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Enzyme Replacement Therapy

Enzyme Therapy for Gaucher Disease

Phase 2

Waitlist Available

Research Sponsored by National Center for Research Resources (NCRR)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Gaucher disease with glucocerebrosidase deficiency confirmed by enzymatic or molecular assay

No neurologic disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Study Summary

This trial is testing an enzyme therapy for patients with Gaucher disease, to see if it is effective and safe.

Who is the study for?

This trial is for individuals with Gaucher disease confirmed by specific tests, who have at least three affected organ systems such as anemia, low platelet count, enlarged organs, bone issues visible on X-ray, lung problems or symptoms that impact daily life or survival.Check my eligibility

What is being tested?

The study is testing the effectiveness and safety of glucocerebrosidase enzyme therapy in treating patients with Gaucher disease. It's a phase II trial which means it focuses on how well the treatment works and monitors side effects.See study design

What are the potential side effects?

While not specified here, common side effects of enzyme replacement therapies like glucocerebrosidase may include allergic reactions, headache, dizziness, abdominal pain and discomfort at the infusion site.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have Gaucher disease confirmed by a specific test.

Select...

I do not have any neurological diseases.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Center for Research Resources (NCRR)Lead Sponsor

537 Previous Clinical Trials

316,992 Total Patients Enrolled

1 Trials studying Gaucher Disease

15 Patients Enrolled for Gaucher Disease

University of PittsburghOTHER

1,719 Previous Clinical Trials

16,342,317 Total Patients Enrolled

3 Trials studying Gaucher Disease

1,688 Patients Enrolled for Gaucher Disease

John BarrangerStudy ChairUniversity of Pittsburgh

1 Previous Clinical Trials

15 Total Patients Enrolled

1 Trials studying Gaucher Disease

15 Patients Enrolled for Gaucher Disease

Media Library

Glucocerebrosidase (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT00004293 — Phase 2

Gaucher Disease Clinical Trial 2023: Glucocerebrosidase Highlights & Side Effects. Trial Name: NCT00004293 — Phase 2

Glucocerebrosidase (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT00004293 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the governmental regulations surrounding this medication?

"While there is some evidence to support the safety of this phase 2 trial's intervention, its efficacy has not been explored yet."

Answered by AI

Are young adults welcome in this clinical trial?

"The age limit to enroll in this trial is set at 65 years old. There are 7 separate studies for patients that are younger than the specified age and 11 for patients that meet or exceed the age limit."

Answered by AI

How does one go about enrolling in this clinical trial?

"Eligibility for this research study requires that patients have a diagnosis of gaucher disease and are between 18-65 years old. Around 24 individuals are needed in total."

Answered by AI

Are there any still participating in this research?

"According to the most recent data on clinicaltrials.gov, this trial is not currently enrolling patients. The listing was created on November 1st, 1999 and has not been updated since June 23rd, 2005. There are 16 other ongoing studies that participants can consider signing up for."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

1999. "Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT00004293.

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