Leukodystrophy, Globoid CellNCT01093105
An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)
An observational study of Leukodystrophy, Globoid Cell, run by Shire.
Trial summary
The objective of this study is to evaluate the natural history of disease progression in infants with globoid cell leukodystrophy (GLD). Read more about this study
- Who is it for?
- Ages Up to 21 months
- Conditions the study is for
- Leukodystrophy, Globoid Cell
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Apr 2010to 1 May 2012
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 months
- All sexes
- No healthy volunteers
- Child
Study population: Subjects with a documented diagnosis of GLD and clinical signs and symptoms consistent with that diagnosis
Who may be able to join
- 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
Show all 19Show fewer
- 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.
Who cannot join
None listed.
About this study
Official title: 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).
Read the 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.
- Conditions
- Leukodystrophy, Globoid Cell
- Keywords
- 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 Disease, Krabbe Leukodystrophy, Krabbe's Disease, Krabbe's Leukodystrophy
- Medical subject headings
- Leukodystrophy, Globoid Cell
What is being measured
The primary outcome is the main question the study is designed to answer.
To measure the change from baseline in growth parameters (eg, weight gain, linear growth, head circumference).
Measured: 1 year
To determine the onset date of inadequate oral nutrition, hydration, and/or ventilation as a biomarker for survival
Measured: 1 year
Secondary outcomes (4)
To assess the change from baseline in clinical parameters of GLD disease progression from a standardized infant neurological examination and infant distress scales.
Measured: 1 year
To assess the change from baseline in clinical parameters described in Hagberg's clinical staging.
Measured: 1 year
To measure the time to absolute survival
Measured: 1 year
To assess the AE experience in this patient population
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2010 (estimated)
- Study start1 Apr 2010 (estimated)
- Primary completion1 May 2012 (estimated)
- Study completion1 May 2012 (estimated)
Why it stopped: Study halted prior to enrollment of first participant
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukodystrophy, Globoid CellStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- PharmaNet, Nextrials, Inc.
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01093105
- Sponsor's protocol ID
- HGT-GLD-056
- Status last verified
- May 2021
- First submitted
- 24 Mar 2010
- Last updated
- 3 Jun 2021
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Prospective, Longitudinal, Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (Krabbe Disease)
- ClinicalTrials.gov ID
- NCT01093105
- Acronym / sponsor ID
- HGT-GLD-056
- Conditions
- Leukodystrophy, Globoid Cell
- Keywords
- 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
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: Study halted prior to enrollment of first participant
- Enrolment
- 0 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Collaborators
- PharmaNet, Nextrials, Inc.
- Study start
- 1 Apr 2010 (estimated)
- Primary completion
- 1 May 2012 (estimated)
- Study completion
- 1 May 2012 (estimated)
- First posted
- 25 Mar 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to evaluate the natural history of disease progression in infants with globoid cell leukodystrophy (GLD).
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 21 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Subjects with a documented diagnosis of GLD and clinical signs and symptoms consistent with that diagnosis
Key inclusion criteria (19 listed)
- 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:
- 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.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To measure the change from baseline in growth parameters (eg, weight gain, linear growth, head circumference).
Time frame: 1 year
To determine the onset date of inadequate oral nutrition, hydration, and/or ventilation as a biomarker for survival
Time frame: 1 year
4 secondary outcomes
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
To assess the change from baseline in clinical parameters described in Hagberg's clinical staging.
Time frame: 1 year
To measure the time to absolute survival
Time frame: 1 year
To assess the AE experience in this patient population
Time frame: 1 year
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
Questions about this trial
What is this clinical trial studying?
An observational study of Leukodystrophy, Globoid Cell, run by Shire.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 3 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To measure the change from baseline in growth parameters (eg, weight gain, linear growth, head circumference). (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2021.