|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
EUCTR |
|
Last refreshed on:
|
14 March 2022 |
|
Main ID: |
EUCTR2008-000084-41-IT |
|
Date of registration:
|
11/08/2009 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
A Multi-center, Open-Label Extension Study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) Treatment in Patients with Late Infantile Metachromatic Leukodystrophy (MLD) - ND
|
|
Scientific title:
|
A Multi-center, Open-Label Extension Study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) Treatment in Patients with Late Infantile Metachromatic Leukodystrophy (MLD) - ND |
|
Date of first enrolment:
|
25/08/2009 |
|
Target sample size:
|
18 |
|
Recruitment status: |
Not Recruiting |
|
URL:
|
https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-000084-41 |
|
Study type:
|
Interventional clinical trial of medicinal product |
|
Study design:
|
Controlled: no
Randomised: no
Open: no
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: yes
Other trial design description: open phase from previous randomised trial
If controlled, specify comparator, Other Medicinial Product:
Placebo:
Other:
|
|
Phase:
|
Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): yes
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): no
|
|
|
Countries of recruitment
|
|
Belgium
|
Denmark
|
France
|
Italy
| | | | |
|
Contacts
|
|
Name:
|
|
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
|
|
|
Name:
|
|
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
|
| |
|
Key inclusion & exclusion criteria
|
Inclusion criteria:
1. The patient`s legally authorized guardian(s) must provide signed informed consent prior to the performance of any study-related activities. (Study-related activities are any procedures that would not have been performed during normal management of the subject). 2. Completion of study HGT-MLD-048 3. The patient and his/her guardian(s) must have the ability to comply with the clinical protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range:
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. Spasticity so severe that it inhibiits the patient from being transported to the treatment site. 2. Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal diseas or any other medical condition that, in the opinion of the investigator, would preclude participatioin 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
Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
|
|
Health Condition(s) or Problem(s) studied
|
metachromatic leukodystrophy (MLD) in late infantile patients
MedDRA version: 9.1
Level: PT
Classification code 10024381
|
|
Intervention(s)
|
Product Name: HGT-1111
Pharmaceutical Form: Concentrate for solution for infusion
Current Sponsor code: rhASA
Concentration unit: IU/kg international unit(s)/kilogram
Concentration type: equal
Concentration number: 200-
|
|
Primary Outcome(s)
|
|
Primary end point(s): Safety endpoints: Adverse events, Vital signs, Urine analysis Laboratory data, Antibody and inhibitory antibody profile
|
|
Main Objective: The primary objective of this study is to provide ongoing treatment with HGT-1111 to patients who have completed study HGT-MLD-048 (previously study rhASA-03) until HGT-1111 is commercially available or the study is terminated by the Sponsor, provided no safety concerns have emerged
|
|
Secondary Objective: The secondary objective of this study is to monitor disease progression, as assessed by functional capacity, neurological examinations, cerebrospinal fluid (CSF) sulfatide concentrations and white matter metabolites by Magnetic Resonance Spectroscopic Imaging (MRSI) and the safety profile of HGT-1111 administered to patients who have completed study HGT-MLD-048
|
|
Secondary ID(s)
|
|
HGT-MLD-049
|
|
2008-000084-41-DK
|
|
Source(s) of Monetary Support
|
|
Ethics review
|
Status: Approved
Approval date: 27/07/2009
Contact:
|
|