metachromatic leukodystrophy (MLD) in late infantile patients MedDRA version: 9.1 Level: PT Classification code 10024381
Conditions
Interventions
Product Name: HGT-1111
Pharmaceutical Form: Concentrate for solution for infusion
Current Sponsor code: rhASA
Concentration unit: IU/kg international unit(s)/kilogram
Concentration type: equal
Concent
Sponsors
Shire Human Genetic Therapies Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. The patient`s legally authorized guardian(s) must provide signed informed consent prior to the performance of any study-related activities. (Study-related activities are any procedures that would not have been performed during normal management of the subject). 2. Completion of study HGT-MLD-048 3. The patient and his/her guardian(s) must have the ability to comply with the clinical protocol. 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
Exclusion criteria
Exclusion criteria: 1. Spasticity so severe that it inhibiits the patient from being transported to the treatment site. 2. Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal diseas or any other medical condition that, in the opinion of the investigator, would preclude participatioin in the trial. 3. Any other medical condition or serious intercurrent illness or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to provide ongoing treatment with HGT-1111 to patients who have completed study HGT-MLD-048 (previously study rhASA-03) until HGT-1111 is commercially available or the study is terminated by the Sponsor, provided no safety concerns have emerged;Secondary Objective: The secondary objective of this study is to monitor disease progression, as assessed by functional capacity, neurological examinations, cerebrospinal fluid (CSF) sulfatide concentrations and white matter metabolites by Magnetic Resonance Spectroscopic Imaging (MRSI) and the safety profile of HGT-1111 administered to patients who have completed study HGT-MLD-048;Primary end point(s): Safety endpoints: Adverse events, Vital signs, Urine analysis Laboratory data, Antibody and inhibitory antibody profile | — |
Countries
Belgium, Denmark, France, Italy
Outcome results
None listed