Study Stopped
Company decision after projects re-evaluation, taken before the study initiation.
A Study to Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain
mECHromatic
Characterization of Metachromatic Leukodystrophy (MLD) in Pediatric Population in Spain: Epidemiological, Clinical, Diagnostic, Therapeutic, and Socioeconomic Aspects
1 other identifier
observational
N/A
1 country
10
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
Started Nov 2023
Shorter than P25 for all trials
10 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 2, 2023
CompletedFirst Posted
Study publicly available on registry
March 6, 2023
CompletedStudy Start
First participant enrolled
November 30, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2024
CompletedJanuary 12, 2024
January 1, 2024
7 months
February 2, 2023
January 10, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (6)
Overall Number of New Cases Diagnosed With MLD During Year 2022
Data for Year 2022 will be considered as a part of retrospective data collection from 01 January 2000 to 31 December 2022 obtained from health records.
1 year
Number of New Cases Diagnosed With MLD per Subgroups of Phenotype During Year 2022
Data for Year 2022 will be considered as a part of retrospective data collection from 01 January 2000 to 31 December 2022 obtained from health records.
1 year
Overall Number of Participants With MLD Alive On 31 December 2022
Data for this outcome measure will be collected on last day of retrospective data collection from 01 January 2000 to 31 December 2022 obtained from health records.
1 day
Number of Participants With MLD Alive On 31 December 2022 per Subgroups of Phenotype
Data for this outcome measure will be collected on last day of retrospective data collection from 01 January 2000 to 31 December 2022 obtained from health records.
1 day
Overall Number of Participants With MLD Alive at Any Time During Year 2022
Data for Year 2022 will be considered as a part of retrospective data collection from 01 January 2000 to 31 December 2022 obtained from health records.
1 year
Number of Participants With MLD Alive at Any Time During Year 2022 per Subgroups of Phenotype
Data for Year 2022 will be considered as a part of retrospective data collection from 01 January 2000 to 31 December 2022 obtained from health records.
1 year
Secondary Outcomes (18)
Number of Late Infantile and Juvenile (Early Juvenile and Late Juvenile) Phenotypes in Participants With MLD
23 years
Age at Symptom Onset in Participants With MLD
23 years
Number of Participants With MLD Categorized Based on Clinical Signs/ Symptoms at Disease Onset
23 years
Age of Participants at Diagnosis of MLD
23 years
Time of Diagnosis Delay
23 years
- +13 more secondary outcomes
Study Arms (1)
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.
Interventions
As this is an observational study, no intervention will be administered.
Eligibility Criteria
Participants diagnosed with late infantile or juvenile MLD in Spain.
You may qualify if:
- Diagnosed with late infantile or juvenile MLD at any time between 01 January 2000 and 31 December 2022 (genetically and/or biochemically confirmed).
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Takedalead
Study Sites (10)
Hospital Clinico Universitario de Santiago
Santiago de Compostela, A Coruna, 15706, Spain
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, 08950, Spain
Hospital Universitario de Cruces
Barakaldo, Bizkaia, 48903, Spain
Hospital Universitario de Donostia
Donostia / San Sebastian, Gipuzkoa, 20014, Spain
Hospital Materno Infantil de Gran Canaria
Las Palmas de Gran Canaria, Las Palmas, 35016, Spain
Hospital Universitario de Canarias
San Cristóbal de La Laguna, Santa Cruz De Tenerife, 38320, Spain
Hospital Universitario Vall dHebron
Barcelona, 08035, Spain
Hospital Universitario Infantil Nino Jesus
Madrid, 28009, Spain
Hospital Universitario Virgen del Rocio
Seville, 41013, Spain
Hospital Universitario y Politecnico La Fe
Valencia, 46026, Spain
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 2, 2023
First Posted
March 6, 2023
Study Start
November 30, 2023
Primary Completion
July 1, 2024
Study Completion
July 1, 2024
Last Updated
January 12, 2024
Record last verified: 2024-01
Data Sharing
- IPD Sharing
- Will not share
Takeda does not provide access to Individual Participant Data when a study is in a very limited (small) study population due to participant privacy concerns such as potential reidentification of study participants.