TerminatedPhase 2

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

Stopped: Terminated prior to planned completion date due to lack of efficacy.

Denmark11 participantsStarted 2008-02-20

Plain-language 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.

Who can participate

Age range3 Years – 6 Years

SexALL

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Inclusion criteria

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

What they're measuring

Days of Exposure to HGT-1111

Timeframe: Baseline until end of study (Week 139)

Trial details

NCT IDNCT00681811

SponsorShire

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2010-10-22

Results submitted2012-09-26

View on ClinicalTrials.gov More Late Infantile Metachromatic Leukodystrophy trials