Graft Versus Host DiseasePhase 2NCT00003270
Chemotherapy, Radiation Therapy, and Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer
A Phase 2 study testing anti-thymocyte globulin, filgrastim and busulfan in people with Graft Versus Host Disease and Leukemia, run by Roswell Park Cancer Institute at 1 site 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. Read more about this study
- Who is it for?
- Ages 5 to 50 years
- Conditions the study is for
- Graft Versus Host Disease, Leukemia, Lymphoma
- What is being tested?
- anti-thymocyte globulin, filgrastim and busulfan
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Sep 1997to 16 Oct 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 5 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS: Histologically proven hematologic malignancy Acute lymphocytic leukemia (ALL): In second or later remission In first remission with poor prognostic features (Philadelphia chromosome positive) Acute myeloid leukemia (AML): In second or later remission In first remission with poor prognostic features, e.g., Arising from myelodysplastic syndrome Cytogenetics with -5, -7, +8, 11q23 abnormalities Complex cytogenetics AML or ALL refractory to induction or in relapse Myelodysplastic syndrome Chronic myelogenous leukemia Severe aplastic anemia or Fanconi's anemia Relapsed Hodgkin's disease Relapsed non-Hodgkin's lymphoma Multiple myeloma No suitable family donor matched for 5 or 6 HLA antigens (A, B, DR) No suitable unrelated donor matched for 6 HLA antigens Cord blood donor available matched for 4-6 out of 6 HLA antigens
PATIENT CHARACTERISTICS: Age: 5 to 50 Performance status: Karnofsky 70-100% Life expectancy: Not specified Hematopoietic: Not specified Hepatic: Bilirubin less than 3 times normal Alkaline phosphatase less than 3 times normal SGOT less than 3 times normal Renal: Creatinine less than 2 times normal OR Creatinine clearance greater than 60 mL/min Cardiovascular: MUGA with ejection fraction at least 50% Pulmonary: DLCO and spirometry at least 60% OR Exercise VO2 max/kg at least 15 mL/min/kg Other: HIV antibody negative Hepatitis B surface antigen negative No active bacterial, viral, or fungal infection
PRIOR CONCURRENT THERAPY: Biologic therapy: Not specified Chemotherapy: Not specified Endocrine therapy: Not specified Radiotherapy: Prior radiotherapy allowed If following limits have not been exceeded, patient may receive total body irradiation: No prior radiation to one entire kidney Whole liver received no greater than 1000 cGy No prior whole abdomen radiotherapy Small bowel received no greater than 3000 cGy Heart received no greater than 1800 cGy No prior whole lung radiotherapy CNS received less than 30 cGy (whole brain or any portion of the spine) Surgery: Not specified
Trial locations and contacts
1 site in 1 country.
United States1 site
Roswell Park Cancer Institute
Buffalo, New York, 14263-0001
About this study
Official title: Cord Blood Transplantation for Hematologic Malignancies and Bone Marrow Failure Syndromes
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 allow doctors to give higher doses of chemotherapy or radiation therapy and kill more cancer cells.
PURPOSE: Phase II trial to study the effectiveness of chemotherapy, radiation therapy, and umbilical cord blood transplantation in treating patients with hematologic cancer.
Read the detailed description
OBJECTIVES: I. Determine the safety, efficacy, and toxicity of using cord blood as a source for stem cell transplantation in patients with hematologic malignancies.
OUTLINE: Patients undergo autologous bone marrow harvesting or peripheral stem cell collection prior to transplant regimen, unless the patient has acute leukemia in relapse, aplastic anemia, or myelodysplastic syndrome. Arm I: Patients eligible to undergo total body irradiation (TBI) first receive cyclophosphamide IV over 2 hours on days -5 and -4, then undergo TBI twice a day on days -3 to -1. Patients also receive antithymocyte globulin (ATG) IV over 10 hours on days -3 to -1. Cord blood is infused on day 0. Arm II: Patients not eligible to receive TBI receive oral busulfan every 6 hours on days -7 to -4 for a total of 16 doses. Cyclophosphamide, ATG, and cord blood are then administered as in arm I. All patients receive cyclosporine on days -2 to 180, methylprednisolone on days 5-180, and filgrastim (G-CSF) from day 1. Patients are followed weekly until day 180 and then monthly for 2 years.
PROJECTED ACCRUAL: A total of 20 patients (10 patients per arm) will be accrued for this study within 4 years.
- Conditions
- Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Being tested
- anti-thymocyte globulin, filgrastim, busulfan, cyclophosphamide, cyclosporine
- Keywords
- recurrent childhood acute lymphoblastic leukemia, recurrent adult Hodgkin lymphoma, Burkitt lymphoma, refractory multiple myeloma, stage I multiple myeloma, stage II multiple myeloma, stage III multiple myeloma, recurrent childhood lymphoblastic lymphoma, recurrent childhood acute myeloid leukemia, recurrent adult acute myeloid leukemia, recurrent adult acute lymphoblastic leukemia, relapsing chronic myelogenous leukemia, childhood diffuse large cell lymphoma, childhood immunoblastic large cell lymphoma, chronic phase chronic myelogenous leukemia, accelerated phase chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia, meningeal chronic myelogenous leukemia, adult acute myeloid leukemia in remission, adult acute lymphoblastic leukemia in remission
- Medical subject headings
- Graft vs Host Disease, Leukemia, Lymphoma, Multiple Myeloma, Neoplasms, Plasma Cell, Myelodysplastic Syndromes, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Hodgkin Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinBiological
IV
filgrastimBiological
IV
busulfanDrug
IV
cyclophosphamideDrug
IV
cyclosporineDrug
IV
IV
bone marrow ablation with stem cell supportProcedure or surgery
IV
umbilical cord blood transplantationProcedure or surgery
IV
radiation therapyRadiation
High energy X-rays
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Patients eligible to undergo total body irradiation (TBI) first receive cyclophosphamide IV over 2 hours on days -5 and -4, then undergo TBI twice a day on days -3 to -1. Patients also receive antithymocyte globulin (ATG) IV over 10 hours on days -3 to -1. Cord blood is infused on day 0 Receives: Biological: anti-thymocyte globulin; Biological: filgrastim; Drug: busulfan; Drug: cyclophosphamide; Drug: cyclosporine; Drug: methylprednisolone; Procedure: bone marrow ablation with stem cell support; Procedure: umbilical cord blood transplantation; Radiation: radiation therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate
Continuous complete remission (for patients in complete remission before treatment) or induced complete remission (for patients not in complete remission before treatment)
Measured: day +100 after Cord Blood Transplant
Secondary outcomes (1)
Progression-free Survival
time to disease progression or death due to any cause
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2004 (estimated)
- Study start4 Sep 1997
- Primary completion16 Oct 2017
- Study completion16 Oct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Roswell Park Cancer Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Barbara Jean Bambach, MD, Study chair, Roswell Park Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00003270
- Sponsor's protocol ID
- DS 97-26
- Other IDs
- DS 97-26
- Status last verified
- Nov 2019
- First submitted
- 1 Nov 1999
- Last updated
- 13 Dec 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 13 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cord Blood Transplantation for Hematologic Malignancies and Bone Marrow Failure Syndromes
- ClinicalTrials.gov ID
- NCT00003270
- Acronym / sponsor ID
- DS 97-26
- Conditions
- Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Keywords
- recurrent childhood acute lymphoblastic leukemia, recurrent adult Hodgkin lymphoma, Burkitt lymphoma, refractory multiple myeloma, stage I multiple myeloma, stage II multiple myeloma, stage III multiple myeloma, recurrent childhood lymphoblastic lymphoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Roswell Park Cancer InstituteUniversity, hospital or other
- Study start
- 4 Sep 1997 (actual)
- Primary completion
- 16 Oct 2017 (actual)
- Study completion
- 16 Oct 2017 (actual)
- First posted
- 27 Apr 2004 (estimated)
- Results
- Posted 13 Dec 2019
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 allow doctors to give higher doses of chemotherapy or radiation therapy and kill more cancer cells.
PURPOSE: Phase II trial to study the effectiveness of chemotherapy, radiation therapy, and umbilical cord blood transplantation in treating patients with hematologic cancer.
Detailed description
OBJECTIVES: I. Determine the safety, efficacy, and toxicity of using cord blood as a source for stem cell transplantation in patients with hematologic malignancies.
OUTLINE: Patients undergo autologous bone marrow harvesting or peripheral stem cell collection prior to transplant regimen, unless the patient has acute leukemia in relapse, aplastic anemia, or myelodysplastic syndrome. Arm I: Patients eligible to undergo total body irradiation (TBI) first receive cyclophosphamide IV over 2 hours on days -5 and -4, then undergo TBI twice a day on days -3 to -1. Patients also receive antithymocyte globulin (ATG) IV over 10 hours on days -3 to -1. Cord blood is infused on day 0. Arm II: Patients not eligible to receive TBI receive oral busulfan every 6 hours on days -7 to -4 for a total of 16 doses. Cyclophosphamide, ATG, and cord blood are then administered as in arm I. All patients receive cyclosporine on days -2 to 180, methylprednisolone on days 5-180, and filgrastim (G-CSF) from day 1. Patients are followed weekly until day 180 and then monthly for 2 years.
PROJECTED ACCRUAL: A total of 20 patients (10 patients per arm) will be accrued for this study within 4 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Biological: anti-thymocyte globulin, Biological: filgrastim, Drug: busulfan, Drug: cyclophosphamide, Drug: cyclosporine, Drug: methylprednisolone, Procedure: bone marrow ablation with stem cell support, Procedure: umbilical cord blood transplantation, Radiation: radiation therapyPatients eligible to undergo total body irradiation (TBI) first receive cyclophosphamide IV over 2 hours on days -5 and -4, then undergo TBI twice a day on days -3 to -1. Patients also receive antithymocyte globulin (ATG) IV over 10 hours on days -3 to -1. Cord blood is infused on day 0 |
Who may qualify
- Ages
- 5 to 50 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 globulinIV | Biological | Arm 1 |
| filgrastimIV | Biological | Arm 1 |
| busulfanIV | Drug | Arm 1 |
| cyclophosphamideIV | Drug | Arm 1 |
| cyclosporineIV | Drug | Arm 1 |
| methylprednisoloneIV | Drug | Arm 1 |
| bone marrow ablation with stem cell supportIV | Procedure or surgery | Arm 1 |
| umbilical cord blood transplantationIV | Procedure or surgery | Arm 1 |
| radiation therapyHigh energy X-rays | Radiation | Arm 1 |
Primary outcome
Overall Response Rate
Time frame: day +100 after Cord Blood Transplant
Continuous complete remission (for patients in complete remission before treatment) or induced complete remission (for patients not in complete remission before treatment)
1 secondary outcome
Progression-free Survival
Time frame: 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Dec 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Graft vs Host Disease
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
1 site 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, filgrastim and busulfan in people with Graft Versus Host Disease and Leukemia, run by Roswell Park Cancer Institute 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?
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 13 Dec 2019. 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?
Yes. Results are posted in the registry (first posted 13 Dec 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Overall Response Rate (time frame: day +100 after Cord Blood 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 13 Dec 2019.