E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pulmonary arterial hypertension (PAH) |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 13.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10064911 |
E.1.2 | Term | Pulmonary arterial hypertension |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
|
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 long-term safety and tolerability of QTI571 in patients with severe pulmonary arterial hypertension. |
|
E.2.2 | Secondary objectives of the trial |
- To evaluate the long-term efficacy of QTI571 as measured by the changes in six minute walk distance (6MWD) compared with the measurement in study CQTI571A2102;
- To assess the time to clinical worsening (TTCW) endpoints including all cause mortality,overnight hospitalization for worsening of pulmonary arterial hypertension, worsening of WHO functional class by at least one level, or a drop in six minute walk distance (6MWD)by 15% both as a composite endpoint and by individual time to clinical worsening events;
- To assess the impact of QTI571 on Medical Resource Utilization. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Written informed consent must be obtained before any assessment is performed;
2. Patients who completed in CQTI571A2102 clinical trial including all Study Completion assessments at the end of study visit met the eligibility criteria for that study and did not meet withdrawal criteria for safety reasons during study conduct. |
|
E.4 | Principal exclusion criteria |
1. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant UNLESS they are using two birth control methods. The two methods can be a double barrier method or a barrier method plus a hormonal method. Adequate
barrier methods of contraception include: diaphragm, condom (by the partner),intrauterine device (copper or hormonal), or sponge with spermicide. Hormonal contraceptives include any marketed contraceptive agent that includes an estrogen and/or
a progestational agent.
2. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG
laboratory test (> 5 mIU/mL).
3. Patients with left ventricular ejection fraction (LVEF) < 45%
4. Patients with thrombocytopenia, platelet count < 50 x109/L (50 x 103/μL).
5. Patients with uncontrolled systemic arterial hypertension, systolic pressure > 160 mm Hg or diastolic pressure >90 mm Hg.
6. Patients with a QTcF > 450 ms for males and > 470 ms for females in the absence of right bundle branch block.
7. Fertile males, defined as all males physiologically capable of conceiving offspring UNLESS the subject agrees to comply with two highly effective contraceptive methods comprising a barrier method (condom or occlusive cap plus spermicide) for the entire
duration of the study, up to the Study Completion visit, and refrain from fathering a child for at least three (3) months following the last study drug administration Periodic
abstinence and withdrawal are not acceptable methods of contraception. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Safety monitored via standard safety laboratories evaluations: ECG, echocardiogram; adverse event reporting; physical examination. |
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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 | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 7 |
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 |
|
E.8.7 | Trial has a data monitoring committee | Yes |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |