Donor Stem Cell Transplant Followed by Cyclophosphamide in Treating Patients With Hematological Diseases

NCT02248597 | Completed Phase 2 Results FDA Drug

• 4 other identifiers: IRB00029210NCI-2014-01898CCCWFU 97214P30CA012197
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

Brief Summary

This pilot clinical trial studies donor stem cell transplant followed by cyclophosphamide in treating patients with hematological diseases. Giving chemotherapy before a donor stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving cyclophosphamide after the transplant may stop this from happening.

Conditions

Graft Versus Host Disease; Hematopoietic/Lymphoid Cancer

Outcome Measures

Primary Outcomes (1)

• 12 Month Disease Free Survival Probability

  The percentage of patients who experience death or disease relapse by one year will be calculated and a corresponding 95% confidence interval will be constructed using the normal approximation for binomial proportions. The survival function will be estimated and plotted using the method of Kaplan and Meier.

  At 12 months

Secondary Outcomes (4)

• Rate of Acute GvHD

  12 months

• Overall Survival

  At 12 months

• Progression Free Survival

  At 12 months

• Relapse-free Mortality

  At 12 months

Study Arms (1)

Treatment (stem cell transplant with GVHD prophylaxis)

EXPERIMENTAL

PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV QD on days -6 to -2. Patients receiving myeloablative conditioning receive busulfan IV every 6 hours for 16 doses on days -7 to -4 and patients receiving reduced intensity conditioning receive busulfan IV every 6 hours for 8 doses on days -5 to -4. Patients also receive cyclophosphamide IV QD on days -3 and -2 TRANSPLANT: Patients undergo stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclophosphamide QD on days 3 and 4, tacrolimus on days 5-180, and mycophenolate mofetil on days 5-35. Allogeneic hematopoietic stem cell transplantation

Drug: fludarabine phosphate; Drug: busulfan; Drug: cyclophosphamide; Procedure: allogeneic hematopoietic stem cell transplantation; Drug: tacrolimus; Drug: mycophenolate mofetil

Interventions

fludarabine phosphate DRUG

Given IV

Also known as: 2-F-ara-AMP, Beneflur, Fludara

Treatment (stem cell transplant with GVHD prophylaxis)

busulfan DRUG

Given IV

Also known as: BSF, BU, Misulfan, Mitosan, Myeloleukon

Treatment (stem cell transplant with GVHD prophylaxis)

cyclophosphamide DRUG

Given IV

Also known as: CPM, CTX, Cytoxan, Endoxan, Endoxana

Treatment (stem cell transplant with GVHD prophylaxis)

allogeneic hematopoietic stem cell transplantation PROCEDURE

Undergo myeloablative or reduced intensity allogeneic stem cell transplant

Treatment (stem cell transplant with GVHD prophylaxis)

tacrolimus DRUG

Also known as: FK 506, Prograf

Treatment (stem cell transplant with GVHD prophylaxis)

mycophenolate mofetil DRUG

Also known as: Cellcept, MMF

Treatment (stem cell transplant with GVHD prophylaxis)

Eligibility Criteria

• Age: 18 Years - 69 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of a hematological malignancy requiring an allogeneic stem cell transplant consistent with the standard of care
• Remission of any acute hematologic malignancy or adequate disease control for chronic malignancies.
• Ages 18-69 years old.
• Available familial haploidentical (4 to 6 out of 8 HLA loci-matched) donor

You may not qualify if:

• Significant organ dysfunction defined as: LV EF \< 50% (evaluated by echocardiogram or MRI), DLCO or FEV1 \< 65% predicted, AST/ALT \> 2.5 x ULN, Bilirubin \> 1.5 x ULN, Serum creatinine \> 2mg/dL, dialysis, or prior renal transplant
• HIV positive (Recipients who are positive for hepatitis B (HBV), hepatitis C (HCV) or human T-cell lymphotropic virus (HTLV-I/II) are not excluded from participation)
• Positive pregnancy test for women of childbearing age.
• Major anticipated illness or organ failure incompatible with survival form transplant.
• Severe psychiatric illness or mental deficiency sufficiently severe as to make compliance with the transplant treatment unlikely and informed consent impossible.

Sponsors & Collaborators

• Wake Forest University Health Sciences: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

Comprehensive Cancer Center of Wake Forest University

Winston-Salem, North Carolina, 27157, United States

Location

MeSH Terms

Conditions

Graft vs Host Disease; Hematologic Neoplasms

Interventions

fludarabine phosphate; Busulfan; Cyclophosphamide; Tacrolimus; Mycophenolic Acid

Condition Hierarchy (Ancestors)

Immune System Diseases; Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

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; Macrolides; Lactones; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids

Results Point of Contact

• Title: Study Nurse
• Organization: Wake Forest Baptist Comprehensive Cancer Center

krike@wakehealth.edu

336-716-4761

Study Officials

• Dianna S. Howard

  Wake Forest University Health Sciences

  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: September 23, 2014
• First Posted: September 25, 2014
• Study Start: February 25, 2015
• Primary Completion: January 18, 2022
• Study Completion: January 18, 2023
• Last Updated: October 11, 2023
• Results First Posted: October 11, 2023

Record last verified: 2023-10

Locations

US (1)