1011003357 in Vitro Expansion and Gene Transfer Using Peripheral Blood Stem Cells

SG

Scott Goebel, MD

Principal Investigator

Status: Enrolling By Invitation Ages: 0 Years - 40 Years Gender: All Genders Phase: N/A 2 Locations

Brief Description

The purpose of this study is to determine whether the quantity of peripheral blood stem cells can be made greater in the laboratory. Healthy male and female volunteer donors between the ages of 18-40 years will be used as a source of normal peripheral blood hematopoietic stem cells (HSC). Health will be assessed by history and physical. Volunteer donors between the ages of 18-40 years with X-linked chronic granulomatous disease will also be used as a source of peripheral blood HSC. X-CGD donors are likely to be male because of the sex-linked inheritance of this disease.

THIS STUDY IS ENROLLING BY INVITATION ONLY - Patients with CGD are typically identified in childhood, and Pediatric Hematology/Oncology physicians tend to continue to follow these patients into adulthood due to this being a relatively rare condition that internal medicine providers do not commonly see. We limited the study to patients over 18 yo so they can provide their own consent. All subjects would be seen and collected under the Pediatric Hematology/Oncology group’s care. Family members or more typically laboratory staff are identified for the normal volunteer donors, also all adults.

Detailed Description

The subject will be asked to take a drug called G-CSF (granulocyte colony stimulating factor) for 3-6 days prior to the procedure to collect cells. G-CSF is a natural human protein that causes the production of granulocytes (infection fighting white blood cells) in the bone marrow. The G-CSF is given subcutaneously (injection under the skin) daily for 3-6 days prior to the collection of the cells. The G-CSF will either be given at the clinic or at home.

If the subject receives G-CSF in the clinic, the subject will need to come to Riley every day during this period. 

If the subject will take the G-CSF at home, the subject will give the injections to themselves. The study staff will teach subjects how to do this and give them more information on how the G-CSF should be stored.  This helps the cells move from the bone marrow to the blood. The subject will need to come to Riley for an outpatient visit prior to the start of the study to complete a blood count (CBC) that will be drawn to check the number of white blood cells (to fight infection), red blood cells (help carry oxygen through the body) and platelets (help blood to clot) before getting G-CSF and before and after the procedure to collect the cells. Blood test(s) may require up to 30 ml (approximately 2 tablespoons) of blood. Also at this visit prior to the start of study all females will be required to have a urine pregnancy test. The nature of the procedure to collect the cells is called leukapheresis (or "apheresis"). Blood is removed through a needle inserted into the vein in one arm where it will pass through tubing attached to the cell separating machine where the blood is separated. Centrifugation is the actual "spinning down" or separation of the various parts of the blood. Mostly white blood cells, some platelets, a small amount of red blood cells and some plasma (the liquid component of the blood) will be collected. The centrifuge instrument will remove some of the white cells and platelets (cells used to form clots) and some plasma (the liquid component of the blood). The total amount of cells and plasma removed will be approximately 200-250 ml or approximately 6-8 fluid ounces. The remainder of the blood will be returned through another needle inserted into the vein of the other arm. The procedure as described will require three to four hours and will be done as an out-patient visit in the Riley Apheresis Center.

Eligibility of study

Inclusion Criteria
Healthy male and female volunteer donors
Volunteer donors with X-linked chronic granulomatous disease

Exclusion Criteria
Pregnancy or nursing mothers
Abnormal baseline CBC, differential or platelet count
Unresolved bacterial or fungal infection (for CGD).
Concurrent use of medications which adversely affect platelet function, such as aspirin and non-steroidal antiflammatory agents.

Interested in participating?

Enrollment in IU School of Medicine clinical trials is managed through our All IN for Health program. Many studies only accept people who are part of an invited group. Visit allinforhealth.info to connect with the study team, or contact your doctor to see if you are eligible.