Reduced-intensity, Related-donor Bone Marrow Transplantation Followed by High-dose Cyclophosphamide for Hematologic Cancers

NCT01135329 | Terminated Phase 2 Results DMC

• 2 other identifiers: J1046NA_00039363
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

This research is being done to learn more about reduced-intensity bone marrow transplantation (BMT), also known as a "mini" transplant for patients with blood cancers, using bone marrow from a relative. The main goal of the study is to determine how quickly the donor's bone marrow "takes" in your body. Other goals include describing how many people accept the bone marrow and how quickly the blood counts come up; describing Graft-versus-host disease (GVHD) and other complications; and describing how many people survive without progressive cancer and survive overall

Conditions

Lymphoma; Leukemia; Myelodysplastic Syndrome

Keywords

Lymphoma; Hodgkin's lymphoma; Non hodgkin's lymphoma; Leukemia; Acute myeloid leukemia (AML); Acute lymphoblastic leukemia(ALL); Chronic myeloid leukemia (CML); Chronic Myelomonocytic (CMML); Myelodysplastic syndrome (MDS); High-risk acute leukemia in first remission; Relapsed leukemia in remission; Cyclophosphamide; High-dose cyclophosphamide; Fludarabine; Busulfan; Allogeneic; Nonmyeloablative; Reduced intensity; Haploidentical; Bone marrow transplant (BMT)

Outcome Measures

Primary Outcomes (2)

• Chimerism in Unsorted Peripheral Blood

  Percentage of participants achieving full-donor chimerism in unsorted peripheral blood.

  Day 60

• Chimerism in CD3+ Sorted Peripheral Blood

  Percentage of participants achieving full-donor chimerism in CD3+ sorted peripheral blood

  Day 60

Secondary Outcomes (6)

• Overall Survival

  1 year

• Progression-free Survival

  1 year

• Incidence of Relapse

  1 year

• Non-relapse Mortality

  1 year

• Incidence of Graft-versus-host-disease (GVHD)

  1 year

Study Arms (1)

BMT Allogenic Transplantation

EXPERIMENTAL

Reduced-intensity transplant with a fludarabine- and busulfan-based preparative regimen. GVHD prophylaxis with cyclophosphamide, tacrolimus, and mycophenolate mofetil.

Drug: Fludarabine; Drug: Busulfan; Drug: Cyclophosphamide; Drug: Mycophenolate Mofetil; Drug: Tacrolimus

Interventions

Fludarabine DRUG

30 mg/m\^2 IV daily on Day -6 through Day -2.

Also known as: Fludara, Flu

BMT Allogenic Transplantation

Busulfan DRUG

1 mg/kg PO OR 0.8 mg/kg IV four times daily on Day -6 through Day -3.

Also known as: Busulfex, Myleran

BMT Allogenic Transplantation

Cyclophosphamide DRUG

50 mg/kg IV daily on Day +3 and Day +4.

Also known as: CTX, Cytoxan, Cy

BMT Allogenic Transplantation

Mycophenolate Mofetil DRUG

15 mg/kg PO three times daily (max daily dose of 3g) starting on Day +5.

Also known as: MMF, CellCept

BMT Allogenic Transplantation

Tacrolimus DRUG

Dosed based on drug levels; begin on Day +5 at 1 mg IV daily.

Also known as: FK506, FK-506, Prograf

BMT Allogenic Transplantation

Eligibility Criteria

• Age: 6 Months - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• First-degree related donor who is at minimum HLA haploidentical
• Eligible diagnoses:
• Low-grade non-Hodgkin's lymphoma or plasma cell neoplasm that has progressed during multiagent therapy, failed at least two prior therapies (excluding single agent rituximab and single agent steroids), or in the case of lymphoma undergone histological conversion:
• Follicular grade 1 or 2 lymphoma
• Follicular lymphoma not otherwise specified
• Marginal zone (or MALT) lymphoma
• Lymphoplasmacytic lymphoma / Waldenstrom's macroglobulinemia
• Hairy cell leukemia
• Small lymphocytic lymphoma (SLL) or chronic lymphocytic leukemia (CLL)
• Prolymphocytic leukemia
• Low grade B-cell lymphoma, unspecified
• Multiple myeloma
• Plasma cell leukemia
• Poor-risk SLL or CLL, defined by an 11q or 17p deletion, histological conversion, or disease progression \< 6 months after a purine analog-containing regimen
• Aggressive lymphoma that has failed at least one prior regimen of multiagent chemotherapy, and patient is either ineligible for autologous BMT or autologous BMT is not recommended:

You may not qualify if:

• Pregnant or breast-feeding
• Uncontrolled infection Note: Infection is permitted if there is evidence of response to medication. Eligibility of HIV infected patients will be determined on a case-by-case basis.
• Any previous BMT within 3 months prior to start of conditioning
• Active extra-medullary leukemia or known active Central Nervous System (CNS) involvement by malignancy. Such disease treated into remission is permitted.

Sponsors & Collaborators

• Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins: lead

Study Sites (1)

The Sydney Kimmel Comprehensive Cancer Center

Baltimore, Maryland, 21231, United States

Location

MeSH Terms

Conditions

Lymphoma; Leukemia; Myelodysplastic Syndromes; Hodgkin Disease; Lymphoma, Non-Hodgkin; Leukemia, Myeloid, Acute; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Interventions

fludarabine; fludarabine phosphate; Influenza Vaccines; Busulfan; Cyclophosphamide; Mycophenolic Acid; Tacrolimus

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Hematologic Diseases; Bone Marrow Diseases; Leukemia, Myeloid; Leukemia, Lymphoid; Myeloproliferative Disorders; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Viral Vaccines; Vaccines; Biological Products; Complex Mixtures; Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Macrolides; Lactones

Results Point of Contact

• Title: Yvette Kasamon
• Organization: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

ykasamo1@jhmi.edu

410-955-8839

Study Officials

• Yvette Kasamon, M.D.

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 1, 2010
• First Posted: June 2, 2010
• Study Start: August 1, 2010
• Primary Completion: May 1, 2012
• Study Completion: May 1, 2012
• Last Updated: May 27, 2026
• Results First Posted: July 3, 2018

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)