A Screening Study Evaluating Disease Status of Gaucher Type I Patients

Gaucher Disease, Type 1 Clinical Trial

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT00795197
• Other study ID #: GZGD02808
• Status: Withdrawn
• Phase: N/A
• First received: November 19, 2008
• Last updated: May 7, 2015
• Start date: December 2008
• Est. completion date: December 2009

Study information

• Verified date: May 2015
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: India: Drugs Controller General of India
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: December 2009
• Est. primary completion date: December 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years to 65 Years

Eligibility

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.

Study Design

N/A

Related Conditions & MeSH terms

• Gaucher Disease
• Gaucher Disease, Type 1

Locations

Country	Name	City	State
India	Sanjay Gandhi Postgraduate Institute of Medical Sciences (SGPGI)	Lucknow
India	Jaslok Hospital and Research Center	Mumbai
United States	The Mount Sinai School of Medicine, Department of Genetics and Genomic Sciences	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

United States,  India,