E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated | |
E.1.1.1 | Medical condition in easily understood language |
Ventricular septal defects are defined as a hole in the heart wall between the two pumping chambers of the heart. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10047298 |
E.1.2 | Term | Ventricular septal defect |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The overall purpose of this study is to test whether β2-agonists will affect the cardiopulmonary exercise capacity of VSD-operated patients compared with un-operated VSD-patients and healthy age- and gender-matched controls |
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E.2.2 | Secondary objectives of the trial |
Furthermore we will investigate if the β2-agonists will affect the pulmonary function and heart-rate-variability of VSD-operated patients compared with un-operated VSD-patients and healthy age- and gender-matched controls. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
≥18 years of age and legally competent to vouch for their own study participation.
Informed and written consent for participation in this trial.
Trial group 1: Surgically corrected for isolated VSD between 1990 and 1998 at Aarhus University Hospital.
Trial group 2: Diagnosed with isolated VSD born between 1977 and 1998 without surgical or percutaneous closure. - Verified by Echocardiography within the last 4 years. If it is more than 4 it will be verified by our doctors as a systolic murmur or echocardiography.
Trial group 3: 18-40 years, with no known medical records of heart and lung disease.
Restrain from strenuous leg exercise 24 hours before inclusion. |
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E.4 | Principal exclusion criteria |
Lack of medical record.
Pregnancy. - Participants will be asked if they are using contraceptives and be told to continue this during the trial and for at least 30 hours (5 times T2) after their last visit. If they are not using spiral or valid contraceptives (contraceptive pills, implants, transdermal patches, vaginal ring or injections) one of our medical experts will judge if the participant is able to undergo the trial. They will likewise be informed to withstand from sexual intercourse during the trial until 30 hours after the second visit.
Currently breastfeeding.
Syndromes, such as Down’s.
Mentally or physically incompetent to perform the ergometer bicycle test.
Thyrotoxicosis.
Pre-trial medical record of arrhythmias except right bundle branch block.
Asthma or other known β2-responsive conditions.
Coronary heart disease.
Severe pulmonary disease.
Diabetes.
Use of the following medication: Xantin-derivates, steroids, diuretics, ipratropium.
Allergy to the active ingredients of Ventoline: Salbutamolsulphate, benzalkoniumchloride |
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E.5 End points |
E.5.1 | Primary end point(s) |
Peak minute ventilation (ml min-1) Peak exercise oxygen uptake (ml O2 kg-1 min-1) Peak workload |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Last patient is scheduled to be included no later than september 30th 2017. After this data analysis will commence. |
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E.5.2 | Secondary end point(s) |
Lung clearance index: LCI 2,5 - Scond - Sacin
Plethymography: TLC, RV, FRCpleth, sRAW,
Spirometry: FEV1 FVC, FEV1/FVC
Diffusion capacity: DLCO, VA (alveolar volume),
Impulse Oscillometry: Diff 5-20
Heart-rate variability: HRV, mean RR interval, RMSSD, arrhythmias discovered
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Last patient is scheduled to be included no later than september 30th 2017. After this data analysis will commence. |
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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 | Yes |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Further evaluation of the cause for lower cardiopulmonary function in VSD operated patients. |
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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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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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Patient recruitment will end September 30th 2017. After this data analysis will commence. If the trial have not included the 90 participants as expected we extend the recruitment period and apply for permits from the legal departments. |
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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 | |
E.8.9.1 | In the Member State concerned days | |