TerminatedPhase 1

Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

Stopped: Due to slow enrolment.

United States, Finland3 participantsStarted 2008-01

Plain-language summary

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

Who can participate

Age range

8 Years

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: * Hurler-Scheie, Scheie form of MPS I, or Hurler 2 years after hematopoietic stem cell transplantation * Spinal cord compression * Has received intrathecal laronidase previously with good response and no significant safety concerns * Age greater than 8 years * Able to provide legal informed consent * Aware of clinical treatment option of observation without treatment or surgical decompression * Negative urine pregnancy test at screening (nonsterile females of child-bearing potential who are sexually active only) Exclusion Criteria: * Severe (Hurler) form of MPS I * Desires surgical or medical treatment of spinal cord compression * Spinal cord compression that warrants immediate surgical intervention * Pregnancy or lactation * Hematopoietic stem cell transplantation within 2 years of study enrollment * Receipt of an investigational drug within 30 days of enrollment * Infusion reactions to laronidase that required medical intervention, prophylaxis, or altered enzyme administration * Significant anti-iduronidase antibody titer * Recent initiation of intravenous laronidase (within past 6 months) * Presence of cervical subluxation or similar external pathology as the major cause of cord compression symptoms for which surgical intervention should be immediately undertaken

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

safety of intrathecal enzyme treatment by blood and spinal fluid tests; improvement or stabilization in neurologic signs and symptoms of spinal cord compression

Timeframe: 1 year

Trial details

NCT IDNCT00786968

SponsorPatricia I. Dickson, M.D.

Sponsor typeINDIV

Study typeINTERVENTIONAL

Primary completion2011-10

View on ClinicalTrials.gov More Spinal Cord Compression trials