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

Shire

Status and phase: Terminated; Phase 2

Conditions: Late Infantile Metachromatic Leukodystrophy

Treatments: Drug: HGT-1111

Study type: Interventional

Funder types: Industry

Identifiers

NCT00681811

2008-000084-41 (EudraCT Number)

HGT-MLD-049

Details and patient eligibility

About

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.

Full description

The primary objective of this study is to provide ongoing treatment of HGT-1111 to patients who have completed study HGT-MLD-048 (previously study rhASA-03 - NCT00633139) until HGT-1111 is commercially available or the study is terminated by the Sponsor, provided no safety concerns have emerged. The secondary objective of this study is to monitor disease progression and the safety profile of HGT-1111 administered to patients who have completed study HGT-MLD-048 (NCT00633139).

Enrollment: 11 patients

Sex: All

Ages: 3 to 6 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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. Completion of study HGT-MLD-048 (NCT00633139)
3. The subject and his/her guardian(s) must have the ability to comply with the protocol

Exclusion criteria

1. Spasticity so severe to inhibit transportation
2. 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
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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

11 participants in 2 patient groups

HGT-1111 100 U/kg

Experimental group

Treatment:

Drug: HGT-1111

HGT-1111 200 U/kg

Experimental group

Treatment:

Drug: HGT-1111