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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00688597




Registration number
NCT00688597
Ethics application status
Date submitted
30/05/2008
Date registered
3/06/2008
Date last updated
17/08/2018

Titles & IDs
Public title
Study to Evaluate the Safety of AT2220 (Duvoglustat) 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 - Duvoglustat

Experimental: Cohort 1 - Regimen 1: Low-dose duvoglustat (2.5 grams [g]) once a day (QD) for 3 days, followed by no drug for 4 days, for 11 weeks.

Experimental: Cohort 2 - Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.

Experimental: Cohort 3 - Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.


Treatment: Drugs: Duvoglustat
Powder in a bottle for dissolution in water for oral administration
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion Of Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
Baseline, Week 11
Secondary outcome [1] 0 0
Change In 6-minute Walk Test (6MWT) From Baseline To End Of Study
Timepoint [1] 0 0
Baseline, Week 11

Eligibility
Key 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 participants who are
screened for the study

- Naïve to enzyme replacement therapy (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 forced vital capacity =30% and
<80% of predicted value, reproducible between screening and baseline (±15%)

- Participants of reproductive potential agree to use reliable methods of contraception
during the study

- Participant or legal representative is willing and able to provide written informed
consent
Minimum age
18 Years
Maximum age
74 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 milliseconds (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 (such as miglustat or 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 the 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
8/12/2008
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
14/12/2009
Sample size
Target
Accrual to date
Final
3
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia

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 was to determine the safety and tolerability of 3 different
doses of duvoglustat (AT2220) in participants affected by Pompe disease. The study also
evaluated the effects of duvoglustat on functional parameters in Pompe disease.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00688597
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Medical Monitor Clinical Research
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/ct2/show/NCT00688597