A Phase 1 interventional study of laronidase in Mucopolysaccharidosis I, Lysosomal Storage Diseases and Spinal Cord Compression, 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
The investigators are studying the use of enzyme replacement therapy into the spinal fluid for treatment of spinal cord compression in the Hurler-Scheie and Scheie forms of mucopolysaccharidosis I (MPS I). 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.
74 studies on the registry are indexed under Mucopolysaccharidosis I; 7 are open to participants now.
This study's enrollment of 4 is below the median of 14 across 47 interventional studies indexed under Mucopolysaccharidosis I.
Browse Mucopolysaccharidosis I 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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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.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
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.
laronidase dose 1.74 mg, route intrathecal, frequency every 30 days, duration three months
Drug: laronidase
0.58 mg/ml solution for intravenous injection, dose 1.74 mg intrathecally once per month for four injections.
Also known as: Aldurazyme
safety of intrathecal enzyme treatment by blood and spinal fluid tests each month
Time frame: four months
improvement in spinal cord compression due to mucopolysaccharidosis I
Time frame: four months
The record gives a start of Nov 2005, a primary completion of Oct 2011 and a completion of Oct 2011. It does not say whether these dates are actual or estimated.
How these figures are worked out, and which studies are left out of them.
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
Patricia I. Dickson, M.D.