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Trials / Suspended

SuspendedNCT04177004

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

A Randomized Pilot Study of Human Lysozyme Goat Milk in Recipients of Standard Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation

Status
Suspended
Phase
Phase 1
Study type
Interventional
Enrollment
53 (estimated)
Sponsor
City of Hope Medical Center · Academic / Other
Sex
All
Age
12 Years
Healthy volunteers
Not accepted

Summary

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.

Detailed description

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

Interventions

TypeNameDescription
PROCEDUREAllogeneic Hematopoietic Stem Cell TransplantationUndergo allo-HCT
DRUGCyclophosphamideGiven IV
DRUGEtoposideGiven IV
RADIATIONFractionated Stereotactic Radiation TherapyUndergo FTBI
DRUGGoat MilkGiven human lysozyme goat milk PO
BIOLOGICALPaliferminGiven IV
DRUGSirolimusGiven PO
DRUGTacrolimusGiven IV and PO

Timeline

Start date
2021-04-30
Primary completion
2026-10-27
Completion
2026-10-27
First posted
2019-11-26
Last updated
2026-04-15

Locations

1 site across 1 country: United States

Regulatory

Source: ClinicalTrials.gov record NCT04177004. Inclusion in this directory is not an endorsement.