Human Lysozyme Goat Milk for the Prevention of Graft Versus Host Disease in Patients With Blood Cancer Undergoing a Donor Stem Cell Transplant

NCT ID: NCT04177004

Last Updated: 2025-12-15

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

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

53 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-04-30

Study Completion Date

2026-10-27

Brief Summary

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This phase I trial studies the side effects of human lysozyme goat milk in preventing graft versus host disease in patients with blood cancer undergoing a donor stem cell transplant. Sometimes the transplanted cells from a donor can cause an immune response against the body's own normal cells (call graft versus host disease). The goat milk in the study is from goats that have been genetically engineered to produce human lysozyme in the milk. Human lysozyme is a natural enzyme found in human milk and acts as an antimicrobial. Lysozyme is key to the digestive health of breast-fed human infants, since it helps the growth of beneficial gut bacteria and reduces the growth of bacteria that causes diarrhea and intestinal disease. Giving human lysozyme goat milk may reduce the rate of graft versus host disease in blood cancer patients undergoing a donor stem cell transplant.

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Detailed Description

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PRIMARY OBJECTIVES:

I. To evaluate the safety and feasibility of human lysozyme goat milk (hLZ) treatment by assessing:

Ia: Type, frequency, severity, attribution, time course and duration of adverse events, including diarrhea, bloodstream/intestinal infections.

Ib. Patients' ability to drink the specified volume (250 ml 3 x/day) of hLZ during the treatment period.

SECONDARY OBJECTIVES:

I. To compare the incidence and severity of adverse events (AE) among hLZ-treated and untreated patients, including diarrhea, bloodstream infections and intestinal infections.

II. To obtain preliminary estimates of gut microbiome diversity, as assessed by the Simpson Index, in hLZ-treated/untreated patients.

III. To compare gut microbiome diversity among hLZ-treated/untreated patients. IV. To obtain a preliminary estimate of the possible association between gut microbiome diversity and bloodstream infections.

V. To obtain a preliminary estimate of the possible association between gut microbiome diversity and acute graft versus host disease (GVHD) cumulative incidence, including time to onset.

VI. To characterize and compare GVHD inflammatory biomarkers (presence, level) among hLZ-treated and untreated patients.

VII. To characterize and compare urinary uindoxyl sulfate, tryptophan and kynurenine levels between hLZ-treated and untreated patients.

IX. To obtain a preliminary estimate of gut microbiome diversity and calorie intake.

X. To estimate overall survival (OS) cumulative incidence (CI) chronic GVHD of relapse/progression, and non-relapse mortality (NRM) at 100 days (excluding chronic GVHD), 6 months, 1 year and 2 years.

OUTLINE: Patients are randomized to 1 of 2 groups.

GROUP A:

CONDITIONING: Patients receive palifermin on days -10 to -8 and days 0 to 2, undergo fractionated total body irradiation (FTBI) on days -7 to -4, and receive cyclophosphamide on days -3 to -2 or etoposide on day -3 per City of Hope (COH) standard operating procedure (SOP) in the absence of disease progression or unacceptable toxicity.

HLZ: Patients receive human lysozyme goat milk orally (PO) three times daily (TID) on days -8 to 28 in the absence of disease progression or unacceptable toxicity.

TRANSPLANT: Patients undergo stem cell transplant on day 0.

GVHD PROPHYLAXIS: Beginning on day -2, patients receive tacrolimus and sirolimus daily per COH SOP in the absence of disease progression or unacceptable toxicity.

GROUP B:

CONDITIONING: Patients receive palifermin on days -10 to -8 and days 0 to 2 per COH SOP, undergo FTBI on days -7 to -4, and receive cyclophosphamide on days -3 to -2 or etoposide on day -3 per COH SOP in the absence of disease progression or unacceptable toxicity.

TRANSPLANT: Patients undergo stem cell transplant on day 0.

GVHD PROPHYLAXIS: Beginning on day -2, patients receive tacrolimus and sirolimus daily per COH SOP in the absence of disease progression or unacceptable toxicity.

After the completion of study treatment, patients are followed up for up to 30-100 days and then up to 2 years after transplant.

Conditions

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Allogeneic Hematopoietic Stem Cell Transplant Recipient Hematopoietic and Lymphoid Cell Neoplasm

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

SUPPORTIVE_CARE

Blinding Strategy

NONE

Study Groups

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Group A (conditioning, goat milk, transplant, prophylaxis)

CONDITIONING: Patients receive palifermin on days -10 to -8 and days 0 to 2, undergo FTBI on days -7 to -4, and receive cyclophosphamide on days -3 to -2 or etoposide on day -3 per COH SOP in the absence of disease progression or unacceptable toxicity.

HLZ: Patients receive human lysozyme goat milk PO TID on days -8 to 28 in the absence of disease progression or unacceptable toxicity.

TRANSPLANT: Patients undergo stem cell transplant on day 0.

GVHD PROPHYLAXIS: Beginning on day -2, patients receive tacrolimus and sirolimus daily per COH SOP in the absence of disease progression or unacceptable toxicity.

Group Type EXPERIMENTAL

Allogeneic Hematopoietic Stem Cell Transplantation

Intervention Type PROCEDURE

Undergo allo-HCT

Cyclophosphamide

Intervention Type DRUG

Given IV

Etoposide

Intervention Type DRUG

Given IV

Fractionated Stereotactic Radiation Therapy

Intervention Type RADIATION

Undergo FTBI

Goat Milk

Intervention Type DRUG

Given human lysozyme goat milk PO

Palifermin

Intervention Type BIOLOGICAL

Given IV

Sirolimus

Intervention Type DRUG

Given PO

Tacrolimus

Intervention Type DRUG

Given IV and PO

Group B (conditioning, transplant, prophylaxis)

CONDITIONING: Patients receive palifermin on days -10 to -8 and days 0 to 2 per COH SOP, undergo FTBI on days -7 to -4, and receive cyclophosphamide on days -3 to -2 or etoposide on day -3 per COH SOP in the absence of disease progression or unacceptable toxicity.

TRANSPLANT: Patients undergo stem cell transplant on day 0.

GVHD PROPHYLAXIS: Beginning on day -2, patients receive tacrolimus and sirolimus daily per COH SOP in the absence of disease progression or unacceptable toxicity.

Group Type ACTIVE_COMPARATOR

Allogeneic Hematopoietic Stem Cell Transplantation

Intervention Type PROCEDURE

Undergo allo-HCT

Cyclophosphamide

Intervention Type DRUG

Given IV

Etoposide

Intervention Type DRUG

Given IV

Fractionated Stereotactic Radiation Therapy

Intervention Type RADIATION

Undergo FTBI

Palifermin

Intervention Type BIOLOGICAL

Given IV

Sirolimus

Intervention Type DRUG

Given PO

Tacrolimus

Intervention Type DRUG

Given IV and PO

Interventions

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Allogeneic Hematopoietic Stem Cell Transplantation

Undergo allo-HCT

Intervention Type PROCEDURE

Cyclophosphamide

Given IV

Intervention Type DRUG

Etoposide

Given IV

Intervention Type DRUG

Fractionated Stereotactic Radiation Therapy

Undergo FTBI

Intervention Type RADIATION

Goat Milk

Given human lysozyme goat milk PO

Intervention Type DRUG

Palifermin

Given IV

Intervention Type BIOLOGICAL

Sirolimus

Given PO

Intervention Type DRUG

Tacrolimus

Given IV and PO

Intervention Type DRUG

Other Intervention Names

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Allogeneic Hematopoietic Cell Transplantation allogeneic stem cell transplantation HSC HSCT (-)-Cyclophosphamide 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate Carloxan Ciclofosfamida Ciclofosfamide Cicloxal Clafen Claphene CP monohydrate CTX CYCLO-cell Cycloblastin Cycloblastine Cyclophospham Cyclophosphamid monohydrate Cyclophosphamide Monohydrate Cyclophosphamidum Cyclophosphan Cyclophosphane Cyclophosphanum Cyclostin Cyclostine Cytophosphan Cytophosphane Cytoxan Fosfaseron Genoxal Genuxal Ledoxina Mitoxan Neosar Revimmune Syklofosfamid WR- 138719 Demethyl Epipodophyllotoxin Ethylidine Glucoside EPEG Lastet Toposar Vepesid VP 16 VP 16-213 VP-16 VP-16-213 VP16 Fractionated Stereotactic Radiotherapy Growth Factor, Recombinant Human Keratinocyte Kepivance Keratinocyte Growth Factor, Recombinant Human Recombinant Human Keratinocyte Growth Factor rhKGF rhu Keratinocyte Growth Factor AY 22989 RAPA Rapamune RAPAMYCIN SILA 9268A WY-090217 FK 506 Fujimycin Hecoria Prograf Protopic

Eligibility Criteria

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Inclusion Criteria

* Documented informed consent of the participant and/or legally authorized representative
* Willingness to be followed for the planned duration of the trial (2 years)
* All subjects must have the ability to understand and the willingness to sign a written informed consent
* Karnofsky performance status \>= 60 per COH SOP
* Patients must be undergoing allogeneic hematopoietic stem cell transplantation (alloHCT) for hematologic malignancies from matched related or matched unrelated donors with 8/8 (A, B, C, DRB 1) high resolution human leukocyte antigen (HLA) donor allele matching
* Patients must be receiving a fractionated total body radiation (FTBI) based- myeloablative conditioning regimen; (acceptable conditioning regimens include total body irradiation \[TBI\] + cyclophosphamide or TBI + etoposide)
* Ejection fraction measured by echocardiogram or multi gated acquisition scan (MUGA) \> 50%
* Diffusing capacity for carbon monoxide (DLCO) adjusted for hemoglobin or forced vital capacity (FVC) \> 50% predicted
* Total serum bilirubin \< 2 times upper limit of normal
* Alanine aminotransferase (ALT)/aspartate aminotransferase (AST) =\< 2.5 x the upper normal limit
* Alkaline phosphatase =\< 2.5 x the upper normal limit
* Measured creatinine clearance more than 60 mL/min
* Agreement by females and males of childbearing potential to use an effective method of birth control or abstain from heterosexual activity for the course of the study through 90 days after the last dose of protocol therapy

* Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for \> 1 year (women only)

Exclusion Criteria

* Failure of research participant to understand the basic elements of the protocol and/or the risks/benefits of participating in this pilot study. A legal guardian may substitute for the research participant
* Research participants receiving any other investigational agents
* Research participants with presence of other active malignancy within 2 years of study entry. Participants with history of prior malignancy treated with curative intent who achieved complete remission (CR) more than 2 years before study entry are eligible. This exclusion rule does not apply to non-melanoma skin tumors and in-situ cervical cancer
* Research participants having any uncontrolled illness including ongoing or active infection. Research participants with known active hepatitis B or C infection; research participants who are human immunodeficiency virus (HIV) seropositive based on testing performed within 4 weeks of enrollment; research participants with any signs or symptoms of active infection, positive blood cultures, or radiological evidence of infections
* Refusing to use contraception up to 90 days post-HCT
* Pregnant and/or breast feeding if a female recipient
* Lactose intolerance or intolerance to milk products
* In the opinion of the principal investigator (PI), the participant has a condition that will preclude them from complying with study treatment
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

City of Hope Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Karamjeet S Sandhu

Role: PRINCIPAL_INVESTIGATOR

City of Hope Medical Center

Locations

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City of Hope Medical Center

Duarte, California, United States

Site Status RECRUITING

Countries

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United States

Facility Contacts

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Karamjeet S. Sandhu

Role: primary

626-218-2405

Other Identifiers

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NCI-2019-06454

Identifier Type: REGISTRY

Identifier Source: secondary_id

19214

Identifier Type: OTHER

Identifier Source: secondary_id

19214

Identifier Type: -

Identifier Source: org_study_id

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