E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pulmonary sarcoidosis associated with cough |
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E.1.1.1 | Medical condition in easily understood language |
Pulmonary sarcoidosis associated with cough |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10037430 |
E.1.2 | Term | Pulmonary sarcoidosis |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
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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 assess efficacy of Aviptadil in comparison to placebo on cough-specific QOL |
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E.2.2 | Secondary objectives of the trial |
-To assess efficacy of Aviptadil in comparison to placebo on cough-specific QoL -To assess efficacy of Aviptadil in comparison to placebo on sarcoidosis specific QoL, on fatigue, on dyspnea on exertion, on patient’s assessment of cough intensity, on parameters of lung function. |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
To assess Transcription patterns |
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E.3 | Principal inclusion criteria |
1. Adult male and female patients ≥ 18 years of age 2. Patient with sarcoidosis and associated chronic cough (LCQ score < 16), any Scadding type, any other organ manifestation as long as it does not require prednisolone therapy > 15 mg, see exclusion criteria 3) 3. Body mass index (BMI) <30 kg/m2 4. Patients supposed to be kept on stable medication for the whole study period 5. Written informed consent obtained according to international guidelines and local laws 6. Negative results for HIV, hepatitis A, B and C within three months prior to study inclusion 7. Ability to understand the nature of the trial and the trial related procedures in German and to comply with them 8. Ability to use the nebulizer in a proper manner 9. Ability to store the IMP according to requirements
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E.4 | Principal exclusion criteria |
1. Patients suffering from other organic causes of cough (e.g. respiratory tract infections, gastroesophageal reflux disease, heart failure, bronchitis associated with any other disorder but sarcoidosis, thoracic tumors, exposure to inhalable irritants, laryngitis etc) 2. Patients treated with ACE-inhibitors or a history of ACE-inhibitor treatment within the last six weeks (ACE-inhibitors are also not allowed to start during the study) 3. Corticosteroids doses > 15 mg prednisolone equivalent per day 4. Immunosuppressive treatment (except for corticosteroids ≤ 15 mg prednisolone equivalent per day) within 3 months prior randomisation or indications to start or intensify immunosuppressive therapy during the study 5. Patients suffering from other chronic or acute severe diseases besides sarcoidosis manifestations that might impair safe participation in the trial according to the treating physician, e.g.: - Chronic kidney disease (CKD) stage 4 and 5 - Liver cirrhosis Child-Pugh score B and C - Intended surgery of heart, lung, liver or abdominal disease during the trial period - Active malignancies requiring chemo- and / or radiotherapy - Chronic bowel diseases not sufficiently controlled 6. Conditions or medications supposed to influence the primary or secondary outcomes according to the local investigator’s appraisal (e.g. newly diagnosed obstructive sleep apnoea syndrome, newly or recently exacerbated psychiatric disease, modification of psychotic disease) 7. Known HIV infection, infectious hepatitis (type A, B or C) or another currently uncontrolled infection 8. Known hypersensitivity to the active substance or any of the excipients 9. Participation in any other interventional clinical trial during this study or during the last 30 days prior to informed consent; simultaneous participation in registry and diagnostic trials is allowed 10. Previous participation in this trial 11. Known persistent abuse of medication, drugs or alcohol 12. Relationship of dependence/employment with the sponsor or the investigator 13. Current or planned pregnancy, nursing period or patients of reproductive potential who are not using effective birth control methods 14. Male subjects with reproductive potential who refuse to use adequate means of contraception during and up to 90 days after stopping treatment with Aviptadil
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E.5 End points |
E.5.1 | Primary end point(s) |
Change in LCQ score from baseline until 24 weeks after randomisation |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
24 weeks after randomisation |
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E.5.2 | Secondary end point(s) |
- Change in LCQ score from baseline until 12 weeks after randomisation -Change in KSQ score from baseline to 12 and 24 weeks after randomisation - Fatigue assessment scale (FAS) week 12 and 24 -Multidimensional fatigue inventory (MFI) week 12 and 24 -Epworth Sleepiness Scale (ESS) week 12 and 24 -Distance-saturation-product (DSP) week 12 and 24 -VAS for cough week 12 and 24 -Lung function parameters at 12 and 24 weeks after randomisation -Cumulative steroid dose up to 24 weeks -6-minute walking distance and distance saturation product at 12 and 24 weeks |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
-week 12 after randomisation -week 12 and 24 after randomisation |
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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 | 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 | 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 | 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 | Yes |
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 | 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
| 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 | 10 |
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 | 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
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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 | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |