A Phase 1 interventional study of laronidase in Spinal Cord Compression, Mucopolysaccharidosis I and Hurler-Scheie Syndrome, sponsored by Patricia I. Dickson, M.D.. Terminated at 2 sites in 2 countries. Open to participants aged 8 Years and older. Per ClinicalTrials.gov, last updated 2013-02-21.
Sponsored by Patricia I. Dickson, M.D. · Phase 1, Interventional, and Treatment
This is a one-year extension study of the use of laronidase into the spinal fluid to treat spinal cord compression in mucopolysaccharidosis I. Mucopolysaccharidosis I is a rare genetic condition due to deficiency of the enzyme alpha-l-iduronidase. Spinal cord compression occurs in this condition due to accumulation of material called glycosaminoglycans (GAG). Laronidase is the manufactured form of the enzyme alpha-l-iduronidase that is deficient in mucopolysaccharidosis I patients. The aim of this study is to determine whether laronidase is safe and effective when given into the spinal fluid as a potential non-surgical treatment for spinal cord compression due to mucopolysaccharidosis I disease. Funding Source -- FDA OOPD
Enzyme replacement therapy (ERT) has been developed for mucopolysaccharidosis I (MPS I), a lysosomal storage disorder. ERT helps many physical ailments due to the disease, but does not treat the central nervous system, due to inability to cross the blood brain barrier. Our purpose is to test delivery of ERT to the spinal fluid via intrathecal injection in patients with MPS I. In this pilot study, we will use recombinant human α-L-iduronidase administered intrathecally once per month for four months to individuals with the Hurler-Scheie and Scheie forms of MPS I and spinal cord compression. If successful, intrathecal delivery could represent a practical, straightforward method of treating central nervous system disease due to lysosomal storage.
52 studies on the registry are indexed under Spinal Cord Compression; 19 are open to participants now.
This study's enrollment of 3 is below the median of 43 across 35 interventional studies indexed under Spinal Cord Compression.
Browse Spinal Cord Compression studies →Patricia I. Dickson, M.D. is the lead sponsor of 3 studies on the registry; none are open to participants now.
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Hurler-Scheie, Scheie form of MPS I, or Hurler 2 years after hematopoietic stem cell transplantation
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A drug or device being studied in a clinical trial.
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Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
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It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
drug laronidase, dose 1.74 mg, route intrathecal, frequency every 30-90 days, duration 1 year
Drug: laronidase
1.74 mg intrathecally every 1-3 months for 1 year
Also known as: Aldurazyme, recombinant human alpha-l-iduronidase
safety of intrathecal enzyme treatment by blood and spinal fluid tests; improvement or stabilization in neurologic signs and symptoms of spinal cord compression
Time frame: 1 year
The record gives a start of Jan 2008, a primary completion of Oct 2011 and a completion of Oct 2011. It does not say whether these dates are actual or estimated.
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This study is terminated, as verified in Feb 2013. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Laronidase