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Clinical Trial Details — Status: Withdrawn

Administrative data

NCT number NCT05755568
Other study ID # TAK-611-5002
Secondary ID
Status Withdrawn
Phase
First received
Last updated
Start date November 30, 2023
Est. completion date July 1, 2024

Study information

Verified date January 2024
Source Takeda
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.


Description:

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.


Recruitment information / eligibility

Status Withdrawn
Enrollment 0
Est. completion date July 1, 2024
Est. primary completion date July 1, 2024
Accepts healthy volunteers No
Gender All
Age group 0 Years to 15 Years
Eligibility 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). Exclusion Criteria: • This study has no exclusion criteria.

Study Design


Related Conditions & MeSH terms


Intervention

Other:
No Intervention
As this is an observational study, no intervention will be administered.

Locations

Country Name City State
Spain Hospital Universitario de Cruces Barakaldo Bizkaia
Spain Hospital Universitario Vall dHebron Barcelona
Spain Hospital Universitario de Donostia Donostia Gipuzkoa
Spain Hospital Sant Joan de Deu Esplugues de Llobregat Barcelona
Spain Hospital Universitario de Canarias La Laguna Santa Cruz De Tenerife
Spain Hospital Materno Infantil de Gran Canaria Las Palmas de Gran Canaria Las Palmas
Spain Hospital Universitario Infantil Nino Jesus Madrid
Spain Hospital Clinico Universitario de Santiago Santiago de Compostela A Coruna
Spain Hospital Universitario Virgen del Rocio Sevilla
Spain Hospital Universitario y Politecnico La Fe Valencia

Sponsors (1)

Lead Sponsor Collaborator
Takeda

Country where clinical trial is conducted

Spain, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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
Primary 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
Primary 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
Primary 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
Primary 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
Primary 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 Number of Late Infantile and Juvenile (Early Juvenile and Late Juvenile) Phenotypes in Participants With MLD 23 years
Secondary Age at Symptom Onset in Participants With MLD Age at symptom onset in participants with MLD in years will be recorded. 23 years
Secondary Number of Participants With MLD Categorized Based on Clinical Signs/ Symptoms at Disease Onset Clinical signs and symptoms include impaired gross motor skills, impaired fine motor skills, impaired language skills, impaired non-verbal communication, cognition, feeding/swallowing difficulties, skeletal deformity, musculoskeletal pain. 23 years
Secondary Age of Participants at Diagnosis of MLD Age of participants at diagnosis of MLD in years will be recorded. 23 years
Secondary Time of Diagnosis Delay Time of diagnosis delay will be computed as age at diagnosis minus age at symptom onset. 23 years
Secondary Number of Participants With Disease Diagnosis Based on Different Suggestive Findings Suggestive findings will include motor, cognitive, behavioural, magnetic resonance imaging (MRI) and magnetic resonance (MR) spectroscopy. 23 years
Secondary Number of Participants With Disease Diagnosis Based on Confirmatory Tests Confirmatory tests will include urine sulfatides, arylsulfatase A (ARSA) activity, ARSA genetic variants. 23 years
Secondary Number of Interactions of Participants With the Healthcare System Post Diagnosis Interactions will include visits to nurses, rehabilitators, specialist, and other healthcare professionals (HCPs), attendance to emergency department, hospitalization, hospitalization days, surgery, and MLD-related procedures. 1 year
Secondary Number of Interactions of Participants With the Healthcare System Post Diagnosis Categorized by Type Interactions will be categorized as: visits to nurses, rehabilitators, specialist, and other HCPs, attendance to emergency department, hospitalization, hospitalization days, surgery, and MLD-related procedures. 1 year
Secondary Number of Participants With Disease Progression Disease progression will be analysed using brain MRI, MR spectroscopy, weight, height, and head circumference, fine motor function, gross motor function, speech function (expressive language), non-verbal communication, musculoskeletal symptoms, eating/drinking ability, cognitive status, social/behavioural function, nerve conduction and sleep quality. 23 years
Secondary Number of Treatment Types Used for MLD and Sequencing 23 years
Secondary Percentage of Participants Under Specific MLD Treatments With Attenuated Disease Progression The percentage of participants under specific MLD treatments with attenuated disease progression will be assessed using the participants' electronic medical records. 23 years
Secondary Number of Participants With Comorbidities and Complications 23 years
Secondary Number of Participants With the Different Types of Symptomatic/Palliative Treatments and Supports 23 years
Secondary Mortality Rate Based on Percentage of Participant-Deaths Per Year Mortality rate is defined as proportion of deaths per year. 23 years
Secondary Proportion of Participant-Deaths Categorized by Cause of Death Proportion of deaths categorized by cause of death will be recorded. 23 years
Secondary Age of Participants With MLD at Death Age of MLD participants at death in years will be recorded. 23 years
Secondary Time From Onset of Symptoms to Death 23 years
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Completed NCT00418561 - Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) Phase 1
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