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A Screening Study Evaluating Disease Status of Gaucher Type I Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00795197
Recruitment Status : Withdrawn (No recruitment of patients)
First Posted : November 21, 2008
Last Update Posted : May 8, 2015
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date November 19, 2008
First Posted Date November 21, 2008
Last Update Posted Date May 8, 2015
Study Start Date December 2008
Actual Primary Completion Date December 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures Not Provided
Original Primary Outcome Measures
 (submitted: November 19, 2008)
  • A gaucher enzyme assay will be performed to confirm deficiency of glucocerebrosidase activity if no historic data is available [ Time Frame: Approximately 1 week ]
  • Demographic and medical history will be recorded [ Time Frame: Approximately 1 week ]
  • A physical examination (including measurement of height and weight) will be performed [ Time Frame: Approximately 1 week ]
  • Vital signs (blood pressure [BP], heart rate [HR], respiratory rate [RR], and temperature) will be measured. [ Time Frame: Approximately 1 week ]
  • Current use of medication and therapies will be recorded. [ Time Frame: Approximately 1 week ]
  • 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 ]
  • Routine clinical laboratory test (serum chemistry, hematology, and urinalysis) will be performed. [ Time Frame: Approximately 1 week ]
  • Total iron binding capacity, ferritin methylmalonic acid (MMA) and homocysteine will be assessed. [ Time Frame: Approximately 1 week ]
  • A neurological examination will be performed. [ Time Frame: Approximately 1 week ]
  • An echocardiogram (ECHO) with Doppler will be performed. [ Time Frame: Approximately 1 week ]
  • A 12-Lead electrocardiogram (ECG) will be performed. [ Time Frame: Approximately 1 week ]
  • 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 ]
  • 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 ]
  • An MRI of the lumbar spine and entire femora will be performed. [ Time Frame: Approximately 1 week ]
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Screening Study Evaluating Disease Status of Gaucher Type I Patients
Official Title A Screening Protocol to Characterize the Disease Status of Gaucher Type 1 Patients for Potential Inclusion in a Subsequent Phase 3 Clinical Study
Brief Summary 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.
Detailed Description

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.

Study Type Observational
Study Design Not Provided
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples Without DNA
Description:
Blood sample/Urine sample
Sampling Method Non-Probability Sample
Study Population Gaucher Disease Type 1
Condition Gaucher Disease, Type 1
Intervention Not Provided
Study Groups/Cohorts Screening Group
Screening Group
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Withdrawn
Actual Enrollment
 (submitted: December 28, 2009)
0
Original Estimated Enrollment
 (submitted: November 19, 2008)
100
Actual Study Completion Date December 2009
Actual Primary Completion Date December 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria

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.
Sex/Gender
Sexes Eligible for Study: All
Ages 16 Years to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries India,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00795197
Other Study ID Numbers GZGD02808
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Sanofi ( Genzyme, a Sanofi Company )
Study Sponsor Genzyme, a Sanofi Company
Collaborators Not Provided
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
PRS Account Sanofi
Verification Date May 2015