Graft Versus Host DiseasePhase 2NCT00003661
Chemotherapy, Radiation Therapy, and Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer
A Phase 2 study testing anti-thymocyte globulin, busulfan and cyclosporine in people with Graft Versus Host Disease and Leukemia, run by Roswell Park Cancer Institute at 17 sites in United States.
Trial summary
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage cancer cells. Umbilical cord blood transplantation may be able to replace cells destroyed by chemotherapy or radiation therapy. Read more about this study
- Who is it for?
- Ages Up to 54 years
- Conditions the study is for
- Graft Versus Host Disease, Leukemia, Lymphoma
- What is being tested?
- anti-thymocyte globulin, busulfan and cyclosporine
- Type of treatment
- Surgery or procedure
- How many people
- 3 participants
- Where
- 17 sitesUnited States
- Study dates
- Jun 1998to Mar 2006
- 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 Up to 54 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS: Histologically confirmed malignancy including: - Pediatric acute lymphocytic leukemia (ALL) in first complete remission with high risk features including presence of t(4;11) or t(9;22), or extreme hyperleukocytosis (initial WBC greater than 500 K/mL), or failure to achieve a complete remission after standard induction therapy - Adult ALL or acute nonlymphoblastic leukemia (ANLL) in first complete remission with t(8;14) translocation or failure to achieve complete remission after standard induction therapy - ALL or ANLL in second or subsequent remission - Chronic myelogenous leukemia in chronic or accelerated phase - Myelodysplastic syndrome with evidence of evolution to acute myeloid leukemia Refractory anemia with excess blasts Refractory anemia with excess blasts in transformation Chronic myelomonocytic leukemia - T-lymphoblastic non-Hodgkin's lymphoma in second or subsequent remission - Stage IV neuroblastoma Must also meet all the following conditions: No HLA-ABC/DR identical related bone marrow or UCB donor No 5/6 antigen matched related bone marrow or UCB donor Condition precludes waiting to search and find a donor in the National Marrow Donor Registry Must have an available serologic matched umbilical cord blood unit in the New York Blood Center's Placental Blood Project No active CNS disease Not eligible for COBLT study (Transplantation of Banked Umbilicial Cord Blood Cells for Use in Clinical Research on Transplantation of Umbilical Cord Blood Stem and Progenitor Cells)
PATIENT CHARACTERISTICS: Age: Under 55 at time of umbilical cord blood transplantation Performance status: Zubrod 0-1 Lansky 80-100% Karnofsky 80-100% Life expectancy: At least 3 months Hematopoietic: Adequate hematologic status at time of back-up bone marrow harvest: WBC at least 3,000/mm3 Absolute neutrophil count at least 1,000/mm3 Platelet count at least 100,000/mm3 Hepatic: ALT/AST no greater than 4 times normal Bilirubin no greater than 2.0 mg/dL Renal: Creatinine no greater than 2.0 mg/dL Creatinine clearance at least 50 mL/min Cardiovascular: Normal cardiac function by echocardiogram or radionuclide scan (shortening fraction or ejection fraction at least 80% of normal value for age) Pulmonary: FVC and FEV1 at least 60% of predicted for age For adults: DLCO at least 60% of predicted Other: No active infections at time of back-up bone marrow harvest or pretransplant reduction Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception HIV negative
PRIOR CONCURRENT THERAPY: Biologic therapy: Not specified Chemotherapy: See Disease Characteristics Endocrine therapy: Not specified Radiotherapy: Not specified Surgery: Not specified
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
United States17 sites
Roswell Park Cancer Institute
Buffalo, New York, 14263-0001
Lineberger Comprehensive Cancer Center, UNC
Chapel Hill, North Carolina, 27599-7295
Medical University of South Carolina
Charleston, South Carolina, 29425-0721
Rush-Presbyterian-St. Luke's Medical Center
Chicago, Illinois, 60612
University of Chicago Cancer Research Center
Chicago, Illinois, 60637
University of South Carolina School of Medicine
Columbia, South Carolina, 29203
Duke Comprehensive Cancer Center
Durham, North Carolina, 27710
University of Florida Health Science Center
Gainesville, Florida, 32610-0296
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Division of Pediatric Surgery
Jacksonville, Florida, 32207
Nemours Children's Clinic
Jacksonville, Florida, 32207
North Shore University Hospital
Manhasset, New York, 11030
Children's Hospital of New Orleans
New Orleans, Louisiana, 70118
New York Blood Center
New York, New York, 10021
St. Christopher's Hospital for Children
Philadelphia, Pennsylvania, 19134-1095
Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104
H. Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, 33612
About this study
Official title: A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Children and Adults With Hematologic Malignancies
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage cancer cells. Umbilical cord blood transplantation may be able to replace cells destroyed by chemotherapy or radiation therapy.
PURPOSE: Phase II trial to study the effectiveness of chemotherapy, radiation therapy, and umbilical cord blood transplantation in treating patients who have hematologic cancer.
Read the detailed description
OBJECTIVES: I. Determine the rates of hematologic and immune reconstitution in pediatric patients with high risk hematologic malignancies in first remission or in second or subsequent remission, and adult patients with acute lymphocytic leukemia (ALL) or acute nonlymphocytic leukemia (ANLL) in second or subsequent remission, who are undergoing high dose chemoradiotherapy followed by unrelated umbilical cord blood (UCB) transplantation. II. Determine the incidence of graft-versus-host disease in this setting. III. Determine whether contamination of umbilical cord blood with maternal cells is a clinical problem in this setting. IV. Describe the incidence of leukemic relapse in these patients after UCB transplantation. V. Describe the incidence of serious infections and secondary lymphoproliferative diseases following transplantation with UCB in these patients. VI. Determine specifically whether larger recipients (greater than 40 kg) can be durably engrafted with unrelated UCB, and determine whether nucleated cell or progenitor cell content of the graft is predictive of hematological engraftment.
OUTLINE: Patients undergo a back-up bone marrow harvest prior to treatment. Patients receive 9 fractions of total body irradiation (TBI) on days -9 to -5, followed by melphalan IV on days -4 to -2 and antithymocyte globulin IV or methylprednisolone IV on days -3 to -1. If TBI is not allowed, busulfan is substituted and administered orally every 6 hours for 4 days on days -8 to -5. On day 0, patients receive umbilical cord blood infusion. Cyclosporine and methylprednisolone begin on day -2 and continue for 6 months. Patients are followed indefinitely for survival and late toxicity.
PROJECTED ACCRUAL: A minimum of 48 patients will be accrued into this study within 4 years.
- Being tested
- anti-thymocyte globulin, busulfan, cyclosporine, melphalan, methylprednisolone
- Keywords
- recurrent childhood acute lymphoblastic leukemia, stage 4S neuroblastoma, recurrent childhood lymphoblastic lymphoma, recurrent adult acute lymphoblastic leukemia, chronic phase chronic myelogenous leukemia, accelerated phase chronic myelogenous leukemia, adult acute myeloid leukemia in remission, adult acute lymphoblastic leukemia in remission, childhood acute myeloid leukemia in remission, childhood acute lymphoblastic leukemia in remission, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, chronic myelomonocytic leukemia, recurrent grade 1 follicular lymphoma, recurrent grade 2 follicular lymphoma, recurrent grade 3 follicular lymphoma, recurrent adult diffuse small cleaved cell lymphoma, recurrent adult diffuse mixed cell lymphoma, recurrent adult diffuse large cell lymphoma, recurrent adult immunoblastic large cell lymphoma
- Medical subject headings
- Graft vs Host Disease, Leukemia, Lymphoma, Myelodysplastic Syndromes, Neuroblastoma, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Chronic-Phase, Leukemia, Myeloid, Accelerated Phase
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinBiological
busulfanDrug
cyclosporineDrug
melphalanDrug
umbilical cord blood transplantationProcedure or surgery
radiation therapyRadiation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2004 (estimated)
- Study startJun 1998
- Primary completionNov 1999
- Study completionMar 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft Versus Host DiseaseStudies of the same condition, open and under way.
- More trials run by Roswell Park Cancer InstituteThe 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Roswell Park Cancer Institute
- Sponsor type
- University, hospital or other
- Investigators
- Barbara Jean Bambach, MD, Study chair, Roswell Park Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00003661
- Sponsor's protocol ID
- CDR0000066754
- Other IDs
- RPCI-RP-9801
- Status last verified
- Mar 2011
- First submitted
- 1 Nov 1999
- Last updated
- 7 Mar 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Children and Adults With Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT00003661
- Acronym / sponsor ID
- CDR0000066754
- Keywords
- recurrent childhood acute lymphoblastic leukemia, stage 4S neuroblastoma, recurrent childhood lymphoblastic lymphoma, recurrent adult acute lymphoblastic leukemia, chronic phase chronic myelogenous leukemia, accelerated phase chronic myelogenous leukemia, adult acute myeloid leukemia in remission, adult acute lymphoblastic leukemia in remission
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Roswell Park Cancer InstituteUniversity, hospital or other
- Study start
- Jun 1998
- Primary completion
- Nov 1999 (actual)
- Study completion
- Mar 2006 (actual)
- First posted
- 4 Aug 2004 (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.
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage cancer cells. Umbilical cord blood transplantation may be able to replace cells destroyed by chemotherapy or radiation therapy.
PURPOSE: Phase II trial to study the effectiveness of chemotherapy, radiation therapy, and umbilical cord blood transplantation in treating patients who have hematologic cancer.
Detailed description
OBJECTIVES: I. Determine the rates of hematologic and immune reconstitution in pediatric patients with high risk hematologic malignancies in first remission or in second or subsequent remission, and adult patients with acute lymphocytic leukemia (ALL) or acute nonlymphocytic leukemia (ANLL) in second or subsequent remission, who are undergoing high dose chemoradiotherapy followed by unrelated umbilical cord blood (UCB) transplantation. II. Determine the incidence of graft-versus-host disease in this setting. III. Determine whether contamination of umbilical cord blood with maternal cells is a clinical problem in this setting. IV. Describe the incidence of leukemic relapse in these patients after UCB transplantation. V. Describe the incidence of serious infections and secondary lymphoproliferative diseases following transplantation with UCB in these patients. VI. Determine specifically whether larger recipients (greater than 40 kg) can be durably engrafted with unrelated UCB, and determine whether nucleated cell or progenitor cell content of the graft is predictive of hematological engraftment.
OUTLINE: Patients undergo a back-up bone marrow harvest prior to treatment. Patients receive 9 fractions of total body irradiation (TBI) on days -9 to -5, followed by melphalan IV on days -4 to -2 and antithymocyte globulin IV or methylprednisolone IV on days -3 to -1. If TBI is not allowed, busulfan is substituted and administered orally every 6 hours for 4 days on days -8 to -5. On day 0, patients receive umbilical cord blood infusion. Cyclosporine and methylprednisolone begin on day -2 and continue for 6 months. Patients are followed indefinitely for survival and late toxicity.
PROJECTED ACCRUAL: A minimum of 48 patients will be accrued into this study within 4 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 54 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
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 |
|---|---|---|
| anti-thymocyte globulin | Biological | – |
| busulfan | Drug | – |
| cyclosporine | Drug | – |
| melphalan | Drug | – |
| methylprednisolone | Drug | – |
| umbilical cord blood transplantation | Procedure or surgery | – |
| radiation therapy | Radiation | – |
Primary outcome
Not reported in the source record.
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 7 Mar 2011.
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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.
Barbara Jean Bambach, MD study chair
Roswell Park Cancer Institute
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing anti-thymocyte globulin, busulfan and cyclosporine in people with Graft Versus Host Disease and Leukemia, run by Roswell Park Cancer Institute at 17 sites 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?
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 7 Mar 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites 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.
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 7 Mar 2011.