E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Rare and ultra-rare glomerulonephritis |
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E.1.1.1 | Medical condition in easily understood language |
Rare and ultra-rare glomerulonephritis |
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E.1.1.2 | Therapeutic area | Not possible to specify |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10018364 |
E.1.2 | Term | Glomerulonephritis |
E.1.2 | System Organ Class | 10038359 - Renal and urinary 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 |
Assessment of safety and effectiveness desmopressin used before percutaneous needle kidney biopsy for bleeding prevention, in the population of patients with rare and ultra-rare glomerulonephritis. |
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E.2.2 | Secondary objectives of the trial |
Assessment of safety and effectiveness desmopressin used before percutaneous needle kidney biopsy for hospitalization time and AESI frequency. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. ≥ 18 years old 2. Ability to provide Informed Consent 3. Qualification by nephrologist to kidney biopsy in accordance to current standards 4. Initial haemoglobin concentration > 8g/dl and PLT count >100 x103/µL 5. Normal range of APTT and INR 6. Blood pressure control defined as SBP<160 mmHg 7. Permitted antiplatelet/antithrombotic drugs: acetylsalicylic acid and heparin 8. No inflammation at the point of biopsy needle insertion
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E.4 | Principal exclusion criteria |
1. Initial sodium concentration <130mmol/l 2. Pregnancy and breastfeeding 3. Anaphylactic shock after desmopressin administration (medical history) 4. Necessity of administration other anti-platelet / anti thrombotic drugs other than acetylsalicylic acid, non-fractionated heparin or low molecular weight heparin 5. Decompensated Heart failure 7. Von Willebrand disease (VWD) type II B 8. As per Investigator opinion a medical situation which may lead to increased intracranial pressure (ICP) 10. Hydronephrosis of the biopsied kidney 11. Usage of any prohibited drug before screening : • ASA in dosage > 75mg per day • Vitamin K antagonist (VKA) • Direct oral anticoagulants (DOAC) • Low-molecular-weight heparin (LMWH) • Unfractionated heparin (UFH) Except situation when dosage of listed above drugs will be adjusted in accordance to protocol
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E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy primary outcome: 1. Number of major and minor bleeding events in 24 h after the procedure which is defined as: • minor bleeding events: macroscopic haematuria; clinically silent hematoma in ultrasound in 24h after biopsy, decrease in haemoglobin concentration >20% in regards to initial values. • Major bleeding events: transfusion of red cells concentrate, embolisation, nephrectomy, bleeding related directly and indirectly death. Safety primary outcome 1. Hyponatremia 24 and 48 h after biopsy
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Efficacy primary outcome: 24 hours after biopsy procedure Safety prrimary outcome: 24 and 48 after biopsy |
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E.5.2 | Secondary end point(s) |
Efficacy secondary outcome :
1. Hospitalization time from kidney biopsy till discharge 2. Cost-effectiveness analysis of desmopressin use Safety secondary outcome Frequency of AEs of special interest: • Tachycardia HR>100 BPM • Hypotension defined as SBP <90 mmHg or MAP <65 mgHg or a systolic blood pressure decrease of 40 mmHg. • Any thrombotic events
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Hospitalization time from kidney biopsy till discharge. |
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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 | 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 | 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 | 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 | 5 |
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |