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

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

Genetics Home Reference related topics:

Pompe disease

U.S. FDA Resources

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

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 ]

Estimated Enrollment:	18
Study Start Date:	July 2008
Estimated Study Completion Date:	May 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

Show 20 Study Locations

Sponsors and Collaborators

Amicus Therapeutics

Investigators


Study Director:	Andrew Shenker, MD, PhD	Amicus Therapeutics

More Information

Responsible Party:	Amicus Therapeutics ( Andrew Shenker, MD, PhD, Vice President, Clinical Research )
Study ID Numbers:	POM-CL-201
First Received:	May 30, 2008
Last Updated:	June 16, 2008
ClinicalTrials.gov Identifier:	NCT00688597
Health Authority:	United States: Food and Drug Administration

Keywords provided by Amicus Therapeutics:

Pompe Disease

Study placed in the following topic categories:

Glycogen storage disease type 2

Metabolism, Inborn Errors

Metabolic Diseases

Genetic Diseases, Inborn

Lysosomal Storage Diseases

Glycogen Storage Disease

Central Nervous System Diseases

Glycogen Storage Disease Type II

Brain Diseases, Metabolic, Inborn

Metabolic disorder

Brain Diseases

Brain Diseases, Metabolic

Additional relevant MeSH terms:

Lysosomal Storage Diseases, Nervous System

Nervous System Diseases

Carbohydrate Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on August 29, 2008

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