- ICH GCP
- US-Register für klinische Studien
- Klinische Studie NCT00055653
Donor Umbilical Cord Blood Transplantation in Treating Patients With Leukemia, Lymphoma, or Nonmalignant Hematologic Disorders
Unrelated Umbilical Cord Blood As An Alternate Source Of Stem Cells Transplantation
RATIONALE: Umbilical cord blood transplantation may be able to replace immune cells that were destroyed by the chemotherapy or radiation therapy that was used to kill cancer cells.
PURPOSE: Phase II trial to study the effectiveness of allogeneic umbilical cord blood transplantation in treating patients who have leukemia, lymphoma, or nonmalignant hematologic disorders.
Studienübersicht
Status
Bedingungen
Intervention / Behandlung
Detaillierte Beschreibung
OBJECTIVES:
- Determine 180-day survival in patients with malignant or nonmalignant hematologic diseases treated with allogeneic umbilical cord blood transplantation. (Severe aplastic anemia, Fanconi anemia, and marrow failure syndromes strata are closed to accrual; adult [over 18 years of age] patient stratum is closed to accrual.)
- Determine disease-free and long-term survival in patients treated with this regimen.
- Determine the incidence of neutrophil engraftment, primary and secondary graft failure, platelet engraftment, and red blood cell engraftment in patients treated with this regimen.
- Determine the incidence and severity of acute and chronic graft-versus-host disease in patients treated with this regimen.
- Determine the incidence of complications, including infection, veno-occlusive disease, and interstitial pneumonitis, in patients treated with this regimen.
- Determine the incidence of relapse, other malignancies, lymphoproliferative disorders, and posttransplantation myelodysplasia in patients treated with this regimen.
- Determine the immune reconstitution in patients treated with this regimen.
OUTLINE: This is a multicenter study. Patients are grouped according to the following strata:
- Stratum I: Malignant disease, 5/6 or 6/6 HLA match, age 18 and under
- Stratum II: Malignant disease, 4/6 HLA match, age 18 and under
- Stratum III: Malignant disease, 3/6 HLA match, age 18 and under
- Stratum IV: Malignant disease, 2/6 or 1/6 HLA match, age 18 and under
- Stratum V (closed to accrual): Severe aplastic anemia, Fanconi anemia, or other marrow failure syndrome
- Stratum VI: Inborn errors of metabolism/storage diseases and other nonmalignant diseases not included in stratum V
- Stratum VII: Malignant disease receiving alternative conditioning regimen comprising busulfan and melphalan
- Stratum VIII (closed to accrual): Adult patients (over age 18)
Conditioning therapy: Patients are assigned to 1 of 5 groups according to diagnosis.
- Group I (malignant disease or severe aplastic anemia [severe aplastic anemia closed to accrual]): Patients undergo total body irradiation (TBI) once or twice daily on days -8 to -4. Patients then receive cyclophosphamide IV on days -3 and -2, methylprednisolone IV on days -3 to 0, and antithymocyte globulin (ATG) IV once or twice daily on days -3 to -1.
- Group II (Fanconi anemia [closed to accrual]): Patients undergo TBI on day -6, and then receive cyclophosphamide IV and fludarabine IV on days -5 to -2, and methylprednisolone IV and ATG IV on days -5 to -1.
- Group III (inborn errors of metabolism/storage disease): Patients receive oral busulfan 4 times daily on days -9 to -6, cyclophosphamide as in group II, and methylprednisolone and ATG as in group I.
- Group IV (other nonmalignant diseases): Patients receive conditioning therapy as in group III. Patients with familial erythrophagocytic lymphohistiocytosis or Langerhans cell histiocytosis also receive etoposide on days -5 to -3.
- Group V (non-TBI regimen for leukemia patients under 2 years of age): Patients receive oral busulfan 4 times daily on days -8 to -5, melphalan IV on days -4 to -2, and methylprednisolone and ATG as in group I.
- Allogeneic umbilical cord blood transplantation: All patients undergo umbilical cord blood transplantation on day 0. Beginning on day 0 or 1, patients receive filgrastim (G-CSF) IV or subcutaneously daily until blood counts recover.
- Graft-versus-host disease prophylaxis: Patients receive cyclosporine (IV or oral) beginning between days -3 and -1 and continuing for 1 year after transplantation and methylprednisolone twice daily beginning on day 1 and continuing until blood counts recover.
Patients are followed weekly for 14 weeks, at 100 days, and at 4, 5, 6, 9, 12, 18, 24, and 36 months.
PROJECTED ACCRUAL: A total of 360 patients will be accrued for this study.
Studientyp
Phase
- Phase 2
Kontakte und Standorte
Studienorte
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California
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Duarte, California, Vereinigte Staaten, 91010-3000
- City of Hope Comprehensive Cancer Center
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Los Angeles, California, Vereinigte Staaten, 90095-1781
- Jonsson Comprehensive Cancer Center, UCLA
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Los Angeles, California, Vereinigte Staaten, 90027-0700
- Children's Hospital Los Angeles
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Orange, California, Vereinigte Staaten, 92868
- Children's Hospital of Orange County
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San Francisco, California, Vereinigte Staaten, 94143-1278
- Children's Medical Center, University of California San Francisco
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District of Columbia
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Washington, District of Columbia, Vereinigte Staaten, 20010-2970
- Children's National Medical Center
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Indiana
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Indianapolis, Indiana, Vereinigte Staaten, 46202-5289
- Indiana University Cancer Center
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Louisiana
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New Orleans, Louisiana, Vereinigte Staaten, 70118
- Children's Hospital of New Orleans
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Massachusetts
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Boston, Massachusetts, Vereinigte Staaten, 02115
- Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute
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Michigan
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Grand Rapids, Michigan, Vereinigte Staaten, 49503
- Spectrum Health and DeVos Children's Hospital
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Minnesota
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Minneapolis, Minnesota, Vereinigte Staaten, 55455
- University of Minnesota Cancer Center
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Missouri
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Kansas City, Missouri, Vereinigte Staaten, 64108
- Children's Mercy Hospital
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Saint Louis, Missouri, Vereinigte Staaten, 63104-1095
- Cardinal Glennon Children's Hospital
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New Jersey
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Hackensack, New Jersey, Vereinigte Staaten, 07601
- Hackensack University Medical Center
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New York
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Buffalo, New York, Vereinigte Staaten, 14263-0001
- Roswell Park Cancer Institute
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Manhasset, New York, Vereinigte Staaten, 11030
- North Shore University Hospital
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Rochester, New York, Vereinigte Staaten, 14642
- James P. Wilmot Cancer Center at University of Rochester Medical Center
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North Carolina
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Durham, North Carolina, Vereinigte Staaten, 27710
- Duke Comprehensive Cancer Center
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Ohio
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Cleveland, Ohio, Vereinigte Staaten, 44106-5065
- Ireland Cancer Center
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Pennsylvania
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Pittsburgh, Pennsylvania, Vereinigte Staaten, 15213
- Children's Hospital of Pittsburgh
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Tennessee
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Nashville, Tennessee, Vereinigte Staaten, 37232-6310
- Vanderbilt-Ingram Cancer Center
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Texas
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Dallas, Texas, Vereinigte Staaten, 75230
- Medical City Dallas Hospital
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Dallas, Texas, Vereinigte Staaten, 75235
- Children's Medical Center of Dallas
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San Antonio, Texas, Vereinigte Staaten, 78229
- Texas Transplant Institute
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Washington
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Seattle, Washington, Vereinigte Staaten, 98109-1024
- Fred Hutchinson Cancer Research Center
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Teilnahmekriterien
Zulassungskriterien
Studienberechtigtes Alter
Akzeptiert gesunde Freiwillige
Studienberechtigte Geschlechter
Beschreibung
DISEASE CHARACTERISTICS:
Diagnosis of 1 of the following hematologic malignancies:
Acute myeloid leukemia (AML)*
- With or without history of myelodysplastic syndromes (MDS)
- Patients in first complete remission (CR) (no greater than 5% blasts in marrow) with translocations t(8;21) and inv(16) are allowed provided they failed first-line induction therapy
Patients in first CR (no greater than 5% blasts in marrow) with translocations t(15;17) are allowed provided at least 1 of the following is true:
- Failed first-line induction therapy
- Molecular evidence of persistent disease
- No patients in first CR and with Down syndrome
Acute lymphoblastic leukemia (ALL)*, meeting 1 of the following criteria:
- Not in first CR (no greater than 5% blasts in marrow)
In first CR and high risk as defined by 1 of the following:
- Hypoploidy (no more than 44 chromosomes)
- Pseudodiploidy with translocations or molecular evidence of t(9;22), 11q23, or t(8;14) (excluding B-ALL) or +MLL gene rearrangement
One of the following elevated WBC levels:
- WBC greater than 100,000/mm^3 if 6 to 12 months of age
- WBC greater than 200,000/mm^3 if between 10 and 17 years of age
- WBC greater than 20,000/mm^3 if 18 years of age and over (adult [over 18 years of age] patient stratum closed to accrual)
- Failed to achieve CR after 4 weeks of induction therapy
B-ALL that is not in first CR or that meets at least 1 of the high-risk criteria specified above
- No translocation t(8;14)
- No blasts with surface immunoglobulins
- CD10 negative
- Undifferentiated leukemia*
- Infant leukemia*
- Biphenotypic leukemia*
Chronic myelogenous leukemia, meeting 1 of the following criteria:
- Accelerated phase
Chronic phase
- At least 1 year from diagnosis without an identified matched unrelated bone marrow donor AND unresponsive to or unable to tolerate interferon
- Blast crisis* (greater than 30% promyelocytes plus blasts in the marrow)
One of the following MDS:
- Refractory anemia
- Refractory anemia with ringed sideroblasts
- Refractory anemia with excess blasts (RAEB)
- RAEB in transformation
- Chronic myelomonocytic leukemia
- Paroxysmal nocturnal hemoglobinuria
Hodgkin's or non-Hodgkin's lymphoma beyond first CR or failed primary induction therapy
- Tumor displays chemosensitivity (greater than 50% reduction in mass size after the most recent therapy) NOTE: *Patients in third or greater medullary relapse or refractory disease (other than primary induction failures) or blast crisis receive the study busulfan/melphalan conditioning regimen)
OR
Diagnosis of one of the following nonmalignant diseases :
Acquired severe aplastic anemia (stratum closed to accrual)
- Unresponsive to medical therapy with anti-thymocyte globulin and/or cyclosporine
Inborn errors of metabolism, including, but not limited to the following:
- Hurler's syndrome
- Adrenoleukodystrophy
- Maroteaux-Lamy syndrome
- Globoid cell leukodystrophy
- Metachromatic leukodystrophy
- Fucosidosis
- Mannosidosis
Fanconi anemia documented by increased chromosomal fragility assays and meeting 1 of the following criteria (stratum closed to accrual):
Severe pancytopenia
- Absolute neutrophil count less than 500/mm^3
- Platelet count less than 20,000/mm^3
- Hemoglobin less than 8 g/dL
- Morphologic evidence of MDS with clonal chromosomal abnormalities
- Leukemia transformation
Other marrow failure syndromes, including any of the following (stratum closed to accrual):
- Blackfan-Diamond syndrome that is unresponsive to medical therapy
- Kostmann's congenital agranulocytosis unresponsive to medical therapy
- Congenital amegakaryocytic thrombocytopenia
- Thrombocytopenia absent radius
Combined immune deficiencies including, but not limited to the following:
- Severe combined immunodeficiency (SCID)
- Wiskott-Aldrich syndrome
- Leukocyte adhesion defect
- Chediak-Higashi disease
- X-linked lymphoproliferative disease
- Adenosine deaminase deficiency
- Purine nucleoside phosphorylase deficiency
- X-linked SCID
- Common variable immune deficiency
- Nezeloff's syndrome
- Cartilage hair hypoplasia
- Reticular dysgenesis
- No active CNS leukemia (cerebrospinal fluid with WBC greater than 5/mm^3 and malignant cells on cytospin)
- No SCID patients who do not require cytoreduction
- No dyskeratosis congenita
- No primary myelofibrosis
- No grade 3 or greater myelofibrosis
Familial erythrophagocytic lymphohistiocytosis patients must not have any of the following:
- Abnormal brain MRI
- Neurologic symptoms
- Lymphocytes and monocytes greater than 7/mm^3 in the cerebrospinal fluid
- No available 5/6 or 6/6 HLA-matched related donor
PATIENT CHARACTERISTICS:
Age
- 55 and under (over 18 closed to accrual)
Performance status
- Karnofsky 70-100% OR
- Lansky 50-100% (patients under 16 years old)
Life expectancy
- Not specified
Hematopoietic
- See Disease Characteristics
Hepatic
- SGOT less than 5 times upper limit of normal
- Bilirubin less than 2.5 mg/dL
Renal
- Creatinine normal for age OR
- Creatinine clearance or glomerular filtration rate greater than 50% of lower limit of normal
Cardiovascular
- LVEF greater than 40% at rest and must improve with exercise* OR
- Shortening fraction greater than 26%* NOTE: *If symptomatic
Pulmonary
- DLCO greater than 45% of predicted* (corrected for hemoglobin)
- FEV_1 and FEC greater than 45% of predicted (corrected for hemoglobin) OR
- Room air oxygen saturation greater than 85%* NOTE: *If symptomatic
Other
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
- No uncontrolled viral, bacterial, or fungal infection
- HIV negative
PRIOR CONCURRENT THERAPY:
Biologic therapy
- See Disease Characteristics
- More than 12 months since prior allogeneic stem cell transplantation with cytoreductive preparative therapy
- More than 6 months since prior autologous stem cell transplantation
Chemotherapy
- See Biologic therapy
Endocrine therapy
- Not specified
Radiotherapy
- Not specified
Surgery
- Not specified
Other
- No prior enrollment on this study
- No continuous life support (e.g., mechanical ventilation) within 1 year after study transplantation (for patients with inborn errors of metabolism)
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Maskierung: Keine (Offenes Etikett)
Mitarbeiter und Ermittler
Sponsor
Ermittler
- Studienstuhl: Philip L. McCarthy, MD, Roswell Park Cancer Institute
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn
Primärer Abschluss (Tatsächlich)
Studienabschluss (Tatsächlich)
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Schätzen)
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Schätzen)
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Schlüsselwörter
- rezidivierendes kleinzelliges Lymphom im Kindesalter
- rezidivierendes großzelliges Lymphom im Kindesalter
- refraktäre Anämie
- refraktäre Anämie mit Ringsideroblasten
- refraktäre Anämie mit übermäßigen Blasten
- refraktäre Anämie mit übermäßigen Blasten in der Transformation
- Chronische myelomonozytäre Leukämie
- de novo myelodysplastische Syndrome
- zuvor behandelte myelodysplastische Syndrome
- sekundäre myelodysplastische Syndrome
- sekundäre akute myeloische Leukämie
- Akute lymphoblastische Leukämie im Kindesalter in Remission
- Akute myeloische Leukämie im Kindesalter in Remission
- Chronische myeloische Leukämie in der chronischen Phase
- atypische chronische myeloische Leukämie
- myelodysplastische/myeloproliferative Erkrankung, nicht klassifizierbar
- unbehandelte akute lymphoblastische Leukämie im Kindesalter
- rezidivierendes/refraktäres Hodgkin-Lymphom im Kindesalter
- chronische myeloische Leukämie in der Blastenphase
- rezidivierende akute lymphoblastische Leukämie im Kindesalter
- Chronische myeloische Leukämie in beschleunigter Phase
- rezidivierende akute myeloische Leukämie im Kindesalter
- rezidivierendes lymphoblastisches Lymphom im Kindesalter
- akute undifferenzierte Leukämie
- unbehandelte akute myeloische Leukämie im Kindesalter und andere myeloische Malignome
Zusätzliche relevante MeSH-Bedingungen
- Pathologische Prozesse
- Erkrankungen des Immunsystems
- Neubildungen nach histologischem Typ
- Neubildungen
- Lymphoproliferative Erkrankungen
- Lymphatische Erkrankungen
- Immunproliferative Erkrankungen
- Erkrankung
- Erkrankungen des Knochenmarks
- Hämatologische Erkrankungen
- Krebsvorstufen
- Lymphom
- Syndrom
- Myelodysplastische Syndrome
- Leukämie
- Präleukämie
- Myeloproliferative Erkrankungen
- Myelodysplastische-myeloproliferative Erkrankungen
- Physiologische Wirkungen von Arzneimitteln
- Molekulare Mechanismen der pharmakologischen Wirkung
- Antiinfektiva
- Autonome Agenten
- Agenten des peripheren Nervensystems
- Enzym-Inhibitoren
- Entzündungshemmende Mittel
- Antirheumatika
- Antimetaboliten, antineoplastisch
- Antimetaboliten
- Antineoplastische Mittel
- Immunsuppressive Mittel
- Immunologische Faktoren
- Antiemetika
- Magen-Darm-Mittel
- Glukokortikoide
- Hormone
- Hormone, Hormonersatzstoffe und Hormonantagonisten
- Neuroprotektive Wirkstoffe
- Schutzmittel
- Antineoplastische Mittel, alkylierend
- Alkylierungsmittel
- Myeloablative Agonisten
- Dermatologische Wirkstoffe
- Antimykotika
- Calcineurin-Inhibitoren
- Methylprednisolon
- Cyclophosphamid
- Melphalan
- Fludarabin
- Fludarabinphosphat
- Busulfan
- Antilymphozyten-Serum
- Cyclosporin
- Cyclosporine
Andere Studien-ID-Nummern
- CDR0000270487
- RPCI-DS-00-22
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