Stem Cell Leukemia of Unclear LineageEarly Phase 1NCT01174940
Test Extracorporeal Photopheresis (ECP) Treatment Before/After Allogeneic Bone Marrow Transplant (BMT) or Peripheral Blood Stem Cell (PBSC) Transplant to Prevent Graft Versus Host Disease
A Early Phase 1 study testing extracorporeal photopheresis in people with Stem Cell Leukemia of Unclear Lineage and Graft Versus Host Disease, run by University of Kansas Medical Center at 1 site in United States.
Trial summary
To study the effect of ECP with Uvadex® in conjunction with a standard myeloablative conditioning regimen on the incidence of acute and chronic GvHD in patients undergoing an allogeneic related or unrelated BMT or PBSC transplant, for treatment of hematologic or lymphoproliferative malignancies. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Stem Cell Leukemia of Unclear Lineage, Graft Versus Host Disease
- What is being tested?
- extracorporeal photopheresis
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2010to Aug 2015
- Phase
- Early Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients are eligible if they have a diagnosis of one of the following hematologic or lymphoproliferative malignancies for which a treatment option would be an allogeneic BMT or PBSC transplant:
- acute myelogenous leukemia
- chronic myelogenous leukemia
- acute lymphocytic/blastic leukemia
- chronic lymphocytic leukemia
- myelodysplastic syndrome
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- non-Hodgkin's lymphoma (expected survival > 60 days)
- Hodgkin's disease (expected survival > 60 days)
- Patients who are candidates for a standard allogeneic BMT or patients who are candidates for a standard allogeneic PBSC transplant.
- Patients must have a suitable HLA- molecular matched (8/10 or more) related or unrelated donor.
- Patients must be physically and psychologically capable of undergoing a BMT or PBSC transplant and the attendant period of strict isolation.
- Patients must test negative for human immunodeficiency virus (HIV).
- Patients must present no evidence of active ongoing infection.
- Patients must have adequate renal, hepatic, pulmonary, and cardiac function to enable the patient to tolerate the extracorporeal volume shifts associated with ECP, as determined by the physician's clinical judgment.
- Platelets ≥ 20,000/cmm.
- Patients ≥ 18 years of age.
- Weight ≥ 40 kg (88 lb).
- Systolic Blood Pressure ≥ 90 mm Hg after the patient has been in a sitting position for five minutes.
- Women of childbearing potential must agree to use a reliable method of birth control for the duration of the study.
- Patients must be willing to comply with all study procedures.
- Signed and dated informed consent must be obtained prior to conducting any study procedures. The parent or legal guardian of a minor must also provide written informed consent.
Who cannot join
- Patients who have received a prior allogeneic BMT or PBSC transplant.
- Hypersensitivity or allergy to psoralen (methoxsalen).
- Contraindication to radiation, cyclophosphamide, CSA, Busulphan or MTX.
- Hypersensitivity or allergy to both heparin and citrate products. (If hypersensitive or allergic to only one of these two products, exclusion does not apply if the other product is strictly used for the patient.)
- Patients whose treatment requires donor lymphocyte infusion up to day 100 post-transplant.
- Participation in another clinical trial for prevention of GvHD within 7 days prior to patient enrollment or concurrent participation in any other clinical study.
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- Active gastrointestinal bleeding.
- Females who are pregnant or lactating.
- Previous treatment with ECP.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Kansas Medical Center
Kansas City, Kansas, 66160
About this study
Official title: A Study of Extracorporeal Photopheresis With UVADEX® in the Setting of a Standard Myeloablative Conditioning Regimen in Related or Unrelated Donor Hematopoietic Stem Cell Transplantation for the Prevention of Graft Versus Host Disease
To study the effect of ECP with Uvadex® in conjunction with a standard myeloablative conditioning regimen on the incidence of acute and chronic GvHD in patients undergoing an allogeneic related or unrelated BMT or PBSC transplant, for treatment of hematologic or lymphoproliferative malignancies.
Read the detailed description
This study is to test the concept that using ECP treatment prior to and after an allogeneic bone marrow transplant (BMT) or peripheral blood stem cell (PBSC) transplant will prevent the development of GvHD. This study is not designed to detect a specific treatment effect. However, some statements about the outcome of the study are possible. A sample size of n = 21 patients could detect a statistically significant difference between the expected rate of GvHD in an untreated population, 60%, and our hypothesized rate, 30%, for the matched-unrelated recipients. This calculation is based on a one-sample, two-sided chi-square test at the 5% level of significance with 80% power.
Patients will receive ECP from day -10 and day -8 before transplant and then from day of engraftment absolute neutrophil count (ANC>500) until day 90 after transplant. Patients who enter the study will receive a BMT or PBSC transplant from a donor who is matched unrelated (8/10 to 10/10 match). Rates of acute GvHD and chronic GvHD that occur in patients are 50-70% for the matched-unrelated donor transplant.
The choice of sample size is 21 patients. The analysis will determine if there are favorable trends for a treatment effect. Comparison on survival, and rates of acute and chronic GvHD will be made with historical controls who have undergone similar myeloablative transplant from an unrelated donor.
- Being tested
- extracorporeal photopheresis
- Keywords
- myelogenous leukemia, lymphocytic leukemia, lymphoblastic leukemia, myelodysplastic syndrome, non-Hodgkin's lymphoma, Hodgkin's disease
- Medical subject headings
- Graft vs Host Disease, Leukemia, Myeloid, Leukemia, Lymphoid, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Lymphoma, Non-Hodgkin, Hodgkin Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
extracorporeal photopheresisDrug
Also called UVAR, UVAR XTS
Patients will receive 2 ECP treatments prior to the commencement of the high dose chemotherapy and then for two consecutive days every two weeks starting from post engraftment (ANC > 500) up to day 90 (total of 10 treatments). This may be given as an outpatient procedure.
The dose of UVADEX® used to inoculate these cells will be calculated based on the treatment volume collected during the plasma/buffy coat collection process, using the following formula:
Treatment Volume in mL X 0.017 of UVADEX® (20 mcg/ml) required for administration into the recirculation bag = Amount of UVADEX® (in mLs) required for administration into the recirculation bag.
After the cells are inoculated with UVADEX®, the buffy coat/plasma suspension is irradiated with ultraviolet-A light and then re-infused back into the patient.
| Study group | Role | What they receive |
|---|---|---|
| Extracorporeal Photopheresis | Experimental | Patients will receive 2 ECP treatments on day -10 and day -8 and then for two consecutive days every two weeks starting from post engraftment (ANC > 500) up to day 90 (total of 10 treatments). This may be given as an outpatient procedure. Receives: Drug: extracorporeal photopheresis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Presence/absence of grade II-IV acute Graft versus Host Disease (aGVHD)
The primary efficacy variable is the presence/absence of grade II-IV acute GvHD within the first 100 days after transplantation
Measured: 100 days after transplant
Secondary outcomes (1)
proportion of patients who develop chronic Graft versus Host Disease (cGVHD) and experience relapse of primary disease.
These secondary efficacy variables for a patient are dichotomous:
- the development of cGvHD during 365 days after transplantation (and which body sites are involved)
- the relapse of primary disease (hematologic or lymphoproliferative malignancy)
- the grade of aGvHD
- the involved sites of cGvHD
Measured: 365 days after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2010 (estimated)
- Study startJun 2010
- Primary completionAug 2014
- Study completionAug 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stem Cell Leukemia of Unclear LineageStudies of the same condition, open and under way.
- More trials run by University of Kansas Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Mallinckrodt
- Responsible party
- Sponsor
- Investigators
- Sunil Abhyankar, MD, Principal investigator, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01174940
- Sponsor's protocol ID
- 12047
- Status last verified
- Jan 2017
- First submitted
- 27 Jul 2010
- Last updated
- 9 Jan 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Extracorporeal Photopheresis With UVADEX® in the Setting of a Standard Myeloablative Conditioning Regimen in Related or Unrelated Donor Hematopoietic Stem Cell Transplantation for the Prevention of Graft Versus Host Disease
- ClinicalTrials.gov ID
- NCT01174940
- Acronym / sponsor ID
- 12047
- Keywords
- myelogenous leukemia, lymphocytic leukemia, lymphoblastic leukemia, myelodysplastic syndrome, non-Hodgkin's lymphoma, Hodgkin's disease
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Collaborators
- Mallinckrodt
- Study start
- Jun 2010
- Primary completion
- Aug 2014 (actual)
- Study completion
- Aug 2015 (actual)
- First posted
- 4 Aug 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To study the effect of ECP with Uvadex® in conjunction with a standard myeloablative conditioning regimen on the incidence of acute and chronic GvHD in patients undergoing an allogeneic related or unrelated BMT or PBSC transplant, for treatment of hematologic or lymphoproliferative malignancies.
Detailed description
This study is to test the concept that using ECP treatment prior to and after an allogeneic bone marrow transplant (BMT) or peripheral blood stem cell (PBSC) transplant will prevent the development of GvHD. This study is not designed to detect a specific treatment effect. However, some statements about the outcome of the study are possible. A sample size of n = 21 patients could detect a statistically significant difference between the expected rate of GvHD in an untreated population, 60%, and our hypothesized rate, 30%, for the matched-unrelated recipients. This calculation is based on a one-sample, two-sided chi-square test at the 5% level of significance with 80% power.
Patients will receive ECP from day -10 and day -8 before transplant and then from day of engraftment absolute neutrophil count (ANC>500) until day 90 after transplant. Patients who enter the study will receive a BMT or PBSC transplant from a donor who is matched unrelated (8/10 to 10/10 match). Rates of acute GvHD and chronic GvHD that occur in patients are 50-70% for the matched-unrelated donor transplant.
The choice of sample size is 21 patients. The analysis will determine if there are favorable trends for a treatment effect. Comparison on survival, and rates of acute and chronic GvHD will be made with historical controls who have undergone similar myeloablative transplant from an unrelated donor.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Extracorporeal Photopheresis | Experimental | Drug: extracorporeal photopheresisPatients will receive 2 ECP treatments on day -10 and day -8 and then for two consecutive days every two weeks starting from post engraftment (ANC > 500) up to day 90 (total of 10 treatments). This may be given as an outpatient procedure. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- Patients are eligible if they have a diagnosis of one of the following hematologic or lymphoproliferative malignancies for which a treatment option would be an allogeneic BMT or PBSC transplant:
- Patients who are candidates for a standard allogeneic BMT or patients who are candidates for a standard allogeneic PBSC transplant.
- Patients must have a suitable HLA- molecular matched (8/10 or more) related or unrelated donor.
- Patients must be physically and psychologically capable of undergoing a BMT or PBSC transplant and the attendant period of strict isolation.
- Patients must test negative for human immunodeficiency virus (HIV).
Key exclusion criteria (9 listed)
- Patients who have received a prior allogeneic BMT or PBSC transplant.
- Hypersensitivity or allergy to psoralen (methoxsalen).
- Contraindication to radiation, cyclophosphamide, CSA, Busulphan or MTX.
- Hypersensitivity or allergy to both heparin and citrate products. (If hypersensitive or allergic to only one of these two products, exclusion does not apply if the other product is strictly used for the patient.)
- Patients whose treatment requires donor lymphocyte infusion up to day 100 post-transplant.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| extracorporeal photopheresisAlso: UVAR, UVAR XTSPatients will receive 2 ECP treatments prior to the commencement of the high dose chemotherapy and then for two consecutive days every two weeks starting from post engraftment (ANC > 500) up to day 90 (total of 10 treatments). This may be given as an outpatient procedure. The dose of UVADEX® used to inoculate these cells will be calculated based on the treatment volume collected during the plasma/buffy coat collection process, using the following formula: Treatment Volume in mL X 0.017 of UVADEX® (20 mcg/ml) required for administration into the recirculation bag = Amount of UVADEX® (in mLs) required for administration into the recirculation bag. After the cells are inoculated with UVADEX®, the buffy coat/plasma suspension is irradiated with ultraviolet-A light and then re-infused back into the patient. | Drug | Extracorporeal Photopheresis |
Primary outcome
Presence/absence of grade II-IV acute Graft versus Host Disease (aGVHD)
Time frame: 100 days after transplant
The primary efficacy variable is the presence/absence of grade II-IV acute GvHD within the first 100 days after transplantation
1 secondary outcome
proportion of patients who develop chronic Graft versus Host Disease (cGVHD) and experience relapse of primary disease.
Time frame: 365 days after transplantation
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jan 2017.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sunil Abhyankar, MD principal investigator
University of Kansas Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing extracorporeal photopheresis in people with Stem Cell Leukemia of Unclear Lineage and Graft Versus Host Disease, run by University of Kansas Medical Center 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 21 inclusion and 9 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 9 Jan 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
Presence/absence of grade II-IV acute Graft versus Host Disease (aGVHD) (time frame: 100 days after transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Jan 2017.