Reduced Intensity Transplant Using Extracorporeal Photopheresis

NCT00179790 | Completed Phase 1

• 1 other identifier: ECP RIT
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

Brief Summary

Stem cell transplantation may be used to cure childhood cancers, and other diseases. Traditionally, stem cell transplants use high doses of chemotherapy and radiation. This regimen may cause significant problems after transplant such as infertility, infection, and graft versus host disease (GVHD). Reduced intensity transplant (RIT) uses medications which weaken the immune system, allowing donor cells to take over. The goal of a RIT is to reduce the risk for complications after transplant. Usually medication is used to weaken the immune system, but there are other options such as extracorporeal photopheresis (ECP) that may be less toxic. ECP is currently used for the treatment of GVHD and certain lymphomas. ECP uses a machine that filters white blood cells from the blood, treats them with ultraviolet (UV) light, and then gives all the cells back to the patient. The patient's immune system becomes weaker, allowing the donor cells to replace those of the patient. Studies involving the use of ECP for conditioning have shown fewer side effects than the use of medications. The primary purpose of this clinical research trial is to evaluate the safety and feasibility of ECP as part of a preparative regimen for RIT in children and young adults.

Conditions

Leukemia; Cancer

Keywords

cancer; hemoglobinopathy; immune deficiency; photopheresis; ECP; extracorporeal photopheresis

Outcome Measures

Primary Outcomes (4)

• To determine the feasibility of using photopheresis as the backbone of a reduced intensity transplant regimen to reduce transplant related mortality and acute and chronic graft versus host disease (GVHD)

  Throughout Treatment

• To determine time to engraftment and the percentage of patients achieving full engraftment by day +100. Engraftment will be defined as > 95% total donor chimerism as determined by restriction fragment length polymorphism (RFLP).

  By Day +100

• To determine the rate of grades III and IV acute GVHD.

  Throughout Treatment

• To evaluate 100 day transplant related mortality

  Through Day +100

Secondary Outcomes (1)

• To establish patterns of biological effects of photopheresis on dendritic cell and CD4/CD8 populations, CD4/CD25 populations, IFN-gamma, TNF-alpha, IL-10, IL-12, and IL-4

  Pre Transplant through 1 year post stem cell transplant

Interventions

Extracorporeal Photopheresis PROCEDURE

Patient will undergo ECP treatment pre and post stem cell transplant infusion

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Weight \> 25 kg
• Patients with acute lymphoblastic leukemia (ALL) who are in CR (complete remission; \< 5% blasts in bone marrow and no active central nervous system disease) who:
• Are in second remission with an initial remission of \< 36 months.
• Patients with "high risk" disease in CR1, defined by karyotype abnormalities such as presence of (9;22) translocation, monosomy 7, or monosomy 5; and/or patients with slow initial response (initial remission not reached within four weeks from diagnosis).
• Are in third (or subsequent) remission
• Experience isolated extramedullary relapse while on therapy
• Have experienced relapse following myeloablative stem cell transplant
• Are WT1+ following induction therapy
• Patients with acute myelogenous leukemia (AML) who:
• Are in first remission and remain WT1 positive.
• Are in second remission
• Are in initial partial remission (\< 20% blasts in bone marrow)
• Experience relapse following myeloablative stem cell transplant
• Patients with relapsed lymphoma whose residual disease appears to be chemo-responsive and non-bulky (\< 5 cm largest diameter)
• Patients with chronic myelogenous leukemia (CML) in chronic phase who:

Sponsors & Collaborators

• Ann & Robert H Lurie Children's Hospital of Chicago: lead
• Mallinckrodt: collaborator

Study Sites (1)

Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, 60625, United States

Location

MeSH Terms

Conditions

Leukemia; Neoplasms; Hemoglobinopathies; Immunologic Deficiency Syndromes

Interventions

Photopheresis

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immune System Diseases

Intervention Hierarchy (Ancestors)

PUVA Therapy; Ultraviolet Therapy; Phototherapy; Therapeutics; Extracorporeal Circulation; Surgical Procedures, Operative

Study Officials

• Jennifer Schneiderman, MD, MS

  Ann & Robert H Lurie Children's Hospital of Chicago

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Attending Physician, Hematology, Oncology,Cell Transplantation

Study Record Dates

• First Submitted: September 9, 2005
• First Posted: September 16, 2005
• Study Start: July 1, 2005
• Primary Completion: June 1, 2010
• Study Completion: January 1, 2015
• Last Updated: March 6, 2017

Record last verified: 2017-03

Locations

US (1)