Metachromatic Leukodystrophy (MLD) Clinical Trial
Official title:
A Single Center, Open-label, Non-randomized, Uncontrolled, Multiple-dose, Dose Escalation Study of the Safety, Pharmacokinetics and Efficacy of Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD. Methodology: This is a single center, open-label study of patients with late infantile MLD. Twelve patients will be enrolled in this study receiving a total of thirteen intravenous infusions of Metazym. One infusion will be given every other week for a period of half a year. After the half year the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period.
Test product, dose, mode of administration, batch No.: The lowest dose level will be evaluated as a single dose of 25 U/kg. The three upper dose levels will be evaluated as repeated doses. Patients in each cohort will receive one dose of enzyme every other week for a period of eight weeks, a total of five doses. Dosing will be performed as follows: Cohort 1: 25 U/kg as a single dose - hereafter 50 U/kg; Cohort 2: 100 U/kg; Cohort 3: 200 U/kg. Patients receiving the lowest dose as a single dose will receive the next dose level as a repeated dose. After twenty six weeks the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period. The dose will be adjusted monthly to account for changes in body weight. The infusion length will be dependent on the dose. Doses of 25 U/kg, 50 U/kg and 100 U/kg will be diluted in 50 ml isotonic sodium chloride and infused over 30 minutes. Infusion of 200 U/kg will be administered in the same manner except for an infusion time of 60 minutes. Duration of treatment: Half a year (26 weeks) ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT03725670 -
Lentiviral Gene Therapy for MLD
|
N/A | |
Withdrawn |
NCT05755568 -
A Study to Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain
|
||
Terminated |
NCT01963650 -
Natural History Study of Children With Metachromatic Leukodystrophy
|
||
Active, not recruiting |
NCT01887938 -
An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT03771898 -
A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
|
Phase 2 | |
Completed |
NCT01510028 -
Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
|
Phase 1/Phase 2 | |
Completed |
NCT01043640 -
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
|
Phase 2 | |
Terminated |
NCT01372228 -
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
|
Phase 1/Phase 2 |