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

Phase 2

Terminated

• Conditions: Late Infantile Metachromatic Leukodystrophy
• Interventions: Drug: HGT-1111

• Registration Number: NCT00681811
• Lead Sponsor: Shire

Brief 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.

Detailed 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).

Study Timeline

• Study Start: 2008-02-20
• Study First Submitted: 2008-05-19
• Study First Submitted QC: 2008-05-19
• Study First Posted: 2008-05-21
• Primary Completion: 2010-10-22
• Study Completion: 2010-10-22
• Results First Submitted: 2012-09-26
• Results First Submitted QC: 2013-09-05
• Results First Posted: 2013-11-08
• Status Verified: 2021-05
• Last Update Submitted: 2021-05-19
• Last Update Posted: 2021-06-08

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 11

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
HGT-1111 200 U/kg	HGT-1111	-
HGT-1111 100 U/kg	HGT-1111	-

Primary Outcome Measures

Name	Time	Method
Days of Exposure to HGT-1111	Baseline until end of study (Week 139)	End of study was defined as until HGT-1111 was commercially available, the participant's participation was discontinued, or the study was terminated by the Sponsor.

Secondary Outcome Measures

Name	Time	Method
Level of White Matter Metabolites	Baseline until end of study (Week 139)	Level of white matter metabolites \[N-acetyl Aspartate (NAA)\] measured at 6-month intervals in HGT-MLD-049 (NCT00681811).
Level of Cerebrospinal Fluid (CSF) Sulfatide	Baseline until end of study (Week 139)	Level of CSF sulfatide measured at 6-month intervals in HGT-MLD-049 (NCT00681811).
Score of Gross Motor Function Measurement (GMFM)	Baseline until end of study (Week 139)	Gross motor function was measured using GMFM-88 at 6-month intervals. The GMFM-88 item scores were summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score was between 0 (minimal) to 3 (maximum). The total GMFM-88 score was between 0 (minimal) to 264 (maximum). Decrease in GMFM score indicates disease progression.

Trial Locations

Locations (1)

Rigshospitalet; 🇩🇰 Copenhagen, Denmark