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

• Register: EUCTR
• Last refreshed on: 17 March 2025
• Main ID: EUCTR2012-003775-20-FR
• Date of registration: 27/06/2013
• Prospective Registration: No
• Primary sponsor: Shire Human Genetics Therapies Inc
• Public title: A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injection
• Scientific title: An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy
• Date of first enrolment: 31/12/2012
• Target sample size: 18
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-003775-20
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 3
• Phase: Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Australia, Brazil, Czech Republic, Denmark, France, Germany, Italy, United Kingdom

Contacts

• Name: Stephanie Haller
• Affiliation: Shire Human Genetics Therapies Inc.

• Name: Stephanie Haller
• Affiliation: Shire Human Genetics Therapies Inc.

Key inclusion & exclusion criteria

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.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Treatment of Metachromatic Leukodystrophy
MedDRA version: 16.0 Level: PT Classification code 10067609 Term: Metachromatic leukodystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Intervention(s)

Product Code: HGT-1110
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Not available
Current Sponsor code: HGT-1110
Other descriptive name: Recombinant Human Arylsulfatase A (rhASA)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 30-

Primary Outcome(s)

Primary end point(s): The primary objective of this study is to collect long-term safety data of intrathecal HGT-1110 treatment in patients who participated through Week 40 of Study HGT-MLD-070. Safety will be measured by the following endpoints:
- Adverse events (by type and severity)
- Changes in clinical laboratory testing (serum chemistry including liver function tests, hematology, and urinalysis), vital signs, physical examinations and the neurological examinations
- Change from baseline in CSF chemistries (including cell counts, glucose, albumin, and protein)
- Development of anti-HGT-1110 antibodies in CSF and/or serum.

Main Objective: To collect long-term safety data in patients with MLD who are receiving HGT-1110 and have participated in study HGT-MLD-070 through Week 40.

Secondary Objective: - To evaluate changes in gross motor function in patients who are receiving IT administration of HGT-1110
- To assess concentrations of HGT-1110 in cerebrospinal fluid (CSF)

Timepoint(s) of evaluation of this end point: - AEs will be collected every other week
- Chemistry/hematology/urinalysis testing will be done every 12 weeks throughout the study
- CSF chemistries will be assessed every other week
- Anti-HGT-1110 antibodies in CSF and/or serum will be assessed every 12 weeks throughout the study

Secondary Outcome(s)

Secondary end point(s): The secondary endpoints of this study are:
- Change from baseline in motor function using the Gross Motor Function Measure-88 (GMFM-88) total raw score
- Concentrations of HGT-1110 in CSF at selected time points after single and repeated study drug administration

Timepoint(s) of evaluation of this end point: - GMFM-88 will be conducted every 12 weeks for the 1st year of treatment and every 24 weeks for the subsequent years
- HGT-1110 in CSF will be evaluated every other week throughout the study.

Secondary ID(s)

HGT-MLD-071

2012-003775-20-DE

Source(s) of Monetary Support

Shire HGT, Inc

Ethics review

Status: Approved
Approval date: 10/07/2012
Contact: