BMT Auto MSCs GvHD Ph 1
| Status: | Recruiting |
|---|---|
| Conditions: | Orthopedic, Hematology |
| Therapuetic Areas: | Hematology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | 12 - Any |
| Updated: | 5/18/2018 |
| Start Date: | January 2015 |
| End Date: | January 2020 |
| Contact: | Ashley Griffin |
| Email: | ashley.griffin@choa.org |
| Phone: | 404-785-0653 |
A Phase I Study of Mesenchymal Stromal Cells for the Treatment of Acute and Chronic Graft Versus Host Disease
Subjects in this study have had an allogeneic (blood or marrow cells from another person)
blood or marrow transplant to treat leukemia, lymphoma or other cancer of the blood, and have
now developed Graft Versus Host Disease (GVHD) that is not responding to standard treatment.
GVHD is when the graft (transplanted bone marrow or blood) attacks the recipient's body. GVHD
occurs early after transplant (acute) and/or sometimes months after transplant (chronic).
Both forms can be life threatening; chronic GVHD can be a lifelong disabling condition.
Mesenchymal stromal cells (MSCs) exist in tissues throughout the body. One place they are
found is in the bone marrow and from here they can be obtained by needle aspiration, the same
way bone marrow samples are obtained to test for leukemia. This study uses autologous MSCs
obtained from the recipient with acute and/or chronic GVHD, which have a lower chance of
being rejected. These MSCs may promote tolerance, helping the donor immune cells accept the
recipient's body.
This trial is being conducted as a step toward testing the long-term hypothesis that freshly
cultured autologous MSC grown in platelet lysate-containing medium will modulate donor T-cell
immune responses and reduce GVHD in allo-HSCT recipients. As a phase I dose escalation trial
of autologous MSC in patients with acute and chronic GVHD, the main aim is to evaluate the
safety of this therapy and its effects on GVHD biomarkers and T-cell phenotype
blood or marrow transplant to treat leukemia, lymphoma or other cancer of the blood, and have
now developed Graft Versus Host Disease (GVHD) that is not responding to standard treatment.
GVHD is when the graft (transplanted bone marrow or blood) attacks the recipient's body. GVHD
occurs early after transplant (acute) and/or sometimes months after transplant (chronic).
Both forms can be life threatening; chronic GVHD can be a lifelong disabling condition.
Mesenchymal stromal cells (MSCs) exist in tissues throughout the body. One place they are
found is in the bone marrow and from here they can be obtained by needle aspiration, the same
way bone marrow samples are obtained to test for leukemia. This study uses autologous MSCs
obtained from the recipient with acute and/or chronic GVHD, which have a lower chance of
being rejected. These MSCs may promote tolerance, helping the donor immune cells accept the
recipient's body.
This trial is being conducted as a step toward testing the long-term hypothesis that freshly
cultured autologous MSC grown in platelet lysate-containing medium will modulate donor T-cell
immune responses and reduce GVHD in allo-HSCT recipients. As a phase I dose escalation trial
of autologous MSC in patients with acute and chronic GVHD, the main aim is to evaluate the
safety of this therapy and its effects on GVHD biomarkers and T-cell phenotype
EPIC MSC2014-002 solution- Autologous Mesenchymal Stromal Cells expanded using pooled human
platelet lysate,is made up of autologous marrow-derived mesenchymal stromal cells ex vivo
expanded numerically for approximately 14 days using pooled human Platelet Lysate (phPL),
harvested from culture on the day of infusion and suspended at a concentration of 4 million
cells/ml in Plasmalyte A with 0.5% human serum albumin. This is a phase I dose-escalation,
open label, non-randomized, non-placebo controlled, single group assignment study to evaluate
the safety and tolerability of EPIC MSC2014-002. The product will be infused intravenously
and will be administered at one of three dose levels: (Dose level 1): Single Cell infusion 2
x 10^6 cells/kg, (Dose Level 2): Two weekly Cell infusions 2 x 10^6 cells/kg , (Dose level
3): Four weekly Cell infusion 2 x 10^6 cells/kg. This Phase I clinical trial will enroll
12-24 subjects with acute or chronic GVHD. The duration of this study for each patient is 1
year. The investigators anticipate that this study will be completed within 3 years of
commencement.
Objectives:
- To determine the safety and tolerability of infusing escalating doses of autologous MSCs
for patients with acute or chronic GVHD.
- To assess the overall response rate of acute and chronic GVHD to autologous MSC
infusion. These data will be used to plan future, larger clinical trials to evaluate the
efficacy of autologous MSCs for the treatment of GVHD.
- To determine the effect of MSC infusion on lymphocyte phenotype, inflammatory biomarkers
and GVHD specific biomarker levels
platelet lysate,is made up of autologous marrow-derived mesenchymal stromal cells ex vivo
expanded numerically for approximately 14 days using pooled human Platelet Lysate (phPL),
harvested from culture on the day of infusion and suspended at a concentration of 4 million
cells/ml in Plasmalyte A with 0.5% human serum albumin. This is a phase I dose-escalation,
open label, non-randomized, non-placebo controlled, single group assignment study to evaluate
the safety and tolerability of EPIC MSC2014-002. The product will be infused intravenously
and will be administered at one of three dose levels: (Dose level 1): Single Cell infusion 2
x 10^6 cells/kg, (Dose Level 2): Two weekly Cell infusions 2 x 10^6 cells/kg , (Dose level
3): Four weekly Cell infusion 2 x 10^6 cells/kg. This Phase I clinical trial will enroll
12-24 subjects with acute or chronic GVHD. The duration of this study for each patient is 1
year. The investigators anticipate that this study will be completed within 3 years of
commencement.
Objectives:
- To determine the safety and tolerability of infusing escalating doses of autologous MSCs
for patients with acute or chronic GVHD.
- To assess the overall response rate of acute and chronic GVHD to autologous MSC
infusion. These data will be used to plan future, larger clinical trials to evaluate the
efficacy of autologous MSCs for the treatment of GVHD.
- To determine the effect of MSC infusion on lymphocyte phenotype, inflammatory biomarkers
and GVHD specific biomarker levels
Inclusion Criteria:
- Age: patients must be ≥12 years old and weigh > (25 kg) at the time of study entry.
- Patients must have received an allogeneic stem cell transplant for a hematologic
malignancy.
- Must have one of the following diagnoses:
- Acute GVHD (grade II-IV) requiring systemic therapy and refractory/unresponsive to
glucocorticoid (>1 mg prednisone-equivalent/kg x 1 week)
- Chronic GVHD that is extensive and not improved despite therapy with glucocorticoid (>
0.5 mg prednisone-equivalent/kg/day) and therapeutic doses of a calcineurin inhibitor
for at least 4 weeks, or worsened within 2 weeks, or overlap syndrome not responding
to glucocorticoid treatment (>1 mg prednisone-equivalent/kg x 1 week)
Exclusion Criteria:
- Active invasive fungal infection requiring treatment with anti-fungal medication.
- Active viral infection requiring treatment with anti-viral medication.
- Persistence/relapse at the time of study entry of the primary malignancy for which the
transplant was performed. Patients with a history of relapsed malignancy who have
achieved a remission at the time of evaluation for study participation will not be
excluded.
- Known T-cell donor chimerism of <50%.
- Documented DLCO <50% (if performed within 90 days of enrollment) or requirement for
supplemental oxygen.
- Pregnancy or breastfeeding. Patients of childbearing capability should agree to use
contraception.
We found this trial at
1
site
Atlanta, Georgia 30322
Principal Investigator: Muna Qayed, M.D.
Phone: 404-785-0653
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