Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Bone Response to Enzyme Replacement in Gaucher's Disease

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: NCT00001416
Recruitment Status : Completed
First Posted : December 10, 2002
Last Update Posted : September 22, 2016
Sponsor:
Information provided by:
National Institutes of Health Clinical Center (CC)

Tracking Information
First Submitted Date  ICMJE November 3, 1999
First Posted Date  ICMJE December 10, 2002
Last Update Posted Date September 22, 2016
Study Start Date  ICMJE December 1993
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE Not Provided
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00001416 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Outcome Measures  ICMJE Not Provided
Original Other Outcome Measures  ICMJE Not Provided
 
Descriptive Information
Brief Title  ICMJE Bone Response to Enzyme Replacement in Gaucher's Disease
Official Title  ICMJE Skeletal Responses to Macrophage-Targeted Glucocerebrosidase in Patients With Type 1 Gaucher's Disease
Brief Summary

The purpose of this study is to examine how the skeleton responds to repeated doses of enzyme replacement therapy in patients with type I Gaucher's disease who have had their spleens removed.

Gaucher disease is a lysosomal storage disease resulting from glycocerebroside accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may occur in adults but occurs most severely in infants, in whom cerebroside also accumulates in neurons. Patients with Gaucher's disease experience enlargement of the liver and spleen and bone destruction. The condition is passed from generation to generation through autosomal recessive inheritance.

Type I is the most common form. It is a chronic non-neuronopathic form, meaning the disease does not affect nerve cells. The symptoms of type I can appear at any age.

In this study patients will be divided into three groups. Each group will receive different doses of enzyme replacement (Ceredase). In addition, two of the three groups will also receive doses of a form of vitamin D (calcitriol). Researchers believe the groups receiving vitamin D will have an improved response as compared to those patients only receiving enzyme replacement.

Patients in each group who respond to enzyme replacement with increases in bone density will be compared to the other treatment groups.

Detailed Description

The purpose of this study is to examine the response of the skeleton to repeated infusions of macrophage-targeted glucocerebrosidase (CEREDASE (Trademark) ) in splenectomized patients with type I Gaucher's disease. The magnitude and rate of development of the skeletal response will be monitored non-invasively. Theoretically, an enhanced response should occur in patients supplemented with pharmacologic doses of 1, 25-dihydroxyvitamin D3 (calcitriol), since calcium absorption and enzyme delivery to bone marrow macrophages should be increased in this setting. These issues will be addressed in a clinical trial that uses a modified factorial design. A total of 57 patients will be assigned to three treatment groups by block randomization.

Group 1: CEREDASE (Trademark) (60 IU/kg q2wks; 0-6 months)

CEREDASE (Trademark) (30 IU/kg q2wks; 7-24 months)

Group 2: Calcitriol (0.25-3.0 micrograms/day; 0-24 months)

CEREDASE (Trademark) (60 IU/kg q2wks; 7-12 months)

CEREDASE (Trademark) (30 IU/kg q2wks; 13-24 months)

Group 3: Calcitriol (0.25-3.0 micrograms/day; 0-24 months)

CEREDASE (Trademark) (60 IU/kg q2wks; 0-6 months)

CEREDASE (Trademark) (30 IU/kg q2wks; 7-24 months)

The number of patients responding to enzyme replacement with a significant decrease in hepatic volume and a significant increase in trabecular bone density of the lumbar spine will be compared between the treatment groups.

Study Type  ICMJE Interventional
Study Phase Phase 2
Study Design  ICMJE Primary Purpose: Treatment
Condition  ICMJE Gaucher's Disease
Intervention  ICMJE Drug: CEREDASE™
Study Arms Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Enrollment  ICMJE
 (submitted: June¬†23,¬†2005)
100
Original Enrollment  ICMJE Same as current
Study Completion Date November 2000
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

Splenectomized Gaucher patients.

Aged 18-45 who have not received enzyme therapy for at least 1 year.

No patients with other illnesses (pulmonary, liver, kidney, bone, hematologic).

Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00001416
Other Study ID Numbers  ICMJE 940050
94-N-0050
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE National Institute of Neurological Disorders and Stroke (NINDS)
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date September 2016

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