Haploidentical Donor Hematopoietic Stem Cell Transplant in Treating Patients With Hematologic Malignancies

NCT01871441 | Terminated Phase 2 Results DMC

• 3 other identifiers: 13D.1272012-104JT 3048
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief Summary

This phase II trial studies how well haploidentical donor hematopoietic stem cell transplant works in treating patients with hematologic malignancies. Giving chemotherapy and total-body irradiation before a donor stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. Giving an infusion of the donor's T cells (donor lymphocyte infusion) may replace the patient's immune cells and help destroy any remaining cancer cells. When the stem cells from a related donor, that closely matches the patient's blood, are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets.

Conditions

Malignant Neoplasm

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Disease-free Survival (DFS)

  Disease free survival (DFS), defined as the time to death, relapse or disease progression.

  1 year

Secondary Outcomes (3)

• Number of Participants With Relapse of Disease

  Up to 1 year

• Rate of Grade III-IV GVHD in Female Recipients With Male Donors

  Up to 1 year

• The Rates of Grade III-IV GVHD in Female Recipients With Male Donors Will be Computed With Corresponding Exact Binomial 95% Confidence Intervals.

  Up to 1 year

Study Arms (1)

Treatment (haploidentical allogeneic HSCT)

EXPERIMENTAL

Patients undergo TBI BID on days -9 to -6, undergo DLI on day -6, and receive cyclophosphamide IV over 2 hours on days -3 and -2. TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42, and mycophenolate mofetil IV BID from day -1 to day 28.

Radiation: Total-body irradiation; Biological: Donor lymphocytes infusion (DLI); Drug: Cyclophosphamide; Procedure: Allogeneic hematopoietic stem cell transplantation (HSCT); Drug: Tacrolimus; Drug: Mycophenolate mofetil

Interventions

Total-body irradiation RADIATION

Undergo TBI

Also known as: TBI

Treatment (haploidentical allogeneic HSCT)

Donor lymphocytes infusion (DLI) BIOLOGICAL

Undergo DLI

Also known as: Allogeneic Lymphocytes, ALLOLYMPH

Treatment (haploidentical allogeneic HSCT)

Cyclophosphamide DRUG

Given IV

Also known as: Endoxan, Cytoxan, Neosar, Procytox, Revimmune, cytophosphane

Treatment (haploidentical allogeneic HSCT)

Allogeneic hematopoietic stem cell transplantation (HSCT) PROCEDURE

Undergo haploidentical allogeneic HSCT

Treatment (haploidentical allogeneic HSCT)

Tacrolimus DRUG

Given IV

Also known as: FK-506, fujimycin, Prograf, Advagraf, Protopic

Treatment (haploidentical allogeneic HSCT)

Mycophenolate mofetil DRUG

Given IV

Also known as: CellCept, Myfortic, MMF

Treatment (haploidentical allogeneic HSCT)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Any patient with a hematologic or oncologic diagnosis without morphological evidence of disease in which allogeneic HSCT is thought to be beneficial.
• Patients must have a related donor who is a two or more allele mismatch at the HLA-A; B; C; DR loci.
• Patients must have adequate organ function:
• LVEF (Left ventricular ejection fraction) of \>50%
• Diffusion Capacity for Carbon Monoxide (DLCO) \>50% of predicted corrected for hemoglobin
• Adequate liver function as defined by a serum bilirubin \<1.8, Aspartate Aminotransferase (AST) or alanine aminotransferase (ALT) \< 2.5X upper limit of normal
• Creatinine clearance of \> 60 ml/min
• Performance status \> 80% (TJU Karnofsky)
• Hematopoietic Comorbidity Index (HCT-CI) Score \< 5 Points
• Patients must be willing to use contraception if they have childbearing potential
• Able to give informed consent, or if decisionally impaired, have a legal next of kin or guardian that can give informed consent

You may not qualify if:

• Performance status \< 80 % (TJU Karnofsky)
• HCT-CI Score \> 5 Points
• Combination of Performance status of \< 80% (TJU Karnofsky) and an HCT-CI of 4 points or more.
• HIV positive
• Active involvement of the central nervous system with malignancy
• Psychiatric disorder that would preclude patients from signing an informed consent
• Pregnancy
• Patients with life expectancy of \< 6 months for reasons other than their underlying hematologic/oncologic disorder
• Patients who have received alemtuzumab within 8 weeks of the transplant admission, or who have recently received horse or rabbit anti-thymocyte globulin and have an ATG level of \> 2 ugm/ml
• Patients who cannot receive cyclophosphamide
• Patients with evidence of another malignancy, exclusive of a skin cancer that requires only local treatment, should not be enrolled on this protocol

Sponsors & Collaborators

• Sidney Kimmel Cancer Center at Thomas Jefferson University: lead

Study Sites (1)

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

Related Links

• Thomas Jefferson University Hospitals

MeSH Terms

Conditions

Neoplasms

Interventions

Whole-Body Irradiation; Cyclophosphamide; Tacrolimus; Mycophenolic Acid

Intervention Hierarchy (Ancestors)

Radiotherapy; Therapeutics; Investigative Techniques; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Macrolides; Lactones; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids

Results Point of Contact

• Title: Dr. Neal Flomenberg
• Organization: Sidney Kimmel Cancer Center at Thomas Jefferson University

dolores.gross@jefferson.edu

215 955-0182

Study Officials

• Dolores Grosso, DNP, CRNP

  Thomas Jefferson 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 4, 2013
• First Posted: June 6, 2013
• Study Start: May 17, 2013
• Primary Completion: December 12, 2013
• Study Completion: October 20, 2016
• Last Updated: May 16, 2025
• Results First Posted: January 9, 2018

Record last verified: 2025-05

Locations

US (1)