JPRN-jRCTs071190008
Recruiting
Phase 2
Study of Intrathecal or Intracerebroventricular administration of Cyclodextrin for Niemann-Pick disease Type C - ITaC study
Matsuo Muneaki0 sites3 target enrollmentMay 17, 2019
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- iemann-Pick disase, type C
- Sponsor
- Matsuo Muneaki
- Enrollment
- 3
- Status
- Recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\)Genetically proven Niemann\-Pick disease type C
- •2\)From two year\-old to fifteen year\-old patient with apparent neurological symptom.
- •3\)Possibility of regular intrathecal administration by lumbar puncture.
- •4\)Possibility of repeated regular visits to Saga University Hospital.
- •5\)Miglustat should be taken at least three months with a constant dosage before the baseline assessment.
- •6\)The patient or the guardian understand poor life prognosis of this intractable disease and no established treatment for it.
- •7\)The patient or the guardian understand that the safety and efficacy of HPBCD is not established,and unexpected adverse events which can lead to a life threatening sequelae could be occurred.
- •8\)The patient or guardian understand risks for lumber puncture.
- •9\)The patient or guardian understand that this treatment will be done in a limited period.
Exclusion Criteria
- •1\)The age at registration is less than two year\-old or more than sixteen year\-old.
- •2\)No neurological symptoms.
- •3\)Past history of hypersensitivity reaction to cyclodextrin or its additives.
- •4\)Suspected central nervous system infection or systemic infection.
- •5\)Difficulty of lumbar puncture due to spinal anomaly.
- •6\)Skin infection of the lumbar puncture site.
- •7\)Neutropenia (less than 1,000/mm3\)
- •8\)Thrombocytopenia (less than 50,000/mm3\)
- •9\)Circulation abnormality in cerebrospinal fluid.
- •10\)Use of anti\-coagulants or bleeding tendancy.
Outcomes
Primary Outcomes
Not specified
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