A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injectio
- Conditions
- Treatment of Metachromatic LeukodystrophyMedDRA version: 20.0Level: PTClassification code 10067609Term: Metachromatic leukodystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Body processes [G] - Genetic Phenomena [G05]
- Registration Number
- EUCTR2012-003775-20-CZ
- Lead Sponsor
- Shire Human Genetics Therapies Inc
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 24
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: 24
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
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 anesthesia 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.
5. The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device
6. The patient’s body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the Investigator
7. The patient has a known or suspected local or general infection
8. The patient is at risk of abnormal bleeding due to a medical condition or therapy
9. The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
10. The patient has a functioning CSF shunt device
11. The patient has shown an intolerance to an implanted device
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method