Clinical trial NCT00633139: Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Official title: A Single Center, Open-Label, Non-Randomized, Uncontrolled, Multiple-Dose, Dose Escalation Study of the Safety, Pharmacokinetics, Efficacy and Long Term Safety of HGT-1111 (Recombinant Human Arylsulfatase A [rhASA, Metazym]) for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Late Infantile Metachromatic Leukodystrophy
Keywords: Metazym
Late infantile
Metachromatic leukodystrophy
Long-term safety
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2008-03-11
Enrollment: 13
Sex: ALL
Minimum age (years): 1
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a single center, open-label study of patients with late infantile MLD. All patients were previous treated 26 weeks in the phase I trial (EudraCT number: 2006-005341-11, NCT00418561). All patients will be offered continuing treatment in this study and will in this protocol receive 13 infusions, whereby the patients total have had 27 infusions of Metazym. One infusion will be given every other week. After a total of 52 weeks of treatment the subjects will continue treatment in a compassionate use protocol. Safety (AE/SAE) will be monitored at every visit.


Eligibility criteria: Inclusion Criteria:

The patients from the Phase I trial must meet the following criteria to be enrolled in the study.

* 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)
* The subject and his/her guardian(s) must have the ability to comply with the clinical protocol

Exclusion Criteria:

* Spasticity so severe to inhibit transportation
* 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
* Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
* 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

Primary outcomes:
measure: Relative Changes (%) in Gross Motor Function Measurement (GMFM)
timeFrame: Baseline, 52 Weeks
measure: Relative Change in Mullen's Scales of Early Learning
timeFrame: Baseline, 52 Weeks
Secondary outcomes:
measure: Change in Cerebrospinal Fluid (CSF) Sulfatide
timeFrame: Baseline, 52 Weeks
Other outcomes:
Locations:
city: Hvidovre
country: Denmark
facility: PhaseOneTrials A/S
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: I Dali C, Groeschel S, Moldovan M, Farah MH, Krageloh-Mann I, Wasilewski M, Li J, Barton N, Krarup C. Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study. Ann Clin Transl Neurol. 2021 Jan;8(1):66-80. doi: 10.1002/acn3.51254. Epub 2020 Dec 17.