MedDataX Logo

URMC Related Haplo-identical Donor BMT

NCT ID: NCT02660281

Last Updated: 2021-11-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

74 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-10-31

Study Completion Date

2021-01-07

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hematological Disease Immune Deficiencies Solid Tumors Myelofibrosis Multiple Myeloma Lymphoma

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Full Intensity TBI-based Conditioning

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

Group Type OTHER

Total Body Irradiation 1200 cGy

Intervention Type RADIATION

1200 cGy TBI in 8 fractions

Fludarabine

Intervention Type DRUG

Fludarabine

Stem Cell Infusion

Intervention Type PROCEDURE

Stem cell infusion

Post-Stem Cell Infusion Cyclophosphamide

Intervention Type DRUG

Cyclophosphamide given after the stem cell infusion

Post-Stem Cell Infusion Mesna

Intervention Type DRUG

Mesna given after the Stem Cell Infusion

Full Intensity Chemo-Only Conditioning

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

Group Type OTHER

Fludarabine

Intervention Type DRUG

Fludarabine

Pre-Stem Cell Infusion Cyclophosphamide

Intervention Type DRUG

Cyclophosphamide given prior to the stem cell infusion

Pre-Stem Cell Infusion Mesna

Intervention Type DRUG

Mesna given prior to the stem cell infusion

Busulfan

Intervention Type DRUG

Busulfan

Stem Cell Infusion

Intervention Type PROCEDURE

Stem cell infusion

Post-Stem Cell Infusion Cyclophosphamide

Intervention Type DRUG

Cyclophosphamide given after the stem cell infusion

Post-Stem Cell Infusion Mesna

Intervention Type DRUG

Mesna given after the Stem Cell Infusion

Reduced Intensity Conditioning

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

Group Type OTHER

Fludarabine

Intervention Type DRUG

Fludarabine

Melphalan

Intervention Type DRUG

Melphalan

Stem Cell Infusion

Intervention Type PROCEDURE

Stem cell infusion

Post-Stem Cell Infusion Cyclophosphamide

Intervention Type DRUG

Cyclophosphamide given after the stem cell infusion

Post-Stem Cell Infusion Mesna

Intervention Type DRUG

Mesna given after the Stem Cell Infusion

Non-Myeloablative Conditioning

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

Group Type OTHER

Total Body Irradiation 1200 cGy

Intervention Type RADIATION

1200 cGy TBI in 8 fractions

Fludarabine

Intervention Type DRUG

Fludarabine

Pre-Stem Cell Infusion Cyclophosphamide

Intervention Type DRUG

Cyclophosphamide given prior to the stem cell infusion

Pre-Stem Cell Infusion Mesna

Intervention Type DRUG

Mesna given prior to the stem cell infusion

Stem Cell Infusion

Intervention Type PROCEDURE

Stem cell infusion

Post-Stem Cell Infusion Cyclophosphamide

Intervention Type DRUG

Cyclophosphamide given after the stem cell infusion

Post-Stem Cell Infusion Mesna

Intervention Type DRUG

Mesna given after the Stem Cell Infusion

Reduced Intensity Conditioning with Addition of Thiotepa

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

Group Type OTHER

Fludarabine

Intervention Type DRUG

Fludarabine

Melphalan

Intervention Type DRUG

Melphalan

Stem Cell Infusion

Intervention Type PROCEDURE

Stem cell infusion

Post-Stem Cell Infusion Cyclophosphamide

Intervention Type DRUG

Cyclophosphamide given after the stem cell infusion

Post-Stem Cell Infusion Mesna

Intervention Type DRUG

Mesna given after the Stem Cell Infusion

Thiotepa

Intervention Type DRUG

Thiotepa

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Total Body Irradiation 1200 cGy

1200 cGy TBI in 8 fractions

Intervention Type RADIATION

Fludarabine

Fludarabine

Intervention Type DRUG

Pre-Stem Cell Infusion Cyclophosphamide

Cyclophosphamide given prior to the stem cell infusion

Intervention Type DRUG

Pre-Stem Cell Infusion Mesna

Mesna given prior to the stem cell infusion

Intervention Type DRUG

Busulfan

Busulfan

Intervention Type DRUG

Melphalan

Melphalan

Intervention Type DRUG

Stem Cell Infusion

Stem cell infusion

Intervention Type PROCEDURE

Post-Stem Cell Infusion Cyclophosphamide

Cyclophosphamide given after the stem cell infusion

Intervention Type DRUG

Post-Stem Cell Infusion Mesna

Mesna given after the Stem Cell Infusion

Intervention Type DRUG

Thiotepa

Thiotepa

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

TBI

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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 -
Minimum Eligible Age

6 Months

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

University of Rochester

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Jeffrey Andolina

Medical Director, Pediatric BMT

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Jeffrey Andolina, MD

Role: PRINCIPAL_INVESTIGATOR

Wilmot Cancer Center

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Wilmot Cancer Institute

Rochester, New York, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

UBMT 15056

Identifier Type: -

Identifier Source: org_study_id