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A Screening Study Evaluating Disease Status of Gaucher Type I Patients

This study has been completed.
Sponsor:
Information provided by:
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00795197
First received: November 19, 2008
Last updated: December 28, 2009
Last verified: December 2009

November 19, 2008
December 28, 2009
December 2008
December 2009   (final data collection date for primary outcome measure)
Not Provided
  • A gaucher enzyme assay will be performed to confirm deficiency of glucocerebrosidase activity if no historic data is available [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • Demographic and medical history will be recorded [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • A physical examination (including measurement of height and weight) will be performed [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • Vital signs (blood pressure [BP], heart rate [HR], respiratory rate [RR], and temperature) will be measured. [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • Current use of medication and therapies will be recorded. [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • Urine or serum pregnancy testing will be performed on women of child-bearing potential. The radiologist must review the patient's pregnancy test results and document a negative test result prior to performing any radiologic study assessments. [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • Routine clinical laboratory test (serum chemistry, hematology, and urinalysis) will be performed. [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • Total iron binding capacity, ferritin methylmalonic acid (MMA) and homocysteine will be assessed. [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • A neurological examination will be performed. [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • An echocardiogram (ECHO) with Doppler will be performed. [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • A 12-Lead electrocardiogram (ECG) will be performed. [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • Spleen and liver volumes will be assessed using spiral computed tomography (CT) scan or magnetic resonance imaging (MRI) (preferred method). [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • At sites where MRI is not available and where spiral CT is used to measure spleen and liver volumes, then an ultrasound of the liver must be performed to evaluate portal hypertension. [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
  • An MRI of the lumbar spine and entire femora will be performed. [ Time Frame: Approximately 1 week ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00795197 on ClinicalTrials.gov Archive Site
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A Screening Study Evaluating Disease Status of Gaucher Type I Patients
A Screening Protocol to Characterize the Disease Status of Gaucher Type 1 Patients for Potential Inclusion in a Subsequent Phase 3 Clinical Study

A screening study to identify and characterize the disease status of patients with Gaucher Type 1 disease for potential inclusion in a subsequent phase 3 clinical study.

The primary objective of this screening study is to identify and characterize the disease status of patients with Gaucher Type 1 disease for potential inclusion in a subsequent Phase 3 clinical study which will evaluate the efficacy and safety of Genz-112638.

This will be a multi-center, multi-national screening study to assess the disease status of patients with Gaucher Type 1 disease utilizing criteria to be eligible for enrollment in a subsequent phase 3 clinical study. The assessment performed in this study are standard of care for the evaluation of symptoms of Gaucher Type 1 disease. No study drug will be administered in this screening study.

Observational
Not Provided
Not Provided
Retention:   Samples Without DNA
Description:

Blood sample/Urine sample

Non-Probability Sample

Gaucher Disease Type 1

Gaucher Disease, Type 1
Not Provided
Screening Group
Screening Group
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
0
December 2009
December 2009   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • The Patient (or their parent/legal guardian) is willing and able to provide written informed consent.
  • The patient has the following manifestations of Gaucher Type 1 disease identified prior to screening: A. Splenomegaly, B. Hepatomegaly, C. Anemia and /or thrombocytopenia.
  • The patient is 16 to 65 years of age. For patients < 18 years of age, the patient's Tanner stage should be ≥ 4.

Exclusion Criteria:

  • The patient has had a partial splenectomy within 36 months prior to screening or has had a total splenectomy.
  • The patient has received miglustat within 3 months prior to screening.
  • The patient has received enzyme replacement therapy within 9 months prior to screening.
  • The patient is known to have evidence of neurologic (e.g., peripheral neuropathy, tremors, seizures, Parkinsonism, or cognitive impairment) or pulmonary involvement (e.g., pulmonary hypertension) as related to Gaucher Type 1 disease.
  • The patient is known to have 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 periostal elevation, increased white cell count, fever, or immobility of > 3 days) in the 12 months prior to screening , as determined by investigator.
  • The patient is transfusion dependent.
  • The patient has ever had any radiation treatment.
  • The patient is known to have prior esophageal varices or liver infarction.
  • The patient is known to have clinically significant disease, other than Gaucher Type 1 disease, including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic, or psychiatric disease, other medical condition, or serious intercurrent illness.
  • The patient is known to have any of the following: Clinically significant family history (sudden cardiac death in 1st or 2nd degree relatives), cardiac medical history (including myocardial infraction [MI] in the past year), or cardiac assessments/symptoms consistent with ischemia.
  • The patient is known to have any of the following: Specific arrythmias or finding on cardiac monitoring such as severe 1st degree atrioventricular (AV) block, any 2nd or 3rd degree AV block, highly frequent or runs (3 or more) of atrial premature contractions (APC's) or premature ventricular contractions (PVC's), complete left and right as well as partial left bundle branch block, or prolonged QT interval.
  • The patient has previously tested positive for the human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen.
  • The patient has received an investigational product within 30 days prior to screening.
  • The patient has a history of cancer, with the exception of basal cell carcinoma.
  • The patient is pregnant or lactating.
Both
16 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   India
 
NCT00795197
GZGD02808
No
Medical Monitor, Genzyme Corporation
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Genzyme, a Sanofi Company
December 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP