A Study to Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain

NCT ID: NCT05755568

Last Updated: 2024-01-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Study Classification

OBSERVATIONAL

Study Start Date

2023-11-30

Study Completion Date

2024-07-01

Brief Summary

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The main aim of this study is to learn about metachromatic leukodystrophy (MLD) in children and teenagers in Spain. This includes checking the number of new MLD cases and the frequency of children with MLD in 2022 in Spain.

There is no treatment involved in this study. Participants' data will be taken from their medical records (charts), which were already collected as a part of their routine care between 01 January 2000 and 31 December 2022.

Detailed Description

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This is a non-interventional, retrospective study of participants diagnosed with late infantile or juvenile MLD whose data will be retrieved from existing health records.

Data from all participants, diagnosed with MLD from 01 January 2000 to 31 December 2022 will be collected from health records, including demographic and clinical data from the pre-diagnosis until the date of data collection or death, whatever occurs first.

The study will be conducted in Spain. The overall duration for data extraction is up to approximately 23 years.

Conditions

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Metachromatic Leukodystrophy (MLD)

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

RETROSPECTIVE

Study Groups

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MLD Participants

Data from medical records of participants diagnosed with late infantile or juvenile MLD at any time from 1 January 2000 to 31 December 2022 will be observed retrospectively.

No Intervention

Intervention Type OTHER

As this is an observational study, no intervention will be administered.

Interventions

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No Intervention

As this is an observational study, no intervention will be administered.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

• Diagnosed with late infantile or juvenile MLD at any time between 01 January 2000 and 31 December 2022 (genetically and/or biochemically confirmed).
Minimum Eligible Age

0 Years

Maximum Eligible Age

15 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Takeda

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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Hospital Clinico Universitario de Santiago

Santiago de Compostela, A Coruna, Spain

Site Status

Hospital Sant Joan de Deu

Esplugues de Llobregat, Barcelona, Spain

Site Status

Hospital Universitario de Cruces

Barakaldo, Bizkaia, Spain

Site Status

Hospital Universitario de Donostia

Donostia / San Sebastian, Gipuzkoa, Spain

Site Status

Hospital Materno Infantil de Gran Canaria

Las Palmas de Gran Canaria, Las Palmas, Spain

Site Status

Hospital Universitario de Canarias

San Cristóbal de La Laguna, Santa Cruz De Tenerife, Spain

Site Status

Hospital Universitario Vall dHebron

Barcelona, , Spain

Site Status

Hospital Universitario Infantil Nino Jesus

Madrid, , Spain

Site Status

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status

Hospital Universitario y Politecnico La Fe

Valencia, , Spain

Site Status

Countries

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Spain

Related Links

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Other Identifiers

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TAK-611-5002

Identifier Type: -

Identifier Source: org_study_id

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