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Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer

19. juli 2021 oppdatert av: Roswell Park Cancer Institute

Allogeneic Blood or Marrow Transplantation for Hematologic Malignancy and Aplastic Anemia

RATIONALE: Giving chemotherapy drugs and total-body irradiation before a donor stem cell helps stop the growth of cancer or abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known which combination chemotherapy regimen is most effective when given before a donor stem cell transplant in treating aplastic anemia or hematologic cancer.

PURPOSE: This phase II/III trial is studying different combination chemotherapy regimens to compare how well they work when given before donor stem cell transplant in treating patients with aplastic anemia or hematologic cancer.

Studieoversikt

Detaljert beskrivelse

OBJECTIVES:

  • Compare the morbidity, mortality, and overall outcome of patients with severe aplastic anemia or hematologic malignancy treated with standard vs novel conditioning regimens followed by allogeneic stem cell transplantation.
  • Examine the influence of donor histocompatibility on outcome by comparing matched/related, mismatched/related (with or without T-cell depletion), and matched/unrelated transplants with stratification for type of preparative regimen.
  • Ensure that patients with uncommon diagnoses will be treated in a uniform fashion with the best therapy available.

OUTLINE: Patients are stratified according to risk of relapse (standard-risk: acute leukemia in first complete remission, chronic myelogenous leukemia in first chronic phase, lymphoma in sensitive first relapse or second remission, primary or untreated myelodysplastic syndromes, or untreated severe aplastic anemia vs high-risk: all others).

Patients are assigned to one of the following conditioning regimens based on diagnosis, risk of relapse, and donor relatedness:

  • Regimen 1: Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
  • Regimen 2: Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3.
  • Regimen 3: Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
  • Regimen 4: Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
  • Regimen 5: Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
  • Regimen 6: Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4.
  • Regimen 7: Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2.
  • Regimen 8: Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1.
  • Regimen 9: Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.

All patients then receive donor stem cell infusions on day 0. Some patients may undergo involved-field radiotherapy 4-8 weeks after transplant.

Patients will be taken off study after a minimum of 4 years of follow up.

PROJECTED ACCRUAL: At least 405 patients will be accrued for this study within 5 years.

Studietype

Intervensjonell

Registrering (Faktiske)

361

Fase

  • Fase 2
  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

    • New York
      • Buffalo, New York, Forente stater, 14263-0001
        • Roswell Park Cancer Institute

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

2 måneder til 66 år (Barn, Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

DISEASE CHARACTERISTICS:

  • Diagnosis of one of the following:

    • Severe aplastic anemia as defined by either of the following:

      • Marrow cellularity (< 25% [or 25-50% cellularity with < 30% of remaining cells hematopoietic in origin])
      • At least 2 of the following abnormal peripheral blood counts:

        • Reticulocyte count < 1% (corrected for hematocrit)
        • Platelet count < 20,000/mm^3
        • Neutrophil count < 500/mm^3
    • Histologically confirmed hematologic malignancy, including any of the following:

      • Acute leukemia

        • Resistant or recurrent disease after combination chemotherapy with at least one standard regimen OR in first remission and at high risk of relapse
        • Acute myeloid leukemia (AML) (antecedent myelodysplastic syndromes [MDS], secondary AML, or high-risk cytogenetic abnormalities)
        • Acute lymphoblastic leukemia (ALL) (high-risk cytogenetic abnormalities)
      • Chronic myeloid leukemia (CML)

        • Chronic phase, accelerated phase, or blast phase
      • Myeloproliferative disorders or MDS, including any of the following:

        • Myelofibrosis
        • Polycythemia vera*
        • Essential thrombocythemia*
        • Refractory anemia
        • Refractory anemia with excess blasts
        • Refractory anemia with excess blasts in transformation
        • Chronic myelomonocytic leukemia NOTE: * Only if transformed to AML or MDS
      • Lymphoproliferative disease

        • Recurrent or persistent, symptomatic disease after first-line chemotherapy, including any of the following:

          • Chronic lymphocytic leukemia (CLL) (≥ 20% marrow involvement)
          • Waldenstrom macroglobulinemia
          • Low-grade non-Hodgkin lymphoma
      • Intermediate or high-grade non-Hodgkin lymphoma, meeting 1 of the following criteria:

        • Resistant or recurrent disease after combination chemotherapy with one standard regimen
        • Lymphoblastic lymphoma or small noncleaved cell lymphoma in first remission and at high risk of relapse
        • CNS disease
        • Bone marrow disease and LDH greater than 300
    • Solid tumor that would otherwise be treated on RPCI-DS-9115 (or equivalent autologous stem transplant protocol) AND has a syngeneic donor
  • Autologous bone marrow transplant not possible (or desirable) due to 1 of the following:

    • History of marrow tumor
    • Inadequate marrow dose
    • Abnormal marrow histology or function prior to storage
    • Thrombocytopenia or leukopenia
    • Marrow cellularity < 20%
  • Histocompatible donor identified

    • Well-matched donor, as defined by 1 of the following:

      • Family member matched for 5 or 6 HLA specificities (A, B, DR)*
      • Unrelated donor meeting compatibility criteria of the National Marrow Donor Program (matched for HLA A, B, and DRB1 antigens)*
      • Identical twin sibling
    • If a compatible cord blood donor is identified and there is no suitable unrelated donor available, patient may receive cord blood transplant NOTE: *Patients ≤ 25 years of age may be singly mismatched at the A or B loci

NOTE: A new classification scheme for adult non-Hodgkin's lymphoma has been adopted by PDQ. The terminology of "indolent" or "aggressive" lymphoma will replace the former terminology of "low", "intermediate", or "high" grade lymphoma. However, this protocol uses the former terminology.

PATIENT CHARACTERISTICS:

Age:

  • 4 to 70

Performance status:

  • Zubrod 0-2 OR
  • Karnofsky 70-100%

Life expectancy:

  • Not specified

Hematopoietic:

  • See Disease Characteristics

Hepatic:

  • Bilirubin < 3 times normal (unless due to disease)
  • Alkaline phosphatase < 3 times normal (unless due to disease)
  • SGOT < 3 times normal (unless due to disease)
  • Hepatitis B surface antigen negative
  • No severe hepatic disease that would preclude study participation

Renal:

  • Creatinine normal
  • Creatinine clearance ≥ 50 mL/min
  • No severe renal disease that would preclude study participation

Cardiovascular:

  • Cardiac ventricular ejection fraction ≥ 50% by MUGA or echocardiogram
  • No uncontrolled or severe cardiovascular disease (e.g., myocardial infarction, congestive heart failure, symptomatic angina, life threatening arrhythmia, or hypertension within the past 6 months)

Pulmonary:

  • DLCO or DLVA ≥ 50% predicted (corrected for hemoglobin or alveolar ventilation)

Other:

  • No serious concurrent medical or psychiatric illness
  • No other serious organ dysfunction (unless due to underlying disease), including the following:

    • Uncontrolled bacterial, viral, or fungal infection
    • Uncontrolled peptic ulcer disease
    • Uncontrolled diabetes mellitus
  • HIV negative
  • Cytomegalovirus status known
  • Not pregnant

PRIOR CONCURRENT THERAPY:

Biologic therapy:

  • Not specified

Chemotherapy:

  • See Disease Characteristics
  • Pretransplant cytoreductive chemotherapy allowed for patients with relapsed or refractory disease

Endocrine therapy:

  • Not specified

Radiotherapy:

  • Not eligible for total-body irradiation if prior radiotherapy exceeded the following limits:

    • Mediastinum: 3,600 cGy
    • Heart: 3,600 cGy
    • Whole lungs: 1,200 cGy
    • Small bowel: 3,600 cGy
    • Kidneys: 1,200 cGy
    • Whole liver: 1,600 cGy
    • Cranial spinal: 3,600 cGy
    • Brain: 4,000 cGy
    • Retina: 4,000 cGy

Surgery:

  • Not specified

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Regimen 1
Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
Gitt IV
Gitt IV
Eksperimentell: Regimen 2
Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3.
Gitt IV
Gitt IV
Eksperimentell: Regimen 3
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
Gitt IV
Given twice daily for 3 days
Eksperimentell: Regimen 4
Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
Gitt IV
Gitt IV
Eksperimentell: Regimen 5
Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
Gitt IV
Gitt IV
Given twice daily for 3 days
Eksperimentell: Regimen 6
Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4.
Gitt IV
Gitt IV
Gitt IV
Eksperimentell: Regimen 7
Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2.
Gitt IV
Gitt IV
Eksperimentell: Regimen 8
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1.
Gitt IV
Gitt IV
Given twice daily for 3 days
Eksperimentell: Regimen 9
Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
Gitt IV
Gitt IV
Gitt IV

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
CR Rate
Tidsramme: day 100
Rate of Complete Remission by Day +100
day 100

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Toxicity/TRM at Day 100
Tidsramme: Day +100
Death due to treatment related causes before day +100 after BMT
Day +100
4 Year PFS
Tidsramme: 4 years
progression free survival estimate at 4 years post BMT (events are disease progression/relapse and death due to any cause)
4 years
4 yr OS
Tidsramme: 4-year
Overall survival estimate at 4 years post BMT
4-year

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Etterforskere

  • Studiestol: Philip L. McCarthy, MD, Roswell Park Cancer Institute

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

19. oktober 1998

Primær fullføring (Faktiske)

12. juli 2019

Studiet fullført (Faktiske)

12. juli 2019

Datoer for studieregistrering

Først innsendt

1. november 1999

Først innsendt som oppfylte QC-kriteriene

26. januar 2003

Først lagt ut (Anslag)

27. januar 2003

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

13. august 2021

Siste oppdatering sendt inn som oppfylte QC-kriteriene

19. juli 2021

Sist bekreftet

1. juli 2021

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Andre studie-ID-numre

  • RP 98-15 (Annen identifikator: Roswell Park Cancer Institute)

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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