A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of MLD patients with high residual level of voluntary function

Shire Pharmaceuticals Ireland Limited0 个研究点目标入组 6 人开始时间: 2008年1月14日最近更新: 2022年4月4日

试验速览

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•Patients must meet the following criteria to be enrolled in the study:
•1. Subject’s legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject).
•2. The patient must have a confirmed diagnosis of MLD as defined by:
•· ASA activity < 10 nmol/h/mg in leukocytes
•· Presence of confirmed pathogenetic mutations in the ARSA gene or elevated sulfatide in urine
•3. The patient must have an age at the time of screening = 1 year and < 6 years.
•4. GMFM raw score ³ 60 at baseline (assessed not more than 4 weeks prior to first dosing).
•5. The patient must have had onset of symptoms before the age of 4 years.
•6. The subject and his/her guardian(s) must have the ability to comply with the clinical protocol.
•7. The patients’ medical record must document that the legal guardian(s) has had independent counseling or a consultation regarding stem cell transplantation in order to assure that the guardian(s) is fully informed regarding the risks and benefits of this alternative.
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Patients will be excluded from this study if they do not meet the specific inclusion criteria, or if any of the following criteria apply:
•1. Spasticity so severe to inhibit transportation
•2. Known multiple sulfatase deficiency
•3. Presence of major congenital abnormality
•4. Presence of known chromosomal abnormality and syndromes affecting psychomotor development
•5. History of stem cell transplantation
•6. Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition that, in the opinion of the Investigator, would preclude participation in the trial
•7. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
•8. Use of any investigational product other than rhASA within 30 days prior to study enrolment or currently enrolled in another study which involves clinical investigations.

发起方

Shire Pharmaceuticals Ireland Limited