Evaluation of efficacy and safety of two different formulas of alfa-Galactosidase A, agalsidase beta (FabrazymeTM) and agalsidase alpha (ReplagalTM) in an equal dose of 0,2 mg/kg in order to detect any differences between these two drugs.
Bron
Verkorte titel
Aandoening
Fabry disease.
Ondersteuning
College voor Zorgverzekeringen (dutch health care insurance board).
Initiator:
Dr. C.E.M. Hollak, internist
Dept. of Internal Medicine, F4-279
Academic Medical Center
PO box 22660
1100 DD Amsterdam
The Netherlands
tel. +31-20-5666071
fax: +31-20-6919743
e-mail: c.e.hollak@amc.uva.nl
Onderzoeksproduct en/of interventie
Geen registraties gevonden.
Uitkomstmaten
Primaire uitkomstmaten
Wall-thickness (septum and left and right ventricle wall) / end-diastolic volume) on echocardiography.
Achtergrond van het onderzoek
Fabry disease is an X-linked disorder caused by the deficiency of the lysosomal enzyme alfa-Galactosidase A (alfa-Gal). Patients with this disorder suffer in childhood from severe pains in hands and feet and develop severe complications later in life such as renal failure, CVA¡¦s and cardiac complications.
Patients with Fabry disease have a reduced life expectancy. Recently two differently alfa-Galactosidase enzyme preparations have received marketing authorization in the EU (orphan drug).
Conclusions on the differences between these products with regard to safety and efficacy cannot be drawn because of the different dosage and evaluation methods performed.
Therefore treatment should be performed according to a standardized treatment approach which allows comparison of both drugs.
Protocol objectives:
To monitor and evaluate the efficacy and safety of two different formulas of alfa-Galactosidase A, agalsidase alpha (FabrazymeTM) and agalsidase beta (ReplagalTM) in an equal dose of 0,2 mg/kg in adults with Fabry disease.
Investigational plan: Symptomatic Fabry patients (aged 18 or older) who fulfil the criteria will receive enzyme therapy for at least 12 months and will be evaluated every 3 months.
Doel van het onderzoek
Evaluation of efficacy and safety of two different formulas of alfa-Galactosidase A, agalsidase beta (FabrazymeTM) and agalsidase alpha (ReplagalTM) in an equal dose of 0,2 mg/kg in order to detect any differences between these two drugs.
Onderzoeksopzet
N/A
Onderzoeksproduct en/of interventie
Patients will receive 0,2mg/kg Fabrazyme (agalsidase beta) or 0,2 mg/kg Replagal (agalsidase alpha), every two weeks for a minumum of 12 months. If there's treatment failure (progression of renal disease, cardiac disease and/or a new cerebral stroke or TIA) during or after this period, patients will be advised to switch to Fabrazyme 1,0 mg/kg/2 wks.
Publiek
Department of Internal Medicine, F4-247,
P.O. Box 22660
A.C. Vedder
Meibergdreef 9
Amsterdam 1100 DD
The Netherlands
+31 (0)20 5664558
a.c.vedder@amc.uva.nl
Wetenschappelijk
Department of Internal Medicine, F4-247,
P.O. Box 22660
A.C. Vedder
Meibergdreef 9
Amsterdam 1100 DD
The Netherlands
+31 (0)20 5664558
a.c.vedder@amc.uva.nl
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
1. The patient must have given written informed consent;
2. Patients must be 18 years or older;
3. Patient must have a current diagnosis of Fabry disease;
4. Patients must have a decreased á-Gal activity or proven alfa-Gal A mutation;
5. Female patients must have a negative pregnancy test, and must use a medically accepted method of contraception;
6. Patients must be willing to comply to the evaluation program;
7. Patients must have a clinical presentation consistent with either typical or atypical Fabry disease. Patients must have at least one major or two minor objective criteria:
Major:
a. Severe acroparesthesias, that cannot satisfactorily be controlled with Carbamazepine;
b. Decreased GFR < 80 ml/min;
c. Proteinuria > 300 mg/ml;
d. Documented CVA;
e. Cardiac infarction;
f. Hypertrophic Non-obstructive Cardiomyopathy resulting in decreased exercise tolerance;
g. Rhythm disturbances necessitating a pacemaker;
h. Multiple lacunar infarctions on MRI;
Minor:
i. Documented TIA;
j. Cardiac hypertrophy on echo or MRI;
k. Atrial fibrillation;
l. Intraventricular conduction abnormality;
m. Sensoric hearing loss as shown on a hearing test;
n. Severe vertigo;
o. Micro-albuminuria > 50 mg/L;
p. Mild to moderate acroparesthesias;br>
q. Gastro-intestinal complaints that can not be explained by other medical conditions than Fabry disease.
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
1. Patient is pregnant or lactating;
2. Patient is unwilling to comply to the evaluation program.
Opzet
Deelname
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
Geen registraties gevonden.
Andere (mogelijk minder actuele) registraties in dit register
Geen registraties gevonden.
In overige registers
Register | ID |
---|---|
NTR-new | NL179 |
NTR-old | NTR216 |
Ander register | : N/A |
ISRCTN | ISRCTN45178534 |