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Selkäytimeen annettu entsyymikorvaus mukopolysakkaridoosi I:n selkäytimen puristuman hoidossa. Kansainvälinen monikeskustutkimus. A study of intrathecal enzyme replacement therapy for spinal cord compression in mucopolysaccharidosis I, MIRC-001. - Intrathecal enzyme replacement in MPS I

Selkäytimeen annettu entsyymikorvaus mukopolysakkaridoosi I:n selkäytimen puristuman hoidossa. Kansainvälinen monikeskustutkimus. A study of intrathecal enzyme replacement therapy for spinal cord compression in mucopolysaccharidosis I, MIRC-001. - Intrathecal enzyme replacement in MPS I

Status
Not yet recruiting
Phases
Unknown
Study type
Interventional
Source
EU CTR
Registry ID
EUCTR2006-005216-27-FI
Enrollment
10
Registered
2007-03-14
Start date
2007-03-26
Completion date
Unknown
Last updated
2012-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mucopolysaccharidosis I H/S, lysosomal storage disease with clinical manifestations such as progressive joint stiffness, growth retardation, corneal clouding, hepatosplenomegaly, cardiac and respiratory dysfunction, and in severe fore, mental retardation MedDRA version: 9.1 Level: LLT Classification code 10056887 Term: Mucopolysaccharidosis IH/S

Interventions

Trade Name: Aldurazyme Pharmaceutical Form: Other descriptive name: LARONIDASE

Sponsors

HUS, Hospital for Children and Adolescents
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion criteria

Inclusion criteria: Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria:

Design outcomes

Primary

MeasureTime frame
Main Objective: ;Secondary Objective: ;Primary end point(s):—

Countries

Finland

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026