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 range8 Years

SexALL

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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

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