WithdrawnNot Applicable

An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)

Stopped: Study halted prior to enrollment of first participant

0Started 2010-04-01

Plain-language summary

The objective of this study is to evaluate the natural history of disease progression in infants with globoid cell leukodystrophy (GLD).

Who can participate

Age range

21 Months

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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.

Exclusion criteria

. 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

To measure the change from baseline in growth parameters (eg, weight gain, linear growth, head circumference).

Timeframe: 1 year

To determine the onset date of inadequate oral nutrition, hydration, and/or ventilation as a biomarker for survival

Timeframe: 1 year

Trial details

NCT IDNCT01093105

SponsorShire

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2012-05-01

View on ClinicalTrials.gov More Leukodystrophy, Globoid Cell trials