E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cystic fibrosis |
Cystic fibrose |
|
E.1.1.1 | Medical condition in easily understood language |
mucoviscoidosis |
taaislijmziekte |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
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 |
The primary study endpoint is the ivacaftor through concentration. |
Het primaire eindpunt is de dalspiegel van ivacaftor |
|
E.2.2 | Secondary objectives of the trial |
- tezacaftor and lumacaftor through concentrations
- hydroxymethyl-ivacaftor and ivacaftor-carboxylate concentrations
- ivacaftor, tezacaftor and lumacaftor concentration at T-max |
- dalspiegels van tezacaftor en lumacaftor
- concentraties van hydroxymethyl-ivacaftor en ivacaftor-carboxylaat
- concentraties op T-max (optional part of the study) ivacaftor, tezacaftor en lumacaftor (topspiegels) |
|
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 |
Kinetics of ivacaftor at Switch Orkambi Symkevi (SOS)
Optional study: ivacaftor, tezacaftor and lumacaftor concentration at T-max |
Kinetiek van ivacaftor tijdens Switch Orkambi Symkevi (SOS)
Optioneel onderzoek: ivacaftor, tezacaftor en lumacaftor concentratie op T-max (topspiegel) |
|
E.3 | Principal inclusion criteria |
• The subject is homozygous for the F508del mutation in the CFTR gene.
• The subject is aged 18 years or older.
• The subject used Orkambi continuous for at least a month before enrolling in the study.
• The subject used Orkambi in a dose of twice-daily 2 tablets of 200/125 mg lum/iva.
• The subject will switch from Orkambi therapy to Symkevi.
• The subject will initiate Symkevi therapy with a dosage of once-daily 1 tablet of 100/150 mg TEZ/IVA and once-daily 1 tablet of 150 mg IVA
• The subject has signed and dated a written informed consent. |
• de proefpersoon heeft een homozygote F508del mutatie in het CFTR gen
• de proefpersoon is 18 jaar of ouder
• de proefpersoon heeft voor inclusie in het onderzoek gedurende ten minste 1 maand continue Orkambi gebruikt
• de proefpersoon heeft een dosering van twee maal per dag 2 tabletten van 200/125 mg LUM/IVA gebruikt
• de proefpersoon zal overstappen van Orkambi (LUM/IVA) naar Symkevi (TEZ/IVA)
• de proefpersoon zal de Symkevi medicatie aanvangen met een dosering van 1 maal per dag 100/150 mg TEZ/IVA en 1 maal per dag 150 mg IVA |
|
E.4 | Principal exclusion criteria |
• The subject is deemed unfit to participate in this study by the treating physician.
• The subject is experiencing an exacerbation needing treatment with IV antibiotics |
• The subject is door de behandelaar als 'onfit voor deelname aan het onderzoek' aangemerkt
• The subject heeft een pulmonale exacerbatie waarvoor behandeling met intraveneuze antibiotica noodzakelijk is, |
|
E.5 End points |
E.5.1 | Primary end point(s) |
- The primary study endpoint is the ivacaftor through concentration. |
Het primaire eindpunt is de dalspiegel van ivacaftor |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
After at least one month of treatment with Orkambi, just before subject is supposed to take the next morning dose Orkambi
After at least one month of treatment with Symkevi, just before subject is supposed to take the next morning dose Symkevi |
Na ten minste 1 maand gebruik van Orkambi, op het moment dat patiënt de volgende ochtenddosis Orkambi moet innemen.
Na ten minste 1 maand gebruik van Symkevi, op het moment dat patiënt de volgende ochtenddosis Symkevi moet innemen |
|
E.5.2 | Secondary end point(s) |
- tezacaftor and lumacaftor through concentrations
- hydroxymethyl-ivacaftor and ivacaftor-carboxylate concentrations
- ivacaftor, tezacaftor and lumacaftor concentration at T-max |
- dalspiegels van tezacaftor en lumacaftor
- concentraties van hydroxymethyl-ivacaftor en ivacaftor-carboxylaat
- concentraties op T-max (optional part of the study) ivacaftor, tezacaftor en lumacaftor (topspiegels) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
After at least one month of treatment with Orkambi, 3-5 hours after subject has taken morning dose Orkambi
After at least one month of treatment with Symkevi, 3-5 hours after subject has taken morning dose Symkevi
|
Na ten minste 1 maand gebruik van Orkambi, 3-5 uur nadat de proefpersoon een ochtenddosis Orkambiheeft ingenomen.
Na ten minste 1 maand gebruik van Symkevi, 3-5 uur nadat de proefpersoon een ochtenddosis Symkevi heeft ingenomen. |
|
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 | 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 | Yes |
E.6.11 | Pharmacogenomic | Yes |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
observationeel |
observational |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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 | 2 |
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | |