A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injectio

Phase 1

• Conditions: Treatment of Metachromatic Leukodystrophy; MedDRA version: 16.0Level: PTClassification code 10067609Term: Metachromatic leukodystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2012-003775-20-FR
• Lead Sponsor: Shire Human Genetics Therapies Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-06-27
• Last Update Posted: 2 October 2017

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

1.Patient has participated in Study HGT-MLD-070 through Week 40.
2.Patient must have no safety or medical issues that contraindicate participation.
3.The patient, patient’s parent or legally authorized representative(s) must provide written informed consent and/or assent (if applicable) prior to performing any study-related activities.

Are the trial subjects under 18? yes
Number of subjects for this age range: 18
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.The patient is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the Investigator.
2.The patient has any known or suspected hypersensitivity to agents used for sedation or is thought to be at an unacceptably high risk for associated potential complications of airway compromise or other conditions
3.The patient is pregnant or breastfeeding.
4.The patient is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or drug delivery device) other than those used in HGT-MLD-070 within 6 months prior to study enrollment or at any time during the study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified