E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10036143 |
E.1.2 | Term | Pompe's disease |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the safety and tolerability of AT2220 in patients with Pompe disease
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E.2.2 | Secondary objectives of the trial |
• To evaluate the effect of AT2220 on functional parameters of Pompe disease • To evaluate the effect of AT2220 on pharmacodynamic parameters of Pompe disease • To evaluate pharmacokinetics of AT2220
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female, 18 to 74 years of age at time of consent 2. Diagnosis of Pompe disease based on clinical assessment, enzyme assay, and/or genotyping. Confirmatory GAA 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 (See Appendix 2) OR sitting FVC ≥ 30% and < 80% of predicted value, with maximum FVC (L) value reproducible (± 15 % ) between Visits 1 and 2 6. Subjects of reproductive potential agree to use reliable methods of contraception during the study 7. Subject is willing and able to provide written informed consent.
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E.4 | Principal 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 a 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 (e.g., a subject with poor reproducibility of assessments between days -28 and -27 may be excluded at the investigator’s discretion)
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E.5 End points |
E.5.1 | Primary end point(s) |
• Treatment-emergent physical exam changes up to end of study (EOS) visit • Treatment-emergent vital signs (blood pressure, heart rate, respiratory rate) changes up to EOS • Treatment-emergent safety laboratory test (hematology, chemistry, urinalysis) abnormalities up to EOS • Treatment-emergent ECG abnormalities up to EOS • Treatment-emergent adverse events (AEs) up to 24 hours after EOS • Treatment-emergent changes in concomitant medications up to EOS • Adverse events leading to permanent discontinuation of study medication • Serious adverse events (SAEs) up to 28 days after study medication discontinuation
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 28 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 28 |