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Clinical Trials/CTRI/2009/091/000695
CTRI/2009/091/000695
Completed
未知

A screening protocol to characterize the disease status of Gaucher Type I patients for potential inclusion in a subsequent phase 3 clinical study.

Genzyme Europe BV0 sites100 target enrollmentTBD
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Overview

Phase
未知
Intervention
Not specified
Conditions
Not specified
Sponsor
Genzyme Europe BV
Enrollment
100
Status
Completed
Last Updated
last year
OverviewInvestigatorsEligibility CriteriaOutcomesSimilar Trials

Overview

Brief Summary

No summary available.

Registry
who.int
Start Date
TBD
End Date
TBD
Last Updated
last year
Study Type
Observational

Investigators

Eligibility Criteria

Inclusion Criteria

  • •1\) The patient (or their parent/legal guardian) is willing and able to provide written informed consent.
  • •2\) The patient has the following manifestations of Gaucher Type 1 disease identified prior to
  • •A. Splenomegaly
  • •B. Hepatomegaly
  • •C. Anemia and/or thrombocytopenia
  • •3\) The patient is 16 to 65 years of age. For patients \<18 years of age, the patient?s Tanner stage should

Exclusion Criteria

  • •The patient has had a partial splenectomy within 36 months prior to screening or has had a total
  • •splenectomy.
  • •2\) The patient has received miglustat within 3 months prior to screening.
  • •3\) The patient has received enzyme replacement therapy within 9 months prior to screening.
  • •4\) The patient is known to have any evidence of neurologic (e.g., peripheral neuropathy, tremor,
  • •seizures, Parkinsonism, or cognitive impairment) or pulmonary involvement (e.g., pulmonary
  • •hypertension) as related to Gaucher Type 1 disease.
  • •5\) The patient has documentation of new pathological bone involvement (e.g. osteonecrosis,
  • •pathological fractures, etc.) or has a bone crisis (pain with acute onset which requires
  • •immobilization of the affected area, narcotics for relief of pain, and may be accompanied by

Outcomes

Primary Outcomes

Not specified

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