Estudio de fase 3B, aleatorizado, multicéntrico, multinacional, abierto, de grupos paralelos de Fabrazyme (agalsidasa beta) en pacientes pediátricos masculinos sin tratamiento previo con enfermedad de Fabry sin síntomas graves

CompletadoFase 3ClinicalTrials.gov

ID: NCT00701415Tipo: INTERVENTIONALInicio: 1 de sept de 2008Fin estimado: 1 de jun de 2015

Resumen

El propósito de este estudio fue determinar si dos regímenes de dosificación alternativos de Fabrazyme (agalsidasa beta) (1,0 mg/kg cada 4 semanas o 0,5 mg/kg cada 2 semanas) eran eficaces en participantes pediátricos sin tratamiento previo y sin síntomas graves. Se preveía tratar a los participantes durante 5 años.

Elegibilidad

Edad mínima: 5 YearsEdad máxima: 18 YearsSexo: MALE

Criterios de inclusión

The participant and/or participant's parent(s)/legal guardian(s) must provide written informed assent/consent prior to any protocol-related procedures being performed.
The participant must had a confirmed diagnosis of Fabry disease as documented by leukocyte α-Galactosidase A (αGAL) activity of \<4 nmol/hr/mg leukocyte (preferred assay; resulted from a central laboratory). If the leukocyte αGAL activity assay was difficult to obtain, the participant might be enrolled based on documented plasma αGAL \<1.5 nmol/hr/mL, with the agreement of the Medical Monitor (resulted from a central laboratory).
The participant must had evidence of globotriaosylceramide (GL-3) accumulation as documented by plasma GL-3 (\>7.0 µg/mL) or urinary GL-3 (\>0.3 mg GL-3/mmol creatinine) levels (results from a central laboratory).
The participant must be male ≥5 and ≤18 years of age.

Criterios de exclusión

Participant had albuminuria (first morning void urinary albumin/creatinine ratio \>30 mg/g on at least 2 out of 3 consecutive samples, each at least 1 week apart).
Participant had a Glomerular Filtration Rate (GFR) by iohexol \<90 L/min/1.73m\^2. In case of properly documented low protein intake, values as low as 80 mL/min/1.73 m\^2 might be acceptable, after consultation with the Medical Monitor.
Participant had documented evidence of stroke or transient ischemic attack (TIA), or if a brain magnetic resonance imaging (MRI) had been performed, bright lesions \>2 mm on T2- or fluid attenuated inversion recovery (FLAIR)- weighted images within the white matter or the basal ganglia.
Participant had severe and recurrent acroparesthesia, judged by the physician as frequent (more than once a week) pain episodes for at least 3 months that influenced daily activities, irrespective of medication.
Participant had an end-diastolic left ventricular posterior wall thickness (LVPWTd) and/or an end-diastolic interventricular septum thickness (IVSTd)≥2 standard deviations (SD) compared to normal (based on body surface area \[BSA\] normal ranges from Kampmann, et al 2000) as read at the study site.
Participant had received prior treatment specific to Fabry Disease.
Participant had participated in a study employing an investigational drug within 30 days of the start of their participation in this study.
Participant had any medical condition or extenuating circumstance, which in the opinion of the Study Investigator, could interfere with study compliance.
Participant had any medical condition or extenuating circumstance, for example diabetes mellitus, which in the opinion of the Study Investigator, could interfere with the interpretation of study results.
Participant was on treatment with angiotensin converting enzyme inhibitors/angiotensin receptor blockers (ACEIs/ARBs).
Participant had any contra-indication mentioned in the labeling of Fabrazyme and/or iohexol (Omnipaque).
Participant or parent(s)/legal guardian(s) was unwilling to comply with the requirements of the protocol.

Intervenciones

biological

Agalsidase beta

Powder for concentrate for solution for infusion 1.0 mg/kg/4 weeks

biological

Agalsidase beta

Powder for concentrate for solution for infusion 0.5 mg/kg/2 weeks

Ubicaciones

Buenos Aires, Argentina

Patrocinadores

PrincipalGenzyme, a Sanofi Company

Aviso: La información de este ensayo proviene de fuentes públicas y tiene carácter exclusivamente informativo. No constituye asesoramiento médico, regulatorio ni de ningún otro tipo. Las traducciones son automatizadas y no poseen validez oficial. Consultá siempre con un profesional de la salud. Ver Términos de Uso.

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