| May 30, 2008 |
| November 9, 2009 |
| November 2008 |
| March 2009 (final data collection date for primary outcome measure) |
| Treatment-emergent Adverse Events [ Time Frame: 11 weeks ] [ Designated as safety issue: Yes ] |
| Same as current |
| Complete list of historical versions of study NCT00688597 on ClinicalTrials.gov Archive Site |
| Change in functional parameters from Baseline to End of Study [ Time Frame: 11 weeks ] [ Designated as safety issue: No ] |
| Same as current |
| |
| Study to Evaluate the Safety of AT2220 in Pompe Disease |
| 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 |
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. |
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. |
| Phase II |
| Interventional |
| Treatment, Non-Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety Study |
| Pompe Disease |
| Drug: AT2220 |
- Experimental: AT2220 low dose, regimen 1, for 11 weeks
- Experimental: AT2220 high dose, regimen 1, for 11 weeks
- Experimental: AT2220 high dose, regimen 2, for 11 weeks
|
| |
| |
| Terminated |
| 3 |
| November 2009 |
| March 2009 (final data collection date for primary outcome measure) |
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
|
| Both |
| 18 Years to 74 Years |
| No |
| Contact information is only displayed when the study is recruiting subjects |
| United States, Australia, Canada, France, Germany, Netherlands, United Kingdom |
| |
| NCT00688597 |
| Mathews Adera, MD, Medical Director, Clinical Research, Amicus Therapeutics |
| POM-CL-201 |
| Amicus Therapeutics |
|
| Study Director: |
Mathews Adera, MD |
Amicus Therapeutics |
|
|
| Amicus Therapeutics |
| November 2009 |
