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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00688597




Registration number
NCT00688597
Ethics application status
Date submitted
30/05/2008
Date registered
30/05/2008
Date last updated
16/05/2011

Titles & IDs
Public title
Study to Evaluate the Safety of AT2220 in Pompe Disease
Scientific 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
Secondary ID [1] 0 0
POM-CL-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pompe Disease 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AT2220
Treatment: Drugs - AT2220
Treatment: Drugs - AT2220

Experimental: Cohort 1 - AT2220 low dose, regimen 1, for 11 weeks

Experimental: Cohort 2 - AT2220 high dose, regimen 1, for 11 weeks

Experimental: Cohort 3 - AT2220 high dose, regimen 2, for 11 weeks


Treatment: Drugs: AT2220
low dose regimen 1
powder in a bottle for dissolution in water for oral administration

Treatment: Drugs: AT2220
high dose, regimen 1

Treatment: Drugs: AT2220
high dose, regimen 2

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Treatment-emergent Adverse Events
Timepoint [1] 0 0
11 weeks
Secondary outcome [1] 0 0
Change in functional parameters from Baseline to End of Study
Timepoint [1] 0 0
11 weeks

Eligibility
Key inclusion criteria
1. Male or female, 18 to 74 years of age inclusive

2. 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.

3. Naïve to ERT or has not received ERT in the 3 months prior to screening

4. Willing not to initiate ERT or other prohibited treatment during study participation

5. Functional grade for arms and/or legs =2 OR sitting FVC =30% and <80% of predicted
value, reproducible between visits 1 and 2 (± 15%)

6. Subjects of reproductive potential agree to use reliable methods of contraception
during the study

7. Subject or legal representative is willing and able to provide written informed
consent
Minimum age
18 Years
Maximum age
74 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Any intercurrent condition that may preclude accurate interpretation of study data

2. Obstructive pulmonary disease

3. Invasive ventilatory support

4. Use of noninvasive ventilatory support >8 hours/day while awake

5. History of QTc prolongation >450 msec for males and >470 msec for females

6. History of allergy or sensitivity to the study drug, including any prior serious
adverse reaction to iminosugars (e.g., miglustat, miglitol)

7. Pregnancy or breast-feeding

8. Current or recent drug or alcohol abuse

9. Treatment with another investigational drug within 30 days of study start

10. Use of prohibited medications =3 months prior to screening

11. Otherwise unsuitable for the study in the opinion of investigator

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Women's and Children's Hospital - North Adelaide
Recruitment postcode(s) [1] 0 0
5006 - North Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
North Carolina
Country [9] 0 0
United States of America
State/province [9] 0 0
Oregon
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
United States of America
State/province [12] 0 0
Washington
Country [13] 0 0
Canada
State/province [13] 0 0
Ontario
Country [14] 0 0
France
State/province [14] 0 0
Paris
Country [15] 0 0
Germany
State/province [15] 0 0
Bremen
Country [16] 0 0
Germany
State/province [16] 0 0
Munchen
Country [17] 0 0
Netherlands
State/province [17] 0 0
Rotterdam
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Amicus Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT00688597
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Mathews Adera, MD
Address 0 0
Amicus Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT00688597