E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Childhood idiopathic nephrotic syndrome (NS) |
Idiopatisk nefrotisk syndrom |
|
E.1.1.1 | Medical condition in easily understood language |
NS is characterized by leaking of protein from the kidneys into the urine. PLasma levels of protein decrease and weight gain and severe edema is observed |
NS viser sig ved at nyrerne bliver utætte og der tabes protein i urinen. Derfor bliver koncentrationen af protein lav i urinen og patienter oplever vægtøgning og svære hævelser. |
|
E.1.1.2 | Therapeutic area | Not possible to specify |
MedDRA Classification |
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 |
NS is characterized by severe weight gain and edema formation. Recently, it was justified that the edema was a result of activation of the epithelial sodium channel (ENaC) in the kidneys and ENaC is blocked by amilorid.
The objective of this study is to investigate if amilorid is superior to placebo treating the edema observed in NS |
NS er karakteriseret ved vægtøgning og svære hævelser pga aktivering af saltkanalen ENaC i nyrernes samlerør. ENaC kan blokkeres af amilorid. Formålet me dette studier er at undersøge om amilorid er bedre end placebo til at behandle ødemerne ved NS |
|
E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- First episode of NS
- Age 1 to 15 yrs |
- Debut af NS
- Ml 1 og 15 år |
|
E.4 | Principal exclusion criteria |
- Relapse of NS or secondare NS
- Fever
- Dehydration
- Treatment with prednisolon/diuretics 1 mth prior inclusion
- PLasma K above 5,5 mmol/l
- Decreased renal function
- Known allergy to amilorid
- Pregnancy |
- Relapse af NS
- Sekundær NS
- Febrilia, temperatur > 38°.
- Dehydratio
- Behandling med prednisolon eller vandrivende 1 mdr. før inklusion.
- Plasma K+ højere end 5,5 mmol/L
- Nedsat nyrefunktion
- Kendt allergi for indholdsstoffer i forsøgslægemidlet
- Graviditet. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Patients weight (kg) and sodium concentration in urine (mmol/L) |
Patienternes vægt (kg) samt urinens koncentration af salt (mmol/L) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Day 1, 5, 10 and 30 |
Dag 1, 3, 5 og 30 |
|
E.5.2 | Secondary end point(s) |
. Edema scores
- Renin/aldosteron in blood/urine
- PLasminogen/plasmin in urine
- uPA concenration in urine
Amilorid concentration in urine
- Urines ability to genere current in patch clamp experiments |
• Ødem og VAS scorer
• Renin og aldosteron koncentration i blod samt aldosteron i urin
• Plasminogen/plasmin koncentration i urin
• uPA uPA aktivitet i urin
• Koncentrationen af amilorid i urinen
• Urins evne til at genererer strøm i patch clampeksperimenter
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Day 1, 3, 5 and 30 |
dag 1, 3, 5 og dag 30 |
|
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) | 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 | 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 | 9 |
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
|
Dont know what LVLS means |
The end of the trial is when the last subject (n=40) is included and has runout the study period of 30 days |
|
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 | |