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Completed

NCT Number: NCT02660281

URMC Related Haplo-identical Donor BMT

This study will be a single-center treatment protocol, designed to validate the process of related donor haploidentical-SCT at the Wilmot Cancer Institute Blood and Marrow Transplant Unit.

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Key information

Conditions

Hematological Disease Blood Protein Disorders Bone Marrow Diseases Cardiovascular Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders Hemostatic Disorders Immune Deficiencies Immune System Diseases Immunoproliferative Disorders Lymphatic Diseases Lymphoma Lymphoproliferative Disorders Multiple Myeloma Myelofibrosis Myeloproliferative Disorders Neoplasms Neoplasms by Histologic Type Neoplasms, Plasma Cell Paraproteinemias Primary Myelofibrosis Solid Tumors Vascular Diseases

Age range

6 month–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Wilmot Cancer Institute

Rochester, New York, 14642, United States

About this study

This study will be a single-center treatment protocol with five possible preparative regimens, designed to validate the process of related donor haploidentical-SCT at the Wilmot Cancer Institute Blood and Marrow Transplant Unit. Enrolled patients will receive chemotherapy +/- radiation as a pre-transplant conditioning regimen. Patients will then receive haploidentical stem cells, either bone marrow or mobilized peripheral blood, followed by GvHD prophylaxis that will include cyclophosphamide. Multiple data points will be collected prior to, during, and following transplantation to ensure safety of the process and to evaluate the stated objectives.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Patient Age:

  • Pediatric (ages 6 months to 18 years)
  • Adult (ages 18-75 years)

Disease:

Congenital and Other Non-malignant Disorders

  • Immunodeficiency disorders (e.g. Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome)
  • Congenital hematopoietic stem cell defects (e.g. Chediak-Higashi Syndrome, Congenital Osteopetrosis, Osteogenesis Imperfecta)
  • Metabolic disorders (e.g. Hurler's Syndrome)
  • Hemoglobinopathies (e.g. Sickle Cell Disease, Thalassemia)
  • Severe aplastic anemia

High-Risk Leukemias

Acute Myelogenous Leukemia

  • Refractory to standard induction therapy (more than 1 cycle required to achieve remission)
  • Recurrent (in CR≥2)
  • Treatment-related AML or MDS
  • Evolved from myelodysplastic syndrome
  • Presence of Flt3 abnormalities
  • FAB M6 or M7
  • Adverse cytogenetics

Myelodysplastic Syndrome

Acute Lymphoblastic Leukemia including T lymphoblastic leukemia

  • Refractory to standard induction therapy (time to CR >4 weeks)
  • Recurrent (in CR ≥2)
  • WBC count >30,000/mcL at diagnosis
  • Age >30 at diagnosis
  • Adverse cytogenetics, such as (t(9:22), t(1:19), t(4:11), other MLL rearrangements.

Chronic Myelogenous Leukemia in accelerated phase or blast crisis

Biphenotypic or undifferentiated leukemia

Burkitt's leukemia or lymphoma

Lymphoma:

  • Large cell, Mantle cell, Hodgkin lymphoma refractory or recurrent, chemosensitive, and ineligible for an autologous stem cell transplant or previously treated with autologous SCT
  • Marginal zone or follicular lymphoma that is progressive after at least two prior therapies

Multiple Myeloma, recurrent following high-dose therapy and autologous SCT or ineligible for an autologous HSCT

Solid tumors, with efficacy of allogeneic HSCT demonstrated for the specific disease and disease status

Graft failure following prior related donor, unrelated donor or UCB transplant

Myelofibrosis

Exclusion criteria

  • Patient Age below 6 months or over 75 years
  • Availability of a 10/10 HLA-matched related or unrelated donor within a reasonable time-frame dictated by the clinical urgency of the transplant
  • Autologous HSCT < 6 months prior to proposed haplo-SCT
  • Pregnant or breast-feeding
  • Current uncontrolled infection
  • Evidence of HIV infection or positive HIV serology
  • Anti-donor HLA antibodies with positive crossmatch and unsuccessful -

Treatment and study plan

Total Body Irradiation 1200 cGy

Radiation

1200 cGy TBI in 8 fractions

Other names: TBI

Fludarabine

Drug

Fludarabine

Pre-Stem Cell Infusion Cyclophosphamide

Drug

Cyclophosphamide given prior to the stem cell infusion

Pre-Stem Cell Infusion Mesna

Drug

Mesna given prior to the stem cell infusion

busulfan

Drug

Busulfan

melphalan

Drug

Melphalan

Stem Cell Infusion

Procedure

Stem cell infusion

Post-Stem Cell Infusion Cyclophosphamide

Drug

Cyclophosphamide given after the stem cell infusion

Post-Stem Cell Infusion Mesna

Drug

Mesna given after the Stem Cell Infusion

Thiotepa

Drug

Thiotepa

Primary outcomes

  1. The number of days from the date of the stem cell infusion to engraftment of absolute neutrophils (ANC) and platelets (PLT) will be determined based on daily CBC and differential counts.

    Time frame: 42 days following the infusion of stem cells for ANC. [If engraftment of ANC does not occur within 42 days, a subsequent transplant will be performed if a donor is available.

    The date of engraftment of ANC is the first of 3 consecutive days of ANC of 500 or higher. The date of engraftment of platelets is the first of 3 consecutive days of platelet counts of 20,000 or higher in the absence of platelet transfusions for at least 7 days prior.

  2. Rate of non-engraftment and secondary graft failure

    Time frame: At 100 days, 6 months, and yearly after transplant from the date of transplant until the date of documented graft failure or the subject's date of death up to 120 months.

    The percentage of patients who fail to engraft ANC will be tabulated as well as the percentage of patients who have primary engraftment of ANC but whose graft then fails as evidenced by pancytopenia and failure of bone marrow function.

Secondary outcomes

  1. Percentage of subjects who develop acute graft-versus-host disease.

    Time frame: 100 days following the infusion of stem cells

    Assess the presence and date of onset of acute GvHD based on the criteria published by Przepiorka et al., Bone Marrow Transplant 1995; 15(6):825-8 as used by the Center for International Blood & Marrow Transplant Research.

  2. Assess the Maximum Overall Grades 0- IV of acute GvHD and Maximum Severity (0-4) by involved organ system in patients who develop acute graft-versus-host disease.

    Time frame: 100 days following the infusion of stem cells

    Assess the severity of acute GvHD based on the criteria published by Przepiorka et al., Bone Marrow Transplant 1995; 15(6):825-8 as used by the Center for International Blood & Marrow Transplant Research.

  3. Percentage of subjects who develop chronic graft-versus-host disease.

    Time frame: Minimally at intervals of 100 days, 6 months and then yearly following the infusion of stem cells until the subject's date of death.or up to 120 months.

    Calculate the percentage of patients who develop chronic graft-versus-host disease based on Sullivan KM, Blood 1981; 57:267 as used by the Center for International Blood & Marrow Transplant Research.

  4. Assess the Maximum Grade: Limited versus Extensive; Maximum Severity: Mild, Moderate or Severe) of chronic GvHD in patients who develop chronic graft-versus-

    Time frame: Minimally at intervals of 100 days, 6 months and then yearly following the infusion of stem cells until the subject's date of death or up to 120 months.

    Assess the severity of chronic GvHD in patients who develop chronic graft-versus-host disease based on Sullivan KM, Blood 1981; 57:267 as used by the Center for International Blood & Marrow Transplant Research.

  5. Disease-free survival

    Time frame: Disease assessments are performed and reported at time points that include 100 days, 6 months, and yearly until documented progression of disease or the date of death or up to 120 months.

    Document and update the length of time a subject survives without recurrence of the disease for which they were transplanted minimally at 100 days. 6 months, and yearly following the infusion of stem cells for as long as the patient survives and remains disease-free.

  6. Time to relapsed disease

    Time frame: Disease assessments are performed and reported at time points that include 100 days, 6 months, and yearly until the date of documented progression or the subject's date of death or up to 120 months.

    Document the time to relapse of the disease for which the patient was Minimally assessments will be performed at 100 days. 6 months, and yearly following the infusion of stem cells for as long as the patient survives and remains disease-free.

  7. Immune reconstitution

    Time frame: At 100 day, 6 month and one year intervals following the infusion of stem cells until the subject's date of death or up to 120 months.

    Evaluate immune reconstitution by measurement of immunoglobulins (IgG, IgA, and IgM), assessment of infections, and lymphocyte analysis

Sponsors and collaborators

Lead sponsor

University of Rochester

Other

Registry information

Official study title

Haploidentical Donor Hematopoietic Stem Cell Transplantation

Acronym: HaploOnly

Important dates

Study start
2015
Primary completion
2019
Study completion
2021
First posted
Jan 21, 2016
Registry last updated
Nov 3, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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