URMC Related Haplo-identical Donor BMT (HaploOnly)

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University of Rochester

Status and phase

Completed
Phase 1

Conditions

Myelofibrosis
Multiple Myeloma
Immune Deficiencies
Lymphoma
Hematological Disease
Solid Tumors

Treatments

Drug: Fludarabine
Drug: Post-Stem Cell Infusion Mesna
Drug: Melphalan
Drug: Busulfan
Drug: Pre-Stem Cell Infusion Mesna
Drug: Post-Stem Cell Infusion Cyclophosphamide
Procedure: Stem Cell Infusion
Drug: Thiotepa
Drug: Pre-Stem Cell Infusion Cyclophosphamide
Radiation: Total Body Irradiation 1200 cGy

Study type

Interventional

Funder types

Other

Identifiers

NCT02660281
UBMT 15056

Details and patient eligibility

About

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.

Full description

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.

Enrollment

74 patients

Sex

All

Ages

6 months to 75 years old

Volunteers

No Healthy Volunteers

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

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

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Parallel Assignment

Masking

None (Open label)

74 participants in 5 patient groups

Full Intensity TBI-based Conditioning
Other group
Description:
Total Body Irradiation 1200 cGy of 150 cGy over 4 or 5 days, days -5 or -4 to -1 Fludarabine 30 mg/m2/day x 3 days, days -6, -5, -4 Stem Cell Infusion, day 0 Post- Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Mesna 50 mg/kg/day x 2 days, days +3 and +4
Treatment:
Radiation: Total Body Irradiation 1200 cGy
Procedure: Stem Cell Infusion
Drug: Post-Stem Cell Infusion Cyclophosphamide
Drug: Post-Stem Cell Infusion Mesna
Drug: Fludarabine
Full Intensity Chemo-Only Conditioning
Other group
Description:
Fludarabine 25 mg/m2/day x 5 days, days -6, -5, -4, -3, -2 Busulfan 130 mg/m2/day x 4 days, days -6, -5, -4, -3 Pre-Stem Cell Infusion Cyclophosphamide 14.5 mg/kg/day x 2 days, days -3 and -2 Pre-Stem Cell Infusion Mesna 14.5 mg/kg/day x 2 days, days -3 and -2 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Post-Stem Cell Infusion Mesna 50 mg/kg/day x 2 days, days +3 and +4
Treatment:
Drug: Pre-Stem Cell Infusion Cyclophosphamide
Procedure: Stem Cell Infusion
Drug: Post-Stem Cell Infusion Cyclophosphamide
Drug: Pre-Stem Cell Infusion Mesna
Drug: Busulfan
Drug: Post-Stem Cell Infusion Mesna
Drug: Fludarabine
Reduced Intensity Conditioning
Other group
Description:
Fludarabine 30 mg/m2/day x 5 days, days -6 to -2 Melphalan 140 mg/m2/day x 1 day, day -2 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Post-Stem Cell InfusionMesna 50 mg/kg/day x 2 days, days +3 and +4
Treatment:
Procedure: Stem Cell Infusion
Drug: Post-Stem Cell Infusion Cyclophosphamide
Drug: Melphalan
Drug: Post-Stem Cell Infusion Mesna
Drug: Fludarabine
Non-Myeloablative Conditioning
Other group
Description:
Fludarabine 30 mg/m2/day x 5 days, days -6 to -2 Pre-Stem Cell Infusion Cyclophosphamide 14.5 mg/kg/day x 2 days, days -3 and -2 Pre-Stem Cell InfusionMesna 14.5 mg/kg/day x 2 days, days -3 and -2 Total Body Irradiation 200 cGy, day -1 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, day +3 and +4 Post-Stem Cell Infusion Mesna 50 mg/kg/day x 2 days, day +3 and +4
Treatment:
Drug: Pre-Stem Cell Infusion Cyclophosphamide
Radiation: Total Body Irradiation 1200 cGy
Procedure: Stem Cell Infusion
Drug: Post-Stem Cell Infusion Cyclophosphamide
Drug: Pre-Stem Cell Infusion Mesna
Drug: Post-Stem Cell Infusion Mesna
Drug: Fludarabine
Reduced Intensity Conditioning with Addition of Thiotepa
Other group
Description:
Fludarabine 30 mg/m2/day x 5 days, days -6 to -2 Thiotepa 8 mg/kg, day -3 Melphalan 140 mg/m2/day x 1 day, day -2 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Post-Stem Cell InfusionMesna 50 mg/kg/day x 2 days, days +3 and +4
Treatment:
Drug: Thiotepa
Procedure: Stem Cell Infusion
Drug: Post-Stem Cell Infusion Cyclophosphamide
Drug: Melphalan
Drug: Post-Stem Cell Infusion Mesna
Drug: Fludarabine

Trial contacts and locations

1

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Data sourced from clinicaltrials.gov

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