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Immunotherapy

Haplo-Cord Transplantation for Blood Cancer

Phase 2

Waitlist Available

Led By Koen van Besien, MD, PhD

Research Sponsored by Weill Medical College of Cornell University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subject must have a confirmed diagnosis of chronic myelogenous leukemia in chronic, accelerated phase or blast-crisis

Age ≥ 18 years

Must not have

Severely decreased Left Ventricular Ejection Fraction (LVEF) or impaired pulmonary function tests (PFT's)

Evidence of chronic active hepatitis or cirrhosis

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years after transplant

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing whether it's more important to identify a graft that is at least 5/6 matched and inherited paternal antigen (IPA) targeted or the nucleated cell dose when doing a haplo cord transplant.

Who is the study for?

This trial is for adults over 18 with various blood cancers or disorders needing a transplant, like leukemia, lymphoma, myeloma, and more. They should be in good enough health to tolerate the procedure (KPS >= 70%) and have acceptable organ function. It's not suitable for those who can find an exact donor match quickly, are pregnant or breastfeeding, have severe heart/lung problems, active hepatitis/cirrhosis, uncontrolled infections or HIV.

What is being tested?

The study tests whether matching a cord blood graft to inherited paternal antigens (IPAs) improves haplo-cord transplant outcomes compared to just focusing on cell dose. Participants receive stem cells from both umbilical cord blood and a half-matched family donor after chemotherapy conditioning but no experimental drugs are used.

What are the potential side effects?

Potential side effects include reactions related to immune suppression such as infection risk increase due to medications like anti-thymocyte globulin and Tacrolimus; chemotherapy-related nausea; fatigue; mouth sores from Fludarabine/Melphalan; infusion reactions from Rituximab; and complications from Total Body Irradiation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with chronic myelogenous leukemia in a specific phase.

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I am 18 years old or older.

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I have been diagnosed with recurrent or refractory lymphoma.

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I have been diagnosed with myelodysplastic syndrome.

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I have been diagnosed with multiple myeloma.

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I have been diagnosed with a chronic blood disease.

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I have been diagnosed with a blood disorder affecting my hemoglobin.

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I have been diagnosed with aplastic anemia.

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I have a blood disorder requiring a stem cell transplant.

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My leukemia has returned or didn't respond to treatment.

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My acute leukemia is in its first remission and is at high risk of coming back.

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I have chronic lymphocytic leukemia that has come back or is hard to treat.

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I don't have a matching donor available quickly.

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My liver and kidney functions are within the required range.

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I am mostly able to care for myself but may not be able to do active work.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My heart's pumping ability is severely reduced, or my lung tests show poor function.

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I have long-term active hepatitis or cirrhosis.

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My HIV is not under control.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years after transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~5 years after transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years after transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of Subjects Who Achieved Engraftment With De-Escalating Umbilical Cord Total Nucleated Cell (TNC) Dose

Secondary study objectives

Impact of IPA Targeting on Transplant Outcome

Impact of NIMA Matching on Transplant Outcome

Long-term Survival of Subjects Undergoing Haplo-cord Transplants

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups

Experimental Treatment

Group I: Cohort 4Experimental Treatment8 Interventions

All subjects in this cohort will receive the minimum required cell dose that is determined following the dose de-escalation portion of the study (cohorts 1 through 3) Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device.

Group II: Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kgExperimental Treatment8 Interventions

All subjects in this cohort will receive a minimal cell dose of 0.5 x 10\^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device.

Group III: Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kgExperimental Treatment8 Interventions

All subjects in this cohort will receive a minimal cell dose of 1 x 10\^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device.

Group IV: Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kgExperimental Treatment8 Interventions

All subjects in this cohort will receive a minimal cell dose of 2 x 10\^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fludarabine

2012

Completed Phase 4

~1860

Melphalan

2008

Completed Phase 3

~1500