Childhood Langerhans Cell HistiocytosisPhase 2NCT00084695
Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases
A Phase 2 study testing anti-thymocyte globulin, busulfan and cyclophosphamide in people with Childhood Langerhans Cell Histiocytosis and Fanconi Anemia, run by Milton S. Hershey Medical Center at 1 site in United States.
Trial summary
RATIONALE: Umbilical cord blood transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy. PURPOSE: This phase II trial is studying how well umbilical cord blood works as a source of stem cells in treating patients with types of cancer as well as other diseases. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia
- What is being tested?
- anti-thymocyte globulin, busulfan and cyclophosphamide
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2003to Dec 2012
- 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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS:
- Diagnosis of malignant or non-malignant disease, including but not limited to any of the following:
- Acute myeloid leukemia or acute lymphoblastic leukemia (ALL) with resistant disease beyond first clinical remission (CR)
- ALL in first CR at high-risk because of 1 of the following factors:
- Hypoploidy
- Pseudodiploidy with translocations t(9;22), t(4;11), or t(8;14)
- Elevated WBC at diagnosis as follows:
- > 100,000/mm^3 for patients 6-12 months of age
- > 50,000/mm^3 for patients 10-20 years of age
- > 20,000/mm^3 for patients 21 years of age
- Burkitt's lymphoma/leukemia
- Chronic myelogenous leukemia in first chronic phase or beyond
- Juvenile myelomonocytic leukemia
- Advanced stage or relapsed lymphoma
- Advanced stage or relapsed solid tumors, including any of the following:
- Neuroblastoma
- Ewing's sarcoma
- Rhabdomyosarcoma
- Myelodysplastic syndromes, excluding patients with grade 3 or 4 myelofibrosis
- Familial erythrophagocytic histiocytosis
- Histiocytosis unresponsive to medical management
- Inborn errors of metabolism
- Langerhans cell histiocytosis unresponsive to medical management
- Immune deficiencies, including:
- Severe combined immune deficiency
- Wiskott-Aldrich
- Hemoglobinopathies, including sickle cell disease and thalassemia
- Severe aplastic anemia
- Fanconi's anemia
- Metabolic storage diseases
- Unrelated cord blood donor must be HLA-identical OR may be mismatched for 1, 2, or 3 HLA-loci (A, B, DR)
- No other existing HLA-identical related donor available at the time of transplantation
PATIENT CHARACTERISTICS:
Age
- 21 and under
Performance status
- Not specified
Life expectancy
- Not specified
Hematopoietic
- See Disease Characteristics
Hepatic
- Not specified
Renal
- Not specified
PRIOR CONCURRENT THERAPY:
Biologic therapy
- Not specified
Chemotherapy
- Not specified
Endocrine therapy
- Not specified
Radiotherapy
- Not specified
Surgery
- Not specified
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033-0850
Kenneth G. Lucas, MD, 717-531-6012, klucas@psu.edu
About this study
Official title: The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells
RATIONALE: Umbilical cord blood transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy.
PURPOSE: This phase II trial is studying how well umbilical cord blood works as a source of stem cells in treating patients with types of cancer as well as other diseases.
Read the detailed description
OBJECTIVES:
Primary
- Determine the impact of the use of umbilical cord blood as a source of hematopoietic stem cells for children with life-threatening oncologic, hematologic, or genetic/metabolic disorders in need of a stem cell transplant.
- Compare the incidence of graft-versus-host disease in patients receiving cord blood transplants in this study with historical data for unrelated donor stem cell transplants.
- Compare the incidence of engraftment in patients receiving cord blood transplants in this study with historical data for unrelated donor stem cell transplants.
OUTLINE:
- Preparative therapy: Patients are treated on 1 of 4 preparative therapy regimens.
- Regimen A: Patients undergo total body irradiation (TBI) two times daily on days -7 to -4. Patients receive cyclophosphamide IV over 30-60 minutes on days -3 and -2 and anti-thymocyte globulin (ATG) IV over at least 6 hours on days -3 to -1.
- Regimen B (patients who do not receive TBI): Patients receive oral busulfan 4 times daily on days -8 to -5, and ATG IV over at least 6 hours and melphalan IV over 15-20 minutes on days -4 to -2.
- Regimen C (patients with Fanconi's anemia and related disorders): Patients undergo TBI on day -6. Patients receive ATG IV over at least 6 hours and methylprednisolone IV on days -5 to -1 and fludarabine IV over 30 minutes and cyclophosphamide IV over 30-60 minutes on days -5 to -2.
- Regimen D: Patients receive oral or IV busulfan 4 times daily on days -9 to -5, ATG IV over at least 6 hours on days -5 to -3, and cyclophosphamide IV over 30-60 minutes on days -5 to -2.
- Cord blood transplant: All patients undergo umbilical cord blood transplantation on day 0.
- Graft-versus-host disease prophylaxis: Patients receive oral or IV cyclosporine twice daily beginning on day -1. Patients also receive methylprednisolone IV twice daily beginning on day 5 and continuing until at least day 28.
PROJECTED ACCRUAL: A total of 25 patients will be accrued for this study.
- Conditions
- Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia, Lymphoma, Myelodysplastic Syndromes, Neuroblastoma
- Being tested
- anti-thymocyte globulin, busulfan, cyclophosphamide, fludarabine phosphate, melphalan
- Keywords
- childhood myelodysplastic syndromes, recurrent childhood rhabdomyosarcoma, unspecified childhood solid tumor, protocol specific, previously treated childhood rhabdomyosarcoma, previously untreated childhood rhabdomyosarcoma, disseminated neuroblastoma, regional neuroblastoma, recurrent neuroblastoma, metastatic Ewing sarcoma/peripheral primitive neuroectodermal tumor, recurrent Ewing sarcoma/peripheral primitive neuroectodermal tumor, recurrent childhood acute lymphoblastic leukemia, juvenile myelomonocytic leukemia, childhood acute lymphoblastic leukemia in remission, childhood Burkitt lymphoma, recurrent childhood lymphoblastic lymphoma, stage III childhood lymphoblastic lymphoma, stage IV childhood lymphoblastic lymphoma, recurrent childhood small noncleaved cell lymphoma, stage III childhood small noncleaved cell lymphoma, stage IV childhood small noncleaved cell lymphoma
- Medical subject headings
- Fanconi Anemia, Leukemia, Lymphoma, Myelodysplastic Syndromes, Neuroblastoma, Sarcoma, Neuroectodermal Tumors, Primitive, Peripheral, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinBiological
Given IV
busulfanDrug
Given orally
cyclophosphamideDrug
Given IV
Given IV
melphalanDrug
Given IV
Given IV
radiation therapyRadiation
Patients undergo radiation therapy two times daily on days -7 to -4.
| Study group | Role | What they receive |
|---|---|---|
| Regimen A | Experimental | Patients undergo total body irradiation (TBI) two times daily on days -7 to -4. Patients receive cyclophosphamide IV over 30-60 minutes on days -3 and -2 and anti-thymocyte globulin (ATG) IV over at least 6 hours on days -3 to -1. Receives: Biological: anti-thymocyte globulin; Drug: cyclophosphamide; Radiation: radiation therapy |
| Regimen B (patients who do not receive TBI) | Experimental | Patients receive oral busulfan 4 times daily on days -8 to -5, and ATG IV over at least 6 hours and melphalan IV over 15-20 minutes on days -4 to -2. Receives: Biological: anti-thymocyte globulin; Drug: busulfan; Drug: melphalan |
| Regimen C (patients with Fanconi's anemia/related disorders) | Experimental | Patients undergo TBI on day -6. Patients receive ATG IV over at least 6 hours and methylprednisolone IV on days -5 to -1 and fludarabine IV over 30 minutes and cyclophosphamide IV over 30-60 minutes on days -5 to -2. Receives: Biological: anti-thymocyte globulin; Drug: cyclophosphamide; Drug: fludarabine phosphate; Drug: methylprednisolone; Radiation: radiation therapy |
| Regimen D | Experimental | Patients receive oral or IV busulfan 4 times daily on days -9 to -5, ATG IV over at least 6 hours on days -5 to -3, and cyclophosphamide IV over 30-60 minutes on days -5 to -2. Receives: Biological: anti-thymocyte globulin; Drug: busulfan; Drug: cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Impact of the use of umbilical cord blood as a source of hematopoietic stem cells
Comparison of the incidence of graft-vs-host disease with historical data
Comparison of the incidence of engraftment with historical data
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2004 (estimated)
- Study startSep 2003
- Primary completionDec 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Childhood Langerhans Cell HistiocytosisStudies of the same condition, open and under way.
- More trials run by Milton S. Hershey 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
- Phase 2
- Primary purpose
- Treatment
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Milton S. Hershey Medical Center
- Sponsor type
- University, hospital or other
- Investigators
- Kenneth G. Lucas, MD, Study chair, Milton S. Hershey Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00084695
- Sponsor's protocol ID
- CDR0000365544
- Other IDs
- PSCI-2003-232
- Status last verified
- Oct 2008
- First submitted
- 10 Jun 2004
- Last updated
- 10 Jan 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells
- ClinicalTrials.gov ID
- NCT00084695
- Acronym / sponsor ID
- CDR0000365544
- Conditions
- Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia, Lymphoma, Myelodysplastic Syndromes, Neuroblastoma
- Keywords
- childhood myelodysplastic syndromes, recurrent childhood rhabdomyosarcoma, unspecified childhood solid tumor, protocol specific, previously treated childhood rhabdomyosarcoma, previously untreated childhood rhabdomyosarcoma, disseminated neuroblastoma, regional neuroblastoma, recurrent neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Milton S. Hershey Medical CenterUniversity, hospital or other
- Study start
- Sep 2003
- Primary completion
- Dec 2012 (estimated)
- First posted
- 11 Jun 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: Umbilical cord blood transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy.
PURPOSE: This phase II trial is studying how well umbilical cord blood works as a source of stem cells in treating patients with types of cancer as well as other diseases.
Detailed description
OBJECTIVES:
Primary
- Determine the impact of the use of umbilical cord blood as a source of hematopoietic stem cells for children with life-threatening oncologic, hematologic, or genetic/metabolic disorders in need of a stem cell transplant.
- Compare the incidence of graft-versus-host disease in patients receiving cord blood transplants in this study with historical data for unrelated donor stem cell transplants.
- Compare the incidence of engraftment in patients receiving cord blood transplants in this study with historical data for unrelated donor stem cell transplants.
OUTLINE:
- Preparative therapy: Patients are treated on 1 of 4 preparative therapy regimens.
- Regimen A: Patients undergo total body irradiation (TBI) two times daily on days -7 to -4. Patients receive cyclophosphamide IV over 30-60 minutes on days -3 and -2 and anti-thymocyte globulin (ATG) IV over at least 6 hours on days -3 to -1.
- Regimen B (patients who do not receive TBI): Patients receive oral busulfan 4 times daily on days -8 to -5, and ATG IV over at least 6 hours and melphalan IV over 15-20 minutes on days -4 to -2.
- Regimen C (patients with Fanconi's anemia and related disorders): Patients undergo TBI on day -6. Patients receive ATG IV over at least 6 hours and methylprednisolone IV on days -5 to -1 and fludarabine IV over 30 minutes and cyclophosphamide IV over 30-60 minutes on days -5 to -2.
- Regimen D: Patients receive oral or IV busulfan 4 times daily on days -9 to -5, ATG IV over at least 6 hours on days -5 to -3, and cyclophosphamide IV over 30-60 minutes on days -5 to -2.
- Cord blood transplant: All patients undergo umbilical cord blood transplantation on day 0.
- Graft-versus-host disease prophylaxis: Patients receive oral or IV cyclosporine twice daily beginning on day -1. Patients also receive methylprednisolone IV twice daily beginning on day 5 and continuing until at least day 28.
PROJECTED ACCRUAL: A total of 25 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Regimen A | Experimental | Biological: anti-thymocyte globulin, Drug: cyclophosphamide, Radiation: radiation therapyPatients undergo total body irradiation (TBI) two times daily on days -7 to -4. Patients receive cyclophosphamide IV over 30-60 minutes on days -3 and -2 and anti-thymocyte globulin (ATG) IV over at least 6 hours on days -3 to -1. |
| Regimen B (patients who do not receive TBI) | Experimental | Biological: anti-thymocyte globulin, Drug: busulfan, Drug: melphalanPatients receive oral busulfan 4 times daily on days -8 to -5, and ATG IV over at least 6 hours and melphalan IV over 15-20 minutes on days -4 to -2. |
| Regimen C (patients with Fanconi's anemia/related disorders) | Experimental | Biological: anti-thymocyte globulin, Drug: cyclophosphamide, Drug: fludarabine phosphate, Drug: methylprednisolone, Radiation: radiation therapyPatients undergo TBI on day -6. Patients receive ATG IV over at least 6 hours and methylprednisolone IV on days -5 to -1 and fludarabine IV over 30 minutes and cyclophosphamide IV over 30-60 minutes on days -5 to -2. |
| Regimen D | Experimental | Biological: anti-thymocyte globulin, Drug: busulfan, Drug: cyclophosphamidePatients receive oral or IV busulfan 4 times daily on days -9 to -5, ATG IV over at least 6 hours on days -5 to -3, and cyclophosphamide IV over 30-60 minutes on days -5 to -2. |
Who may qualify
- Ages
- Up to 21 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 globulinGiven IV | Biological | Regimen A, Regimen B (patients who do not receive TBI), Regimen C (patients with Fanconi's anemia/related disorders), Regimen D |
| busulfanGiven orally | Drug | Regimen B (patients who do not receive TBI), Regimen D |
| cyclophosphamideGiven IV | Drug | Regimen A, Regimen C (patients with Fanconi's anemia/related disorders), Regimen D |
| fludarabine phosphateGiven IV | Drug | Regimen C (patients with Fanconi's anemia/related disorders) |
| melphalanGiven IV | Drug | Regimen B (patients who do not receive TBI) |
| methylprednisoloneGiven IV | Drug | Regimen C (patients with Fanconi's anemia/related disorders) |
| radiation therapyPatients undergo radiation therapy two times daily on days -7 to -4. | Radiation | Regimen A, Regimen C (patients with Fanconi's anemia/related disorders) |
Primary outcome
Impact of the use of umbilical cord blood as a source of hematopoietic stem cells
Comparison of the incidence of graft-vs-host disease with historical data
Comparison of the incidence of engraftment with historical data
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2014.
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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.
Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center
Hershey, United States
Kenneth G. Lucas, MD study chair
Milton S. Hershey Medical Center
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, busulfan and cyclophosphamide in people with Childhood Langerhans Cell Histiocytosis and Fanconi Anemia, run by Milton S. Hershey 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?
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: status not confirmed, as of the sponsor's last update on 10 Jan 2014. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Impact of the use of umbilical cord blood as a source of hematopoietic stem cells. The study lists 3 primary outcomes in all. 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 10 Jan 2014.