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LeukemiaPhase 1/2NCT00179790

Reduced Intensity Transplant Using Extracorporeal Photopheresis

A Phase 1/2 study testing Extracorporeal Photopheresis in people with Leukemia and Cancer, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Any age
Conditions the study is for
Leukemia, Cancer
What is being tested?
Extracorporeal Photopheresis
How many people
22 participants
Where
1 siteUnited States
Study dates
Jul 2005to Jan 2015
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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
Show all 25Show fewer
  • 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:
  • Don't achieve remission (molecular or cytogenetic) by 1 year of diagnosis with therapy (imatinib mesylate or interferon)
  • Have a rising quantitative bcr/abl on imatinib mesylate (molecular relapse)
  • Had developed accelerated phase regardless of therapy but are now back in second chronic phase
  • Patients with recurrent solid tumors (neuroblastoma, Ewing's sarcoma, melanoma, rhabdomyosarcoma)
  • Patients with myelodysplastic syndrome
  • Patients with refractory anemia (RA) and refractory anemia with excess blasts (RAEB) are eligible, but refractory anemia with excess blasts in transformation (RAEB-T) patients are only eligible if treated to < 20% blasts with chemotherapy
  • Patients with selected immunodeficiencies such as Wiskott-Aldrich syndrome or hyper-IgM syndrome
  • Patients with metabolic diseases such as Niemann-Pick or adrenoleukodystrophy
  • Patients with bone marrow failure syndromes, including aplastic anemia
  • Adequate venous access

Who cannot join

None listed.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Ann & Robert H Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60625

Other trials in United States

About this study

Official title: Reduced Intensity Stem Cell Transplant in Children and Young Adults Utilizing Photopheresis, Fludarabine, and Busulfan

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.

Read more

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.

Read the detailed description

This study tests the feasibility of a reduced intensity preparative regimen for stem cell transplant including extracorporeal photopheresis (ECP), busulfan, and fludarabine in patients with leukemia, lymphoma, and certain non-malignant diseases. The current reduced intensity protocol includes busulfan, fludarabine, and anti-thymocyte immunoglobulin. ECP is currently used in diseases such as chronic GVHD and cutaneous T cell lymphoma. The mechanism of ECP has not been defined. It is hypothesized that exposure of white blood cells to ultraviolet light with 8-methoxypsoralen initiates an apoptotic cellular cascade. Apoptotic cells are recognized and removed by the reticuloendothelial system, initiating the secretion of anti-inflammatory cytokines and the reduction of proinflammatory cytokines. Antigen presenting cells then regulate immune responses through the induction of tolerance.

Here we incorporate the use of ECP, fludarabine, and busulfan in the preparative regimen, followed by ECP as prophylaxis for acute graft versus host disease. We hypothesize that photopheresis is safe and feasible, and patients will have similar rates of engraftment with less GVHD as those treated with current reduced intensity protocols. The use of ECP prior to transplant provides immunosuppression promoting host engraftment. Furthermore, the introduction of ECP following transplant may be able to induce tolerance thereby reducing rates of GVHD.

Conditions
Leukemia, Cancer
Being tested
Extracorporeal Photopheresis
Keywords
cancer, hemoglobinopathy, immune deficiency, photopheresis, ECP, extracorporeal photopheresis
Medical subject headings
Leukemia, Neoplasms, Hemoglobinopathies, Immunologic Deficiency Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Extracorporeal PhotopheresisProcedure or surgery

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

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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)

    Measured: Throughout Treatment

  2. 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).

    Measured: By Day +100

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

    Measured: Throughout Treatment

  4. To evaluate 100 day transplant related mortality

    Measured: Through Day +100

Secondary outcomes (1)

  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

    Measured: Pre Transplant through 1 year post stem cell transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Sep 2005 (estimated)
  2. Study startJul 2005
  3. Primary completionJun 2010
  4. Study completionJan 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
22 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00179790
Sponsor's protocol ID
ECP RIT
Status last verified
Mar 2017
First submitted
9 Sep 2005
Last updated
6 Mar 2017
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Extracorporeal Photopheresis in people with Leukemia and Cancer, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 25 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 6 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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) (time frame: Throughout Treatment). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00179790.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Mar 2017.

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