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Graft vs Host DiseasePhase 2NCT00048789

Extracorporeal Photopheresis to Treat Chronic Graft-Versus-Host Disease

A Phase 2 study testing Extracorporeal Photopheresis in people with Graft vs Host Disease, run by National Institute of Allergy and Infectious Diseases (NIAID) 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

This study will examine the safety and effectiveness of extracorporeal photopheresis (ECP) for treating chronic graft-versus-host disease (GvHD). GvHD is a common complication of stem cell transplantation using donated stem cells. Read more about this study

Who is it for?
Ages 4 years and older
Conditions the study is for
Graft vs Host Disease
What is being tested?
Extracorporeal Photopheresis
Type of treatment
Surgery or procedure
How many people
25 participants
Where
1 siteUnited States
Study dates
4 Nov 2002to 29 Jan 2009
Phase
Phase 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 4 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Objective evidence of chronic graft versus host disease involving the skin, lungs, gastrointestinal tract, liver, or eyes.
  • Progressive or stable GvHD despite a minimum of 30 days treatment with a calcineurin inhibitor (cyclosporine or tacrolimus) at therapeutic plasma levels, plus at least one other another agent with a different mechanism of action used concurrently for at least 30 days at doses known to be therapeutic for cGvHD. In most cases this second agent will be a corticosteroid administered either as high level pulse dosing (greater than or equal to mg/kg/day prednisone or equivalent in pulses of 3 days or longer for at least two occasions during the qualifying 30 days) and/or daily dosing (greater than or equal to 0.25mg/kg) or alternate day dosing (greater than or equal to 0.50 mg/kg) prednisone or equivalent. In other cases where use of steroids is medically undesirable or contraindicated, the second agent may be mycophenolate mofetil, sirolimus, hydroxychloroquine, clofazimine, thalidomide, azathioprine, pentostatin, etanercept, Infliximab, Rituximab or Daclizumab used at doses and schedule known to provide benefit to patients with cGvHD. Patients in whom calcineurin inhibitors are medically contraindicated may also be eligible for enrollment if there has been at least a 30 day trial of prednisone (or equivalent) at the doses/schedules noted above plus one of the second agents listed above. Patients in whom both calcineurin inhibitors and steroids are medically contraindicated or in whom these agents can be used at only limiting doses may also be eligible if at least two of the alternate agents noted above have been tried for 30 days without further improvement in symptoms of cGvHD. Patients who have stabilization of disease on calcineurin inhibitors plus steroids (or the other combinations noted above), but in whom these medications cannot be tapered without disease flare are also eligible.
  • Adequate (as determined by PI) renal, hepatic, and cardiac reserve to enable the patient to tolerate Extracorporeal Photopheresis.
  • The patient's malignancy (if applicable) is in complete remission or the patient is not a candidate for further immune-based anti-tumor therapy (such as donor lymphocyte infusions or immunomodulatory cytokines) due to severity of GvHD.
  • WBC greater than or equal to 1000 and platelets greater than or equal to 25,000.
  • Weight greater than or equal to 30kg.
Show all 15Show fewer
  • Female patients not pregnant and agree to use a reliable method of birth control during the treatment period
  • Systolic blood pressure greater than or equal to 90 mm Hg.
  • Karnofsky score or Lansky score greater than or equal to 30.
  • Predicted life expectancy of at least 3 months.
  • A minimum of 100 days following stem cell transplantation.
  • A minimum of 30 days without a response to, and 7 days following cessation of, certain investigational 2nd-line agents for treatment of GvHD (specifically: thalidomide, azathioprine, pentostatin, etanercept, Infliximab, Rituximab or Daclizumab).
  • History of failure to achieve any clinical benefit from a previous treatment with ECP where at least 3 months (12 weeks) of twice a week every other week therapy was administered.
  • Signed and witnessed informed consent.
  • Hemoglobin greater than 9.5, hematocrit greater than 28.5 (patients not meeting this inclusion criterion may be treated with iron supplementation and/or erythropoietin/darbepoetin until their hgb/hct are in the Inclusion range and may then be reconsidered for study entry).

Who cannot join

  • Hypersensitivity or allergy to psoralen (methoxypsoralen).
  • Hypersensitivity to both heparin and/or citrate products.
  • Patients who are unable to visit the NIH clinical center twice a week for at least the first four week treatment period of ECP treatments; or for the NIH or arranged home treatments with ECP thereafter; or for the every 4 week medical evaluation visits to NIH.
  • Frank gastrointestinal bleeding due to GvHD (occult blood positivity excluded).
  • Patients taking a cancer chemotherapeutic agent for continued treatment of malignancy.
  • Patients undergoing radiation therapy.
  • The patient's malignancy (if applicable) is NOT in complete remission or the patient is a candidate for further immune-based anti-tumor therapy (such as donor lymphocyte infusions or immunomodulatory cytokines) to treat GvHD.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • National Institutes of Health Clinical Center, 9000 Rockville Pike

    Bethesda, Maryland, 20892

Other trials in United States

About this study

Official title: Treatment of Chronic Graft Versus Host Disease With Extracorporeal Photopheresis

This study will examine the safety and effectiveness of extracorporeal photopheresis (ECP) for treating chronic graft-versus-host disease (GvHD). GvHD is a common complication of stem cell transplantation using donated stem cells. It occurs when the donor's T-lymphocytes (a type of immune cell) see the patient's cells as foreign and mount an immune response to reject them. The attack can cause skin rash, mouth sores, liver or lung inflammation, lack of appetite, and muscle stiffness. Chronic GvHD can cause serious illness, and even death, from the long-term effects of immune dysfunction and from toxic effects of medications (such as cyclosporine and prednisone) used to treat it.

ECP is an experimental treatment designed to stop the lymphocytes from attacking the body. It involves collecting some of the cells that cause GvHD, treating them with a combination of drug and light therapy and returning them to the body. Sixty to 80 percent of patients with chronic GvHD improve with ECP treatment, and some patients can stop treatment with prednisone or cyclosporine, or reduce the drug dosages.

Read more

Patients with chronic GvHD whose condition has not improved after a minimum 14-day course of cyclosporine and prednisone may be eligible for this study. Patients must be able to travel to the NIH Clinical Center in Bethesda, Maryland, twice a week during the 3-month study period.

Upon entering the study, participants will have a baseline evaluation to measure the extent of GvHD. This assessment includes blood tests, eye and dental examinations, skin biopsy for patients with skin involvement, and CT scans and lung function tests to look for possible lung involvement. Biopsies of the lung, liver, mouth, or eye may be requested if needed to confirm GvHD in these tissues. The skin will be photographed before starting ECP treatment and once a month during the treatment period. Following baseline tests, participants will undergo treatment and evaluations as follows:

ECP Treatment

Patients will have blood drawn to collect lymphocytes causing GvHD. This may be done with a special needle or catheter (tube inserted into a vein) or for patients who need or prefer it with a temporary central venous catheter similar to that used for the stem cell transplantation. Patients will have three 2- to 3-hour treatments a week for the first week and two treatments a week after that for a total of 25 treatments over 3 months. Patients who do not tolerate the treatment...

Read the detailed description

This is a phase 2 study of extracorporeal photopheresis (ECP) for treatment of chronic graft versus host disease following allogeneic hematopoietic stem cell transplantation. Extracorporeal exposure of human mononuclear cells to ultraviolet A radiation following photosensitization with 8-methoxypsoralen has proven to be an effective treatment for cutaneous T-cell lymphoma. Small, single institution studies have suggested efficacy in other T-cell mediated diseases such as solid organ rejection and graft versus host disease. This a phase 2 study of ECP for treatment of chronic graft versus host disease. Eligible patients must have objective evidence of chronic graft versus host disease that is refractory to conventional therapy or must have steroid-dependent disease that does not permit steroid dose reduction. The primary objective of this study is to better define the safety, efficacy and mechanism of action of ECP for treatment of patients with chronic graft versus host disease.

Being tested
Extracorporeal Photopheresis
Keywords
Psoralen, Refractory, Transplantation, Stem Cell, UVA, Graft-versus-host Disease, GVHD
Medical subject headings
Graft vs Host Disease

What participants receive

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

  • Extracorporeal PhotopheresisProcedure or surgery

What is being measured

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

  1. Determination of response rate including degree of improvement; steroid sparing effect; types of cGVHD problems which respond to this therapy; assessment of problems or side effects or adverse events associated with the procedure.

Timeline

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

  1. First posted8 Nov 2002 (estimated)
  2. Study start4 Nov 2002
  3. Study completion29 Jan 2009

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
25 participants
IDs, dates and publications
ClinicalTrials.gov ID
NCT00048789
Sponsor's protocol ID
030025
Other IDs
03-I-0025
Status last verified
29 Jan 2009
First submitted
7 Nov 2002
Last updated
2 Jul 2017
Results posted
No

Publications

  • Akpek G, Zahurak ML, Piantadosi S, Margolis J, Doherty J, Davidson R, Vogelsang GB. Development of a prognostic model for grading chronic graft-versus-host disease. Blood. 2001 Mar 1;97(5):1219-26. doi: 10.1182/blood.v97.5.1219.

    PubMed 11222363

  • Sullivan KM, Witherspoon RP, Storb R, Deeg HJ, Dahlberg S, Sanders JE, Appelbaum FR, Doney KC, Weiden P, Anasetti C, et al. Alternating-day cyclosporine and prednisone for treatment of high-risk chronic graft-v-host disease. Blood. 1988 Aug;72(2):555-61.

    PubMed 3042042

  • Yoo EK, Rook AH, Elenitsas R, Gasparro FP, Vowels BR. Apoptosis induction of ultraviolet light A and photochemotherapy in cutaneous T-cell Lymphoma: relevance to mechanism of therapeutic action. J Invest Dermatol. 1996 Aug;107(2):235-42. doi: 10.1111/1523-1747.ep12329711.

    PubMed 8757769

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Extracorporeal Photopheresis in people with Graft vs Host Disease, run by National Institute of Allergy and Infectious Diseases (NIAID) 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 15 inclusion and 7 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 2 Jul 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 phase mean?

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?

Determination of response rate including degree of improvement; steroid sparing effect; types of cGVHD problems which respond to this therapy; assessment of problems or side effects or adverse events associated with the procedure.. 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 NCT00048789, which also lists 3 linked publications.

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 2 Jul 2017.

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