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Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD

NCT00681811 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2021-06-08

Study results available
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Summary

This is a multi-center, open-label, extension study of patients with late infantile MLD who have previously completed clinical study HGT-MLD-048 (NCT00633139), defined as the completion of all Week 52 procedures. This group of patients will be offered ongoing treatment with HGT-1111 in this protocol. One infusion will be given every other week until the product is commercially available, the patient discontinues, or the study is terminated by the Sponsor, provided no safety issues have emerged.

Conditions

  • Late Infantile Metachromatic Leukodystrophy

Interventions

DRUG

HGT-1111

Patients currently dosed with 100 U/kg or 200 U/kg will continue this treatment. Patients dosed with 50 U/kg will be equally randomized to treatment on 100 U/kg or 200 U/kg. The dose will be adjusted every 6-week to account for changes in body weight.The infusion length will be dependent on the dose. Infusion of 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.

Sponsors & Collaborators

  • Shire

    lead INDUSTRY

Principal Investigators

  • Study Director · Takeda

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
3 Years
Max Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-02-20
Primary Completion
2010-10-22
Completion
2010-10-22

Countries

  • Denmark

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00681811 on ClinicalTrials.gov