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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT01963650
Other study ID # HGT-MLD-092
Secondary ID
Status Terminated
Phase
First received
Last updated
Start date November 2, 2015
Est. completion date April 8, 2016

Study information

Verified date March 2021
Source Takeda
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

The purpose of this study is evaluate the natural course of disease progression related to gross motor function in children with metachromatic leukodystrophy (MLD).


Description:

Metachromatic leukodystrophy (MLD) is an inherited, autosomal recessive disorder of lipid metabolism characterized by deficient activity of the lysosomal enzyme, arylsulfatase A (ASA). MLD is a rare genetic disease that occurs in most parts of the world. The estimated overall incidence of the disease in the western world is approximately 1 in 100,000 live births. This study is a multicenter, observational, longitudinal study that plans to enroll up to 30 patients with onset of MLD-related signs and symptoms prior to 30 months of age and who are less than 12 years of age. Patients will participate in this study for approximately 114 weeks (Screening through Follow-up) and will be assessed at defined intervals for disease status.


Recruitment information / eligibility

Status Terminated
Enrollment 1
Est. completion date April 8, 2016
Est. primary completion date April 8, 2016
Accepts healthy volunteers No
Gender All
Age group N/A to 12 Years
Eligibility Inclusion Criteria: 1. Confirmed diagnosis of MLD by both: - arylsulfatase A (ASA) deficiency by assay in leukocytes AND - elevated sulfatide in urine 2. Appearance of the first symptoms of disease at or before 30 months of age. 3. A GMFM-88 total (percent) score greater than or equal to 40 at the screening examination. 4. The patient is less than 12 years of age at the time of enrollment. 5. The patient and his/her parent or legally authorized representative(s) must have the ability to comply with the clinical protocol. 6. Patient's parent or legally authorized representative(s) must provide written informed consent prior to performing any study-related activities. Study-related activities are any procedures that would not have been performed during normal management of the patient. Exclusion Criteria: 1. History of hematopoietic stem cell transplantation. 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. Any other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the study. 4. The patient is enrolled in another clinical study that involves the use of any investigational product (drug or device) within 30 days prior to study enrollment or at any time during the study.

Study Design


Locations

Country Name City State
Argentina Hospital Universitario Austral Pilar
Belgium Universitair Ziekenhuis Antwerpen (UZA) (University Hospital Antwerpen) Edegem
Brazil Hospital de Cllnicas de Porto Alegre (HCPA) / UFRGS Porto Alegre
Canada Montreal Children's Hospital Westmount
Denmark Copenhagen University Hospital, Rigshospitalet Copenhagen
France Hôpital De Bicêtre Le Kremlin Bicêtre
Germany Univesitatsklinikum Tubingen Klinik fur Kinder und Jugendmedizin Tubingen
Japan Faculty Of Medicine, Osaka University Graduate School Of Medicine Osaka
Japan The Jikei University School Of Medicine - Institute Of Dna Medicine Tokyo
Turkey Hacettepe Universitesi Tip Fakultesi Onkoloji Hastanesi Ankara
United States Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
United States Children's Hospital Of Pittsburgh Pittsburgh Pennsylvania
United States Harbor UCLA Pediatrics Torrance California
United States Children's National Health System Washington District of Columbia

Sponsors (1)

Lead Sponsor Collaborator
Shire

Countries where clinical trial is conducted

United States,  Argentina,  Belgium,  Brazil,  Canada,  Denmark,  France,  Germany,  Japan,  Turkey, 

Outcome

Type Measure Description Time frame Safety issue
Primary The primary endpoint of this study is the change from baseline in motor function using the GMFM-88 total (percent) score. Week 0 to Week 104
Secondary The change from baseline in ability to swallow as assessed by the Functional Endoscopic Evaluation of Swallowing. Week 0 to Week 104
Secondary The change from baseline in nerve conduction as measured by the electroneurography. Week 0 to Week 104
Secondary The change from baseline in the adaptive behavior composite standard score as measured by the Vineland Adaptive Behavior Scales. Week 0 to Week 104
Secondary The change from baseline in domain-specific Caregiver Observed MLD Functioning and Outcomes Reporting Tool. Week 0 to Week 104
Secondary The change from baseline in cognitive function using the Mullen Scales of Early Learning. Week 0 to Week 104
Secondary Reporting of any study procedure-related nonserious AEs and/or any SAEs Week 0 to Week 114
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