Clinical trial NCT00501228: Tissue Repair in Stem Cell Transplant Recipients
Official title: RhG-CSF (Filgrastim) Treatment of Severe Epithelial/Endothelial or Solid Organ-Specific Tissue Damage In Stem Cell Transplant Recipients
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Graft-Versus-Host Disease
Keywords: Graft-Versus-Host Disease
Stem Cell Transplantation
Tissue Repair
Organ-Specific Tissue Damage
Filgrastim
RhG-CSF
Lead sponsor: M.D. Anderson Cancer Center
Sponsor class: OTHER
Collaborators: name: Amgen
First posted: 2007-07-16
Enrollment: 2
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Primary Objective:

1. To determine whether rhG-CSF treatment will increase the frequency of donor-derived cells contributing to repair of damaged epithelial/endothelial or solid organ-specific tissue caused by graft-versus-host disease (GVHD) in patients who underwent sex-mismatched stem cell transplantation.

Secondary Objective:

1. To determine whether rhG-CSF treatment can alleviate GVHD-induced damage to epithelial/endothelial or solid organ-specific tissue.

Detailed description: It has been found that cells circulating in the blood are capable of forming cells lining the oral cavity, skin cells, and/or cells of various organs. RhG-CSF is used to support cell recovery after stem cell transplantation and is commercially available.

Before treatment starts, participants will have at least one (and up to three) biopsy(ies) of damaged tissue performed to find out about the severity of tissue damage. A biopsy is taken with a large needle. Women able to have children must have a negative blood pregnancy test.

Participants in this study will receive rhG-CSF as an injection under the skin once a day over one week. This will be repeated every other week for a total of 4 weeks. Blood tests (about 2 teaspoons each) will be performed 3 times while at M. D. Anderson or once a week while outpatient to make sure that the white blood count stays in a safe range. Participants will have at least one (and up to three) biopsy(ies) again performed about 8 weeks after the start of rhG-CSF treatment. An additional biopsy at 3 months after the start rhG-CSF treatment will only be performed in case your regular treatment follow up requires it, and not for research purposes only.

Participants will be taken off study if severe side effects occur. The study will end after the last biopsy or biopsies are taken, about 3 months after the start of rhG-CSF treatment.

This is an investigational study. RhG-CSF is FDA approved and commercially available, though its use in this study is investigational. A maximum of 5 patients will be treated on this protocol. All will be enrolled at M. D. Anderson.

Eligibility criteria: Inclusion Criteria:

1. Patients who underwent allogeneic bone marrow or peripheral blood stem cell transplantation.
2. Patients should have engrafted with WBC concentration \>3000/ul. Patients should have acute GVHD overall \> grade 2 or chronic GVHD.
3. Patients with acute GVHD or chronic GVHD including patients refractory to steroid treatment.
4. Donors and patients must be of different gender.
5. Patients must sign an informed consent indicating that they are aware of the investigational nature of this study in keeping with the policies of the hospital.
6. The only acceptable consent form is the one attached at the end of this protocol.
7. Patients agree to biopsy tissue areas unaffected by GVHD for only research purposes.

Exclusion Criteria:

1. Patients who are allergic to rhG-CSF.
2. Patients who had any prior allogeneic stem cell transplantation using a sex mismatched donor other than the donor used for the previous stem cell allotransplantation.

Primary outcomes:
measure: Number of Donor Derived Cells After G-CSF Therapy
timeFrame: Baseline + 8 Weeks post transplant
Secondary outcomes:
Other outcomes:
Locations:
city: Houston
country: United States
facility: U.T.M.D. Anderson Cancer Center
state: Texas
Study officials:
affiliation: M.D. Anderson Cancer Center
name: Martin J. Korbling, MD
role: PRINCIPAL_INVESTIGATOR
References: