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The SHP-GCB-402 Study for Gaucher Disease in Rochester, MN

The SHP-GCB-402 study for Gaucher disease is evaluating the effect velaglucerase alfa (VPRIV®) has on bone pain and bone density in people with type I Gaucher disease.

 

Phase: – IV

Health Condition: – Gaucher’s disease

Sponsored by: FCBVIO

Stipend: Contact Research Site Directly for Stipend

Length of Study: 102 Weeks

Additional Qualifying Information / Important Inclusions or Exclusions: 

Gender: Both

Age: 18 – 65

Washout Period: N/A

In-patient or stay overnight at the research center?: NO

 

Eligibility:

  • Be between 18 and 65 years of age (inclusive)

  • Have type I Gaucher disease

  • Not have received enzyme replacement therapy or substrate reduction therapy in the past year

  • Not have received any osteoporosis-specific treatment in the past year

Rochester, MN 59905, United States.

 

Study Scavenger Provides the Below Helpful Information

What Is Gaucher Disease?

Gaucher disease is one of the inherited metabolic disorders known as lipid storage diseases. Lipids are fatty materials that include oils, fatty acids, waxes, and steroids (such as cholesterol and estrogen). Gaucher disease is caused by a deficiency of the enzyme glucocerebrosidase. Fatty materials can accumulate in the brain, spleen, liver, lungs, bone marrow, and kidneys.

Gaucher disease can affect anyone, occurring in up to 1 in 40,000 live births in the general population. Gaucher disease is more common among Jews of Ashkenazi (Eastern European) descent, occurring in approximately 1 in 450 within this population. As many as 1 in 10 may carry the mutated gene responsible for Gaucher disease.

Symptoms may begin in early life or adulthood and include skeletal disorders and bone lesions that may cause pain and fractures, enlarged spleen and liver, liver malfunction, anemia, and yellow spots in the eyes. Signs of Gaucher disease type 1, the most common form in western countries, can vary widely from person to person. Some people experience severe symptoms, while others have none at all. If you have Gaucher disease, you may have symptoms such as:

– Swollen belly due to spleen and liver enlargement
– Bone pain and easily fractured bones
– Anemia (low blood counts) and fatigue
– Bleeding and bruising problems

 –  National Institute of Health

In-Patient/Out-Patient Explained

There are two main reasons clinical research trials are done in an isolated inpatient setting where the patient stays overnight at a research clinic: 1) To protect the study participant:   Some study agents, usually challenge products, can make some people quite sick.  Study participants stay on the inpatient unit to receive 24 hour nursing care during these types of studies. Participants stay on the isolation unit until their laboratory test show they are no longer shedding the infectious agent.  2) To protect the public:  An infectious study agent that is given to a study participant may be harmful to the general public’s health.  Therefore, study participants stay on the inpatient unit until the possibility of giving the infection to someone else has passed.

An outpatient vaccine trial is a study that does not require an overnight stay in the hospital or research clinic.  Participants are given an investigational agent and have scheduled clinic visits to monitor how well they tolerate the investigational agent. Most studies require the clinic staff to follow up with subjects on a daily, weekly or monthly schedule.  Clinic visits can range from 2 to 4 hours, depending on the study and the type of visit.  –  National Institute of Health

Screening Visit

Screening Visit:  Potential candidates for a Study will participate in an initial screening visit to determine if they are appropriate for the Study.  When you first attend the screening visit for phases I-III, the study is explained in detail and you can ask any questions. If you decide to participate, the study nurse will review the informed consent form with you and ask for your signature and permission to proceed with the screening evaluations. –  –  National Institute of Health

Washout Period Explained

A period during a clinical trial when the trial subjects receive no active medication. The wash-out period is typically used to generate patient baseline data.   Every clinic has a washout period during which you are not allowed to do a study at the same clinic or any other clinic.

The minimum period is 30 days since last dose, however some washouts go based on last study procedure (such as out patient visits or how much blood you gave during the study).   The washout period may vary from study to study depending on the half-life of the drug. Certain studies like radio-labeled drugs will have a 1 year washout before you can do another radio-labeled drug.   In addition to the clinic’s washout policy, the sponsor may impose a longer washout period.  Some studies will have a longer washout before you can sign up.

In general, you can sign up and screen for another study during the washout period as long as the check in date is at least 30 days after your last dose in the previous study.  However, depending on how fast your body recovers from a study, you may want to wait the full period to avoid problems with screening.

People who screen too soon after a study typically have lower red blood cell, hemoglobin, hematocritand iron.  If these levels are too low, you could get banned from participating in studies because having low levels is unhealthy and unsafe for participating in a study.

Be forewarned that many clinic participate in the VCT (Verified Clinical Trials) program which tracks when you do a study to ensure the next study meets washout periods.  I do not mention these clinics because it shouldn’t matter.  You know the rules and if you break them, the consequences can be dire like being banned from a clinic.  Sometimes the only way to drive this message home is to allow people to find out the hard way. –  –  National Institute of Health