natgillin/translations-raw huggingface dataset:
After this, the design of the study was changed so that the patients received NutropinAq given either three times a week or once a day.
Nato je bila zasnova študije spremenjena tako, da so bolniki prejemali zdravilo
Annual reports to be submitted as part of the annual reassessment
Letno poročilo se predloži kot del letne ponovne ocene
ATC code: A16AB15.
Oznaka ATC: A16AB15.
Patients n=33
Bolniki n = 33
The reconstituted vials should stand on the table for about 5-10 minutes.
Rekonstituirane viale morajo stati na mizi približno 5-10 minut.
Date of first authorisation: 23/03/2018
Datum prve odobritve: 23/03/2018
The following information is intended for healthcare professionals only.
Naslednje informacije so namenjene samo zdravstvenemu osebju:
Quality of Life
Kakovost življenja
This being an approval under exceptional circumstances and pursuant to Article 14(8) of Regulation (EC) No 726/2004, the MAH shall conduct, within the stated timeframe, the following measures:
Ker gre za odobritev v izjemnih okoliščinah in ob upoštevanju člena 14(8) Uredbe (ES) št. 726/2004, mora imetnik dovoljenja za promet v določenem časovnem okviru izvesti naslednje ukrepe:
Lamzede Last Observation
Lamzede Zadnje opazovanje
This is known as an infusion-related reaction and can sometimes be severe.
To se imenuje reakcija, povezana z infuzijo, ki je včasih lahko resna.
Thereafter each vial should be tilted and rolled gently for 15-20 seconds to enhance the dissolution process.
Nato je treba vsako vialo nagniti in 15-20 sekund nežno valjati, da se izboljša postopek raztapljanja.
Lamzede 12 months
Lamzede 12 mesecev
In the most severe cases, the current medical standards for emergency treatment are to be observed.
V najresnejših primerih je treba upoštevati trenutne zdravstvene standarde za nujno zdravljenje.
See sections 4.4 and 5.1.
Glejte poglavji 4.4 in 5.1.
This leaflet was last revised in {MM/YYYY}.
Navodilo je bilo nazadnje revidirano {MM/LLLL}.
(<1/10,000) or not known (cannot be estimated from the available data).
< 1/1.000), zelo redki (< 1/10.000) ali neznana (ni mogoče oceniti iz razpoložljivih podatkov).
All patients who experienced IRRs recovered.
Vsi bolniki, ki so imeli IRR, so okrevali.
There is a potential for immunogenicity.
Obstaja možnost imunogenosti.
This may improve the symptoms of the disease.
Z njim lahko izboljšamo simptome bolezni.
Lamzede may be given to children and adolescents at the same dose and frequency as in adults.
Zdravilo Lamzede se lahko daje otrokom in mladostnikom z enakim odmerkom in pogostnostjo kot pri odraslih.
Only the volume corresponding to the recommended dose should be administered.
Aplicirati je treba samo količino, ki ustreza priporočenemu odmerku.
When this enzyme is missing or does not work properly, these sugar compounds build up inside cells and cause the signs and symptoms of the disease.
Ko tega encima primanjkuje ali ko ne deluje pravilno, se te sestavine sladkorja kopičijo v celicah in povzročajo znake in simptome bolezni.
Pre-treatment with antihistamines and/or corticosteroids may prevent subsequent reactions in those cases where symptomatic treatment was required.
Predhodno zdravljenje z antihistaminiki in/ali kortikosteroidi lahko prepreči kasnejše reakcije v primerih, ko je bilo potrebno simptomatsko zdravljenje.
Patients with the most severe rapidly progressing phenotype (with a deterioration within one year and central nervous system involvement) were excluded.
Bolniki z najresnejšim, hitro napredujočim fenotipom (poslabšanje v enem letu in vpletenost osrednjega živčnega sistema) so bili izključeni.
As no patients older than 41 years have been identified across Europe, no relevant use in elderly patients is expected.
Ker v Evropi nismo našli bolnikov, starejših od 41 let, ne pričakujemo potrebne uporabe pri starejših bolnikih.
Aseptic technique is to be used during preparation.
Med pripravo je treba uporabljati aseptično tehniko.
The effects of velmanase alfa were more evident in patients younger than 18 years.
Učinki velmanaze alfa so bili bolj očitni pri bolnikih, mlajših od 18 let.
(1) MCID: minimal clinically important difference
Nobeno področje (1) MCID: najmanjša klinično pomembna razlika (minimal clinically important difference)
Patients were diagnosed based on alpha-mannosidase activity <10% of normal activity in blood leukocytes.
Bolniki so bili diagnosticirani na podlagi aktivnosti alfamanozidaze < 10 % običajne aktivnosti v krvnih levkocitih.
Loss of consciousness
Izguba zavesti
a) The number of vials to be used should be calculated based on the individual patient's weight.
a) Potrebno število vial za uporabo je treba preračunati na podlagi telesne mase posameznega bolnika.
In patients over 18 years old a stabilisation has been demonstrated.
Pri bolnikih, starejših od 18 let, je bila dokazana stabilizacija.
If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before this medicine is used.
Nosečnost in dojenje Če ste noseči ali dojite, menite, da bi lahko bili noseči ali načrtujete zanositev, se posvetujte z zdravnikom, preden dobite to zdravilo.
The development of antibodies has not been shown to affect clinical efficacy or safety.
Ni bilo dokazano, da bi razvoj protiteles vplival na klinično učinkovitost ali varnost.
In case the patient's condition deteriorates during ERT, cessation of treatment should be considered.
Če se bolnikovo stanje med ERT poslabša, je treba razmisliti o prenehanju zdravljenja.
Chemical and physical in-use stability has been demonstrated for 24 hours at 2°C - 8°C.
Kemična in fizikalna stabilnost med uporabo je bila dokazana 24 ur pri temperaturi 2 °C- 8 °C.
If the number of calculated vials includes a fraction, it should be rounded up to the next whole number.
Če število izračunanih vial ni celo število, ga je treba zaokrožiti navzgor na naslednje celo število.
As the accumulation of end organ damage progresses over time, it is more difficult for the treatment to reverse the damage or to show improvements.
Ker kopičenje poškodb tarčnega organa sčasoma napreduje, je pri zdravljenju težje izničiti poškodbe ali pokazati izboljšanje.
Placebo 12 months
Placebo 12 mesecev
In total 15 patients (7 paediatrics and 8 adults) received active treatment and 10 patients received placebo (5 paediatrics and 5 adults).
Skupaj 15 bolnikov (7 pediatričnih in 8 odraslih) je prejemalo aktivno zdravljenje, 10 bolnikov pa je prejemalo placebo (5 pediatričnih in 5 odraslih).
Each carton contains 1, 5 or 10 vials.
Vsaka škatla vsebuje 1, 5 ali 10 vial.
For further instructions, see section 4.4.
Za nadaljnja navodila glejte poglavje 4.4.
The vials should reach ambient temperature (between 15°C and 25°C) prior to reconstitution.
Viale morajo pred rekonstitucijo doseči sobno temperaturo (med 15 °C in 25 °C).
The results observed in patients below 18 years of age showed an improvement.
Rezultati, ki so jih opazili pri bolnikih, mlajših od 18 let, so pokazali izboljšanje.
For patients weighing less than 18 kg, and receiving less than 9 mL reconstituted solution, the infusion rate should be calculated so that the infusion time is ≥50 minutes.
Za bolnike, ki tehtajo manj kot 18 kg in prejemajo manj kot 9 ml rekonstituirane raztopine, je treba hitrost infundiranja preračunati tako, da je čas infundiranja ≥ 50 minut.
Severe allergic reaction to the active substance or to any of the excipients listed in section 6.1.
Huda alergijska reakcija na učinkovino ali katero koli pomožno snov, navedeno v poglavju 6.1.
Lamzede has no or negligible influence on the ability to drive and use machines.
Zdravilo Lamzede nima vpliva ali ima zanemarljiv vpliv na sposobnost vožnje in upravljanja strojev.
This means that due to the rarity of the disease, it has not been possible to obtain complete information on this medicinal product.
To pomeni, da zaradi redkosti bolezni ni bilo mogoče pridobiti vseh podatkov o zdravilu.
- If you have an infusion-related reaction you may be given additional medicines to treat or help prevent future reactions.
- Če doživite reakcijo, povezano z infuzijo, lahko dobite dodatna zdravila za zdravljenje ali preprečevanje reakcij v prihodnje.
The infusion duration should be a minimum of 50 minutes.
Infundiranje mora trajati najmanj 50 minut.
- if you are allergic to velmanase alfa or any of the other ingredients of this medicine (listed in section 6).
- če ste alergični na velmanazo alfa ali katero koli sestavino tega zdravila (navedeno v poglavju 6).
A slower infusion rate may be prescribed when clinically appropriate according to the physician's judgment, for example at the beginning of the treatment or in case of previous infusion-related reactions (IRRs).
Nižjo hitrost infundiranja se lahko predpiše, če je to po zdravnikovi presoji klinično primerno, na primer na začetku zdravljenja ali v primeru predhodnih reakcij, povezanih z infuzijo (IRR-infusion-related reactions).
- Most of the time you can still be given Lamzede even if you experience an infusion-related reaction.
- Večinoma lahko zdravilo Lamzede prejemate še naprej, čeprav doživite reakcijo, povezano z infuzijo.
Due to the nature of the medicinal product, the reconstituted solution may occasionally contain some proteinaceous particles in form of thin white strands or translucent fibers which will be removed by the in-line filter during infusion (see point e).
Zaradi narave zdravila lahko rekonstituirana raztopina občasno vsebuje proteinske delce v obliki tankih belih lis ali prosojnih vlaken, ki bodo odstranjeni z linijskim filtrom med samim infundiranjem (glejte točko e).
Acute renal failure was of moderate severity leading to temporary discontinuation of the study treatment and fully resolved within 3 months.
Akutna odpoved ledvic je bila zmerne resnosti in je povzročila začasno prekinitev zdravljenja v študiji, popolnoma pa je izzvenela v 3 mesecih.
Patients
Bolniki
As expected for a protein of this size, the steady-state volume of distribution was low (0.27 L/kg), indicating distribution confined to plasma.
Kot pričakovano za protein te velikosti, je bil volumen porazdelitve v stanju dinamičnega ravnovesja majhen (0,27 l/kg), kar kaže, da je porazdelitev omejena na plazmo.
Most side effects occur during the infusion or shortly after (“infusion-related reaction”, see section 2 Warnings and precautions).
Večinoma se neželeni učinki pojavijo med infundiranjem ali kmalu po njem („reakcija, povezana z infuzijo“, glejte poglavje 2 Opozorila in previdnostni ukrepi).
Paediatric population No dose adjustment is necessary for the paediatric population.
Pediatrična populacija Prilagajanje odmerka pri pediatrični populaciji ni potrebno.
A pharmacodynamic effect with statistically significant decrease of serum oligosaccharides in comparison to placebo was demonstrated.
Dokazan je bil farmakodinamični učinek s statistično pomembnim zmanjšanjem oligosaharidov v serumu v primerjavi s placebom.
Infusion-related reaction
Reakcija, povezana z infuzijo
4.7 Effects on ability to drive and use machines
4.7 Vpliv na sposobnost upravljanja vozil in strojev
Minimum infusion time (min)
Najkrajši čas infundiranja (min)
Overall response
Splošni odziv
rhLAMAN-10 study n=33
Študija rhLAMAN-10 N = 33
All were mild or moderate in severity and none were reported as a serious adverse event.
Vse so bile blage do zmerne resnosti in o nobeni niso poročali kot o resnem neželenem učinku.
In such a case, treatment with an antihistamine and corticosteroids should be considered as being appropriate.
V takem primeru je treba razmisliti o primernem zdravljenju z antihistaminiki in kortikosteroidi.
Do not use this medicine after the expiry date which is stated on the label and the carton after ‘EXP'.
Tega zdravila ne smete uporabljati po datumu izteka roka uporabnosti, ki je naveden na nalepki in na škatli poleg oznake „Uporabno do/EXP“.
IRRs include hypersensitivity in 3 patients and anaphylactoid reaction in 1 patient.
IRR vključujejo preobčutljivost pri 3 bolnikih in anafilaktoidno reakcijo pri 1 bolniku.
The maximum infusion rate is 25 mL/hour (see section 4.2).
Največja hitrost infundiranja je 25 ml/uro (glejte poglavje 4.2).
An integrated database was created by pooling cumulative databases from all studies with velmanase alfa.
Celovito zbirko podatkov so ustvarili z združevanjem skupnih zbirk podatkov iz vseh študij z velmanazo alfa.
*Velmanase alfa is produced in mammalian Chinese Hamster Ovary (CHO) cells using recombinant DNA technology.
*Velmanaza alfa se proizvaja v sesalskih ovarijskih celicah kitajskega hrčka (CHO-Chinese hamster ovary) s pomočjo tehnologije rekombinantne DNA.
The management of IRRs should be based on the severity of the reaction and includes slowing the infusion rate, treatment with medicinal products such as antihistamines, antipyretics and/or corticosteroids, and/or stopping and resuming treatment with increased infusion time.
Obvladovanje IRR mora temeljiti na resnosti reakcij in vključuje: zmanjšanje hitrosti infundiranja, zdravljenje z zdravili, kot so antihistaminiki, antipiretiki in/ali kortikosteroidi, in/ali ustavitev in nadaljevanje zdravljenja z daljšim časom infundiranja.
Treatment with velmanase alfa for 12 months (n=15)
Zdravljenje z velmanazo alfa 12 mesecev (n = 15)
Metabolism and nutrition disorders Increased appetite
Presnovne in prehranske motnje povečan apetit
For infusion rate see section “Method of administration”.
Za hitrost infundiranja glejte poglavje „Način uporabe“.
The most common adverse reactions observed were weight increase (18%), IRRs (9%), diarrhoea (12%), headache (9%), arthralgia (9%), increased appetite (6%) and pain in extremity (6%).
Najbolj pogosto opaženi neželeni učinki so bili povečanje telesne mase (18 %), IRR (9 %), driska (12 %), glavobol (9 %), artralgija (9 %), povečanje apetita (6 %) in bolečine v okončinah (6 %).
A post-hoc multiparametric responders analysis supports the benefit of longer treatment with velmanase alfa in 87.9% of responders in at least 2 domains at last observation (table 4).
Analiza post-hoc multiparametričnih odzivnih oseb podpira korist daljšega zdravljenja z velmanazo alfa pri 87,9 % odzivnih oseb na vsaj 2 področjih pri zadnjem opazovanju (preglednica 4).
The patient should be observed for IRRs for at least one hour after the infusion according to clinical conditions and the physician's judgment.
Bolnika je treba opazovati za IRR najmanj eno uro po infundiranju, v skladu s kliničnimi pogoji in po zdravnikovi presoji.
The effects of treatment with velmanase alfa should be periodically evaluated and discontinuation of treatment considered in cases where no clear benefits could be observed.
Učinke zdravljenja z velmanazo alfa je treba redno ocenjevati in razmisliti o prenehanju zdravljenja, če ni mogoče opaziti nedvomnih koristi.
Data suggest that the beneficial effects of the treatment with velmanase alfa diminish with the increase of disease burden and disease-related respiratory infections.
Podatki kažejo, da koristni učinki zdravljenja z velmanazo alfa izginjajo z večanjem bremena bolezni in okužb dihal, povezanih z boleznijo.
The vial should not be inverted, swirled, or shaken.
Viale ni dovoljeno obračati, vrteti ali stresati.
Nevertheless, the absorption of any ingested milk-containing velmanase alfa in the breastfed child is considered to be minimal and no untoward effects are therefore anticipated.
Vendar pa prevladuje mnenje, da je absorpcija zaužitega mleka, ki vsebuje velmanazo alfa, pri dojenem otroku minimalna, zato neželeni učinki niso pričakovani.
Proteins larger than 50,000 Da, such as velmanase alfa, are not eliminated renally.
Proteini, ki so večji od 50.000 Da, kot je velmanaza alfa, se ne izločajo prek ledvic.
The patient later experienced epileptic seizures that were considered not related.
Bolnik je imel pozneje epileptične napade, ki verjetno niso bili povezani.
From the total reconstituted volume, only 22 mL (corresponding to 44 mg) should be administered.
Od skupne količine za rekonstitucijo je treba aplicirati samo 22 ml (ustreza 44 mg).
Treatment with placebo for 12 months (n=10)
Zdravljenje s placebom 12 mesecev (n = 10)
f) When the last syringe is empty, the dosage syringe is replaced with a 20 mL syringe filled with sodium chloride 9 mg/mL (0.9%) solution for injection.
f) Ko je zadnja brizga prazna, se odmerno brizgo zamenja z 20-ml brizgo, napolnjeno z 9 mg/ml (0,9 %) raztopine natrijevega klorida za injiciranje.
3 years.
50 minut.
Patient's weight (44 kg) × dose (1 mg/kg) = Patient dose (44 mg) 44 mg divided by 10 mg/vial = 4.4 vials, therefore, 5 vials should be reconstituted.
Bolnikova telesna masa (44 kg) × odmerek (1 mg/kg) = odmerek za bolnika (44 mg) 44 mg deljeno z 10 mg/vialo = 4,4 viale, torej je treba rekonstituirati 5 vial.
Administration of velmanase alfa may result in an IRR, including anaphylactoid reaction (see section 4.8).
Uporaba velmanaze alfa lahko povzroči IRR, vključno z anafilaktoidno reakcijo (glejte poglavje 4.8).
- If the infusion-related reactions are severe and/or there is a loss of effect from this medicine, your doctor will perform a blood test to check for antibodies that might affect the outcome of your treatment.
- Če so reakcije, povezane z zdravilom, resne in/ali zdravilo izgubi svoj učinek, vam bo zdravnik opravil krvne preiskave za preverjanje prisotnosti protiteles, ki lahko vplivajo na rezultat zdravljenja.
As velmanase alfa aims at normalizing alpha-mannosidase in alpha-mannosidosis patients, Lamzede should be used during pregnancy only when strictly needed.
Ker je cilj velmanaze alfa normalizacija alfa-manozidaze pri bolnikih z alfa manozidozo, je treba zdravilo Lamzede med nosečnostjo uporabljati le, ko je nujno potrebno.
The patient received saline infusion in a hospital setting and was then discharged after 6-hour observation.
Bolnik je v bolnišnici prejel infuzijo fiziološke raztopine, nato pa je bil po 6-urnem opazovanju odpuščen.
In the clinical studies, one patient experienced acute renal failure considered possibly related to the study treatment.
V kliničnih študijah je eden od bolnikov doživel akutno odpoved ledvic, ki bi lahko bila povezana z zdravljenjem v študiji.
Other medicines and Lamzede
Druga zdravila in zdravilo Lamzede
If severe allergic or anaphylactic-type reactions occur, immediate discontinuation of velmanase alfa is recommended and current medical standards for emergency treatment are to be followed.
Če pride do hude alergijske ali anafilaktične reakcije, priporočamo takojšnjo prekinitev zdravljenja z velmanazo alfa, poleg tega pa je treba upoštevati trenutne zdravstvene standarde za nujno zdravljenje.
For the management of adverse reactions, see sections 4.4 and 4.8.
Za obvladovanje neželenih učinkov glejte poglavji 4.4 in 4.8.
A total of 2 serious adverse reactions (loss of consciousness in 1 patient and acute renal failure in 1 patient) were observed.
Opazili so skupno 2 resna neželena učinka (izguba zavesti pri 1 bolniku in akutna odpoved ledvic pri 1 bolniku).
The safety profile of velmanase alfa in clinical studies involving children and adolescents was similar to that observed in adult patients.
Pediatrična populacija Varnostni profil velmanaze alfa v kliničnih študijah, ki vključujejo otroke in mladostnike, je bil podoben kot pri odraslih bolnikih.
If not used immediately, in-use storage times and conditions prior to use are the responsibility of the user and would normally not be longer than 24 hours at 2°C to 8°C.
Če ga ne porabite takoj, je za čas shranjevanja med uporabo in pogoje shranjevanja pred uporabo odgovoren uporabnik, vendar shranjevanje običajno ne sme presegati 24 ur pri temperaturi od 2 °C do 8 °C.
This medicine is only to be used under the supervision of a doctor experienced in the treatment of alpha-mannosidosis or other similar diseases and should only be given by a healthcare professional.
Zdravilo se lahko uporablja samo pod nadzorom zdravnika, ki je usposobljen za zdravljenje alfa manozidoze ali drugih podobnih bolezni in ga lahko dajejo samo zdravstveni delavci.
If you experience an infusion-related reaction, you must tell your doctor immediately.
Če doživite reakcijo, povezano z infuzijo, se morate nemudoma posvetovati z zdravnikom.
Use of velmanase alfa in the age group 6 to 17 years is supported by evidence from clinical studies in paediatric (19 out of 33 patients) and adult patients.
Uporaba velmanaze alfa v starostni skupini od 6 do 17 let je podprta z dokazi iz kliničnih študij pri pediatričnih (19 od 33 bolnikov) in odraslih bolnikih.
No treatment was administered.
Bolnika niso zdravili.
If the volume of the solution exceeds one syringe capacity, the required number of syringes should be prepared in order to replace the syringe quickly during the infusion.
Če količina raztopine presega prostornino brizge, je treba pripraviti potrebno število brizg, da omogočite hitro zamenjavo brizge med infundiranjem.
What Lamzede is and what it is used for What you need to know before Lamzede is used How Lamzede is used
Kaj je zdravilo Lamzede in za kaj ga uporabljamo Kaj morate vedeti, preden boste prejeli zdravilo Lamzede Kako uporabljati zdravilo Lamzede
This medicine must not be used if the reconstituted solution contains opaque particles or is discoloured.
Tega zdravila ne smete uporabiti, če rekonstituirana raztopina vsebuje motne delce ali če je obarvana.
The maximum dose of velmanase alfa in clinical studies was a single administration of 100 units/kg (approximately corresponding to 3.2 mg/kg).
Največji odmerek velmanaze alfa v kliničnih študijah je bil enkratni odmerek 100 enot/kg (ustreza približno 3,2 mg/kg).
(1) Preferred terms considered as IRR as described in the section below (2) Selected adverse reaction as described in the section below
(1) Prednostni izrazi za IRR, kot so opisane v poglavju spodaj
The maximum infusion rate is 25 mL/hour.
Največja hitrost infundiranja je 25 ml/uro.
It is given for the treatment of non-neurological symptoms of the disease.
Zdravilo je namenjeno zdravljenju simptomov bolezni, ki niso nevrološke narave.
- If the infusion-related reaction is severe, your doctor will stop the infusion immediately and start giving you appropriate medical treatment.
- Če je reakcija, povezana z infuzijo, resna, bo vaš zdravnik nemudoma prekinil infundiranje in vas začel zdraviti z ustreznim zdravilom.
In both cases the patients recovered without sequelae.
V obeh primerih sta bolnika okrevala brez posledic.
<18 years(2) ≥18 years(2)
< 18 let(2)
Consequently hepatic and renal impairment are not expected to affect the pharmacokinetic of velmanase alfa.
Posledično ni pričakovati, da bi jetrna in ledvična okvara vplivali na farmakokinetiko velmanaze alfa.
The infusion time can be calculated from the following table:
Hitrost infundiranja se lahko preračuna s pomočjo naslednje preglednice:
What Lamzede is and what it is used for
Kaj je zdravilo Lamzede in za kaj ga uporabljamo
The IRRs observed in clinical studies of velmanase alfa were characterised by a rapid onset of symptoms and were of mild to moderate severity.
IRR, ki so jih opazili v kliničnih študijah velmanaze alfa, so imele značilen hitri nastop simptomov in so bile po resnosti blage do zmerne.
Animal studies do not show evidence of impaired fertility.
Študije na živalih niso pokazale škodljivih vplivov na plodnost.
Filter needles must not be used during preparation.
Med pripravo ni dovoljeno uporabljati igel s filtrom.
Lamzede should be used during pregnancy only when medically necessary.
Zdravilo Lamzede je dovoljeno med nosečnostjo uporabljati le, če je to potrebno iz zdravstvenih razlogov.
There is no experience with patients younger than 6 years of age.
Pri bolnikih, mlajših od 6 let, ni izkušenj.
Animal studies do not indicate direct or indirect harmful effects with respect to pregnancy, embryonal/foetal development, parturition or postnatal development (see section 5.3).
Študije na živalih ne kažejo neposrednih ali posrednih škodljivih učinkov na nosečnost, razvoj zarodka/plodu, poroda ali postnatalnega razvoja
Contents of the pack and other information What Lamzede contains
Vsebina pakiranja in dodatne informacije Kaj vsebuje zdravilo Lamzede
All of these adverse reactions were non-serious.
Nobeden od neželenih učinkov ni bil resen.