- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01093105
An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)
May 30, 2021 updated by: Shire
A Multicenter, Prospective, Longitudinal, Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (Krabbe Disease)
The objective of this study is to evaluate the natural history of disease progression in infants with globoid cell leukodystrophy (GLD).
Study Overview
Status
Withdrawn
Conditions
Detailed Description
This study is being conducted to gather prospective data on disease progression in infants diagnosed with GLD.
This study will be performed using protocol-defined, standardized assessments including clinical, developmental, and neurologic measures.
All study visits will be conducted in the subject's home.
No travel to the study site is necessary.
Study Type
Observational
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 1 year (Child)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
Subjects with a documented diagnosis of GLD and clinical signs and symptoms consistent with that diagnosis
Description
Inclusion Criteria:
Subjects must meet all of the following criteria to be considered eligible for this study:
- The subject has a documented diagnosis of GLD as evidenced by GALC enzyme activity or a GALC genotype that is predictive of GLD.
The subject must have clinical signs and symptoms consistent with the diagnosis of infantile GLD including at least 2 of the following:
- Chronic difficulty with feeding or unexplained irritability or "fisting" or other signs of abnormal increased tone
- CT or MRI imaging, if performed during diagnostic evaluation prior to enrollment, consistent with GLD
- Failure to meet at least 2 age-specific developmental milestones consistent with GLD
- Loss of deep tendon reflexes or abnormal visual fixation or optic atrophy
- The subject has documented onset of signs and symptoms consistent with GLD at <12 months of age and is <21 months of age at time of study entry.
- The subject was born at a gestational age of 35-41 weeks.
- The subject had a birth weight of ≥2 kg.
- At study entry, the subject must be able to maintain oral nutrition and hydration without the use of supportive measures, defined as use of a feeding tube.
- At study entry, the subject must be able to maintain ventilation without the use of invasive supportive measures, defined as use of a breathing tube.
- The subject must be able, in the opinion of the Investigator, to accommodate the protocol requirements, including feasibility of study visits.
- The subject's parent(s) or legal guardian must have voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the subject's parent(s), or legal guardian.
Exclusion Criteria: Subjects who meet any of the following criteria are not eligible for this study:
- The subject has neurologic, hearing or vision impairment, difficulty swallowing or feeding, respiratory complications, behavioral disturbances, or other medical conditions that are not due to GLD and are likely to confound the scientific integrity or interpretation of study assessments, as determined by the Investigator.
- The subject has received treatment with any investigational drug or a device within the 30 days prior to study enrollment through study completion.
- The subject has received a cord blood or bone marrow transplant or is planning to receive one during the study.
- The subject's parent(s) or legal guardian is unable to understand the nature, scope, and possible consequences of the study, or does not agree to comply with the protocol defined schedule of assessments.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
To measure the change from baseline in growth parameters (eg, weight gain, linear growth, head circumference).
Time Frame: 1 year
|
1 year
|
To determine the onset date of inadequate oral nutrition, hydration, and/or ventilation as a biomarker for survival
Time Frame: 1 year
|
1 year
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
To assess the change from baseline in clinical parameters of GLD disease progression from a standardized infant neurological examination and infant distress scales.
Time Frame: 1 year
|
1 year
|
To assess the change from baseline in clinical parameters described in Hagberg's clinical staging.
Time Frame: 1 year
|
1 year
|
To measure the time to absolute survival
Time Frame: 1 year
|
1 year
|
To assess the AE experience in this patient population
Time Frame: 1 year
|
1 year
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Anticipated)
April 1, 2010
Primary Completion (Anticipated)
May 1, 2012
Study Completion (Anticipated)
May 1, 2012
Study Registration Dates
First Submitted
March 24, 2010
First Submitted That Met QC Criteria
March 24, 2010
First Posted (Estimate)
March 25, 2010
Study Record Updates
Last Update Posted (Actual)
June 3, 2021
Last Update Submitted That Met QC Criteria
May 30, 2021
Last Verified
May 1, 2021
More Information
Terms related to this study
Keywords
- Krabbe Disease
- Leukodystrophy, Globoid Cell
- Classic Globoid Cell Leukodystrophy
- Early-Onset Globoid Cell Leukodystrophy
- Globoid Cell Leukodystrophy
- Globoid Cell Leukoencephalopathy
- Infantile Globoid Cell Leukodystrophy
- Late-Onset Globoid Cell Leukodystrophy
- Leukodystrophy, Globoid Cell, Classic
- Leukodystrophy, Globoid Cell, Early-Onset
- Leukodystrophy, Globoid Cell, Infantile
- Leukodystrophy, Globoid Cell, Late-Onset
- Psychosine
- galactosylsphingosine (psychosine) beta-galactosidase
- Psychosine-UDP galactosyltransferase
- Krabbe Leukodystrophy
- Krabbe's Disease
- Krabbe's Leukodystrophy
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Demyelinating Diseases
- Genetic Diseases, Inborn
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Leukoencephalopathies
- Hereditary Central Nervous System Demyelinating Diseases
- Leukodystrophy, Globoid Cell
Other Study ID Numbers
- HGT-GLD-056
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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