diseases
Nervous System Diseases
Central Nervous System Diseases
Glycogen Storage Disease Type II

Brain Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Carbohydrate Metabolism, Inborn Errors
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on November 27, 2009