NEW GENE THERAPY TRIAL FOR ADRENOELUKODYSTROPHY NUEVO ENSAYO CLÍNICO DE TERAPIA GENICA PARA ADRENOLEUCODISTROFIA. Boston- Dr Asif Paker (especialista en leucodistrofia que disertó en el 1º Encuentro de Familias con Leucodistrofias organizado por la Fundación Lautaro te Necesita en Bs As. diciembre 2014) In 2009 the results of the gene therapy trial for childhood cerebral adrenoleukodystrophy (ALD) performed in Paris by Pr. Patrick Aubourg and Dr. Nathalie Cartier and largely funded by ELA were published. Now a large follow-up clinical trial using an improved viral vector has been authorized in the US and will be developped by bluebird bio, Inc. If proven efficient, this new trial, should allow getting all the required authorizations to market the drug in the future. You will find below a sumary of the study. Status: Authorized; patient recruitment active (Boston site) Drug: Lentiviral vector Lenti-D carrying the normal human ABCD1 gene Objective: To evaluate the safety and efficacy of the treatment in childhood cerebral ALD Start date of the study: August 2013 Estimated end of the trial: August 2018 Director of the study: Dr. Asif Paker, bluebird bio, Inc. Principal investigators Dr. David Williams, Boston Children’s Hospital, Boston, MA, USA Dr. Christine Duncan, Children’s Hospital, Boston, MA, USA Dr. Florian Eichler, Massachusetts General Hospital, Boston, MA, USA Location: Boston Children Hospital / Massachussetts General Hospital, Boston, MA, USA Funding: bluebird bio Reference : ALD-102 study, Starbeam study, NCT01896102 Patients Fifteen children suffering from the cerebral form of ALD at a pre-symptomatic stage Experimental protocol Collection of bone marrow stem cells from the patient blood and isolation of the CD34+ stem cells Transfer of the gene into the CD34+ stem cells using the viral vector. Myeloablative conditioning of the patient (destruction of the bone marrow). Reinfusion of the corrected cells in the patient (autotransplant). Inclusion criteria Age: up to 17 years old Gender: Male Active childhood cerebral adrenoleukodystrophy Neurological function score ≤ 1 Exclusion criteria Receipt of an allogeneic transplant or gene therapy. Availability of a willing 10/10 matched sibling donor. Use of statins, Lorenzo’s oil or diets aiming at lowering very long chain fatty acids. Note : subjects must discontinue use of these medications at time of consent. Impossibility to perform MRI studies. Hematological, hepatic, renal, pulmonary or cardiac compromise. Infections and other conditions. Patient follow-up Patients will be followed-up several years after their gene therapy treatment. Additional information bluebird bio announces the first patient treated – October 28, 2013 Other centers will be opened in the future in the United States and in Europe. When new centers will be authorized, we will update the section « Clinical trials » of our website. Contacts Tara O’Meara, bluebird bio, Inc. – Telephone : +1 617-797-2555, e-mail : clinicaltrials@bluebirdbio Colleen Dansereau, Boston Children’s Hospital/Massachusetts General Hospital – Telphone : +1 617-919-7008, e-mail : [email protected] Source: clinicaltrials.gov/ct2/show/NCT01896102 starbeamstudy
Posted on: Tue, 30 Dec 2014 03:01:41 +0000
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