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

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Study to Evaluate the Safety of AT2220 in Pompe Disease

This study is currently recruiting participants.

Study NCT00688597. Last updated on November 19, 2008. Information provided by Amicus Therapeutics

This Tabular View shows the required WHO registration data elements as marked by ^†

Descriptive Information Fields

Brief Title ^†

Study to Evaluate the Safety of AT2220 in Pompe Disease

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

Brief Summary

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.

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.

Study Phase

Phase II

Study Type ^†

Interventional

Study Design ^†

Treatment, Non-Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety Study

Primary Outcome Measure ^†

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

Secondary Outcome Measure ^†

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

Condition ^†

Pompe Disease

Intervention ^†

Drug: AT2220

MEDLINE PMIDs

Links

Recruitment Information Fields

Recruitment Status ^†

Recruiting

Enrollment ^†

Start Date ^†

November 2008

Completion Date

May 2009

Eligibility 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

Gender

Both

Ages

18 Years to 74 Years

Accepts Healthy Volunteers

Contacts ^††

Location Countries ^†

United States, Canada, France, Germany, Netherlands, United Kingdom

Administrative Information Fields

NCT ID ^†

NCT00688597

Organization ID

POM-CL-201

Secondary IDs ^††

Study Sponsor ^†

Amicus Therapeutics

Collaborators ^††

Investigators ^†

Study Director:

Andrew Shenker, MD, PhD

Amicus Therapeutics

Information Provided By

Amicus Therapeutics

Verification Date

November 2008

First Received Date ^†

May 30, 2008

Last Updated Date

November 19, 2008

^† Required WHO trial registration data element.
^†† WHO trial registration data element that is required only if it exists.