Hematopoietic and Lymphoid Cell NeoplasmPhase 1NCT03531281
Human Lysozyme Goat Milk in Treating Patients With Blood Cancer Undergoing Donor Stem Cell Transplant
A Phase 1 study testing Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide and Etoposide in people with Hematopoietic and Lymphoid Cell Neoplasm and Hematopoietic Cell Transplantation Recipient, run by City of Hope Medical Center at 1 site in United States.
Trial summary
This randomized pilot phase I trial studies the side effects of human lysozyme goat milk in treating patients with blood cancer undergoing a donor stem cell transplant. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Read more about this study
- Who is it for?
- Ages 12 to 60 years
- Conditions the study is for
- Hematopoietic and Lymphoid Cell Neoplasm, Hematopoietic Cell Transplantation Recipient
- What is being tested?
- Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide and Etoposide
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Dec 2018to 30 Dec 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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)
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- Ejection fraction measured by echocardiogram or multi gated acquisition scan (MUGA) > 45%
- 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; the updated Schwartz formula should be used for pediatric patients (>= 5 to 12 years old)
- 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)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
City of Hope Medical Center
Duarte, California, 91010
About this study
Official title: A Randomized Pilot Study of Human Lysozyme Goat Milk in Recipients of Standard Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation
This randomized pilot phase I trial studies the side effects of human lysozyme goat milk in treating patients with blood cancer undergoing a donor stem cell transplant. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving human lysozyme goat milk to patients undergoing a donor stem cell transplant may stop this from happening.
Read the detailed description
PRIMARY OBJECTIVES:
- To evaluate the safety and feasibility of human lysozyme goat milk (hLZ) treatment by assessing type, frequency, severity, attribution, time course and duration of adverse events, including diarrhea, bloodstream/intestinal infections.
II. To evaluate the safety and feasibility of hLZ treatment by assessing patient compliance, the patients' ability to drink the specified volume (250 ml 3 x/day) of hLZ during the treatment period.
SECONDARY OBJECTIVES:
- 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.
- 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 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 arms.
ARM I:
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 infusion 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.
ARM II:
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 infusion 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 completion of study treatment, patients are followed up for up to 2 years.
- Being tested
- Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide, Etoposide, Goat Milk, Laboratory Biomarker Analysis
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery
Also called Allogeneic Hematopoietic Cell Transplantation, Allogeneic Stem Cell Transplantation, HSC, HSCT
Undergo allo-HCT
CyclophosphamideDrug
Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida
Given IV
EtoposideDrug
Also called Demethyl Epipodophyllotoxin Ethylidine Glucoside, EPEG, Lastet, Toposar
Given IV
Goat MilkDrug
Given human lysozyme goat milk PO
Laboratory Biomarker AnalysisOther
Correlative studies
PaliferminBiological
Also called Growth Factor, Recombinant Human Keratinocyte, Kepivance, Keratinocyte Growth Factor, Recombinant Human, Recombinant Human Keratinocyte Growth Factor
Given IV
SirolimusDrug
Also called AY 22989, RAPA, Rapamune, Rapamycin
Given PO
TacrolimusDrug
Also called FK 506, Fujimycin, Hecoria, Prograf
Given IV and PO
Total-Body IrradiationRadiation
Also called Total Body Irradiation, Whole-Body Irradiation
Undergo FTBI
| Study group | Role | What they receive |
|---|---|---|
| Arm I (goat milk, transplant conditioning/prophylaxis) | Experimental | 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 infusion 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. Receives: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Drug: Cyclophosphamide; Drug: Etoposide; Drug: Goat Milk; Other: Laboratory Biomarker Analysis; Biological: Palifermin; Drug: Sirolimus; Drug: Tacrolimus; Radiation: Total-Body Irradiation |
| Arm II (transplant conditioning/prophylaxis) | Active comparator | 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 infusion 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. Receives: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Drug: Cyclophosphamide; Drug: Etoposide; Other: Laboratory Biomarker Analysis; Biological: Palifermin; Drug: Sirolimus; Drug: Tacrolimus; Radiation: Total-Body Irradiation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility of drinking human lysozyme goat milk (hLZ)
Feasibility of drinking hLZ will be evaluated by assessment of patients' ability to drink the specified volume (250 ml 3 x per day) of hLZ during the safety lead-in phase.
Measured: Up to +28 days post-transplant or date of discharge
Unacceptable toxicity
The modified Bearman Scale will be used to define unacceptable toxicity events. Unacceptable toxicity in a given patient is defined as either of the following that are considered at least possibly related to drinking hLZ milk: GI toxicity grade III or IV per Bearman scale or inability to consume hLZ milk for >7 days.
Measured: Up to 28 days post-transplant or date of discharge
Adverse events
Incidence and severity of adverse events will be reported according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version (v)4.03. Observed toxicities will be summarized in terms of type (organ affected or laboratory determination), severity, time of onset, duration, probable association with the study treatment and reversibility or outcome.
Measured: Up to 100 days post-transplant
Volume of hLZ consumed
Tolerability is defined as the ability to consume >= 150 ml/day over the treatment period.
Measured: Up to 28 days post-transplant or date of discharge
Secondary outcomes (15)
Cumulative incidence (CI) of chronic GVHD
Chronic graft versus host disease is scored according to NIH Consensus Staging. The first day of chronic GvHD onset will be used to calculate CI incidence curves, with relapse/death prior to onset considered competing events. CI of chronic GVHD will be estimated using the method described by Gooley et al (1999).
Measured: At 6 months
Cumulative incidence (CI) of chronic GVHD
Chronic graft versus host disease is scored according to NIH Consensus Staging. The first day of chronic GvHD onset will be used to calculate CI incidence curves, with relapse/death prior to onset considered competing events. CI of chronic GVHD will be estimated using the method described by Gooley et al (1999).
Measured: At 1 year
Cumulative incidence (CI) of chronic GVHD
Chronic graft versus host disease is scored according to NIH Consensus Staging. The first day of chronic GvHD onset will be used to calculate CI incidence curves, with relapse/death prior to onset considered competing events. CI of chronic GVHD will be estimated using the method described by Gooley et al (1999).
Measured: At 2 years
CI of non-relapse mortality (NRM)
Non-relapse mortality (NRM) is defined as death occurring in a patient from causes other than relapse. NRM is measured from start of treatment until non-disease related death, or last follow-up, whichever comes first. The cumulative incidence of NRM will be calculated reflecting relapse as a competing risk. CI of NRM will be estimated using the method described by Gooley et al (1999).
Measured: At 100 days
CI of NRM
Non-relapse mortality (NRM) is defined as death occurring in a patient from causes other than relapse. NRM is measured from start of treatment until non-disease related death, or last follow-up, whichever comes first. The cumulative incidence of NRM will be calculated reflecting relapse as a competing risk. CI of NRM will be estimated using the method described by Gooley et al (1999).
Measured: At 1 year
CI of NRM
Non-relapse mortality (NRM) is defined as death occurring in a patient from causes other than relapse. NRM is measured from start of treatment until non-disease related death, or last follow-up, whichever comes first. The cumulative incidence of NRM will be calculated reflecting relapse as a competing risk. CI of NRM will be estimated using the method described by Gooley et al (1999).
Measured: At 2 years
CI of relapse/progression
The event is relapse/progression. Time to this event is measured from start of treatment. Death without relapse/progression is considered a competing risk. Surviving patients with no history of relapse are censored at time of last follow-up. CI of relapse/progression will be estimated using the method described by Gooley et al (1999).
Measured: At 100 days
CI of relapse/progression
The event is relapse/progression. Time to this event is measured from start of treatment. Death without relapse/progression is considered a competing risk. Surviving patients with no history of relapse are censored at time of last follow-up. CI of relapse/progression will be estimated using the method described by Gooley et al (1999).
Measured: At 1 year
CI of relapse/progression
The event is relapse/progression. Time to this event is measured from start of treatment. Death without relapse/progression is considered a competing risk. Surviving patients with no history of relapse are censored at time of last follow-up. CI of relapse/progression will be estimated using the method described by Gooley et al (1999).
Measured: At 2 years
Gut microbiome diversity
Gut microbiome diversity will be assessed by the Simpson Index and compared between hLZ-treated/untreated patients. Association between treatment and gut microbial diversity will be assessed by Fisher's Exact test.
Measured: Day - 8 +/- 3, Day 0, Day +7, Day +14, Day +21, Day +28
CI of acute graft versus host disease (aGVHD)
Acute graft versus host disease will be graded according to the Consensus Grading. The first day of acute GvHD onset at a certain grade will be used to calculate cumulative incidence curves for that GvHD grade; relapse/death prior to onset will be considered competing events. CI of aGVHD will be estimated using the method described by Gooley et al (1999). Association between gut microbial diversity (inverse Simpson index: high [>4], intermediate [2-4], and low [<2]) or treatment and CI of aGVHD will be assessed by Gray's test.
Measured: At 100 days
Incidence of bloodstream infections
Incidence of bloodstream infections and infectious enterocolitis will be evaluated and a preliminary estimate of the association between gut microbiome diversity and bloodstream infections will be obtained. Association between gut microbial diversity (inverse Simpson index: high [>4], intermediate [2-4], and low [<2]) or treatment and CI of bloodstream infections will be assessed by Gray's test.
Measured: Up to 100 days
Overall survival (OS)
Patients are considered a failure for this endpoint if they die, regardless of cause. The time to this event is the time from start of treatment until death, or last follow-up, whichever comes first. OS will be estimated using the product-limit method of Kaplan and Meier.
Measured: At 100 days
Overall survival (OS)
Patients are considered a failure for this endpoint if they die, regardless of cause. The time to this event is the time from start of treatment until death, or last follow-up, whichever comes first. OS will be estimated using the product-limit method of Kaplan and Meier.
Measured: At 1 year
Overall survival (OS)
Patients are considered a failure for this endpoint if they die, regardless of cause. The time to this event is the time from start of treatment until death, or last follow-up, whichever comes first. OS will be estimated using the product-limit method of Kaplan and Meier.
Measured: At 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2018
- Study start30 Dec 2018 (estimated)
- Primary completion30 Dec 2022 (estimated)
- Study completion30 Dec 2022 (estimated)
Why it stopped: Administratively withdrawn
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic and Lymphoid Cell NeoplasmStudies of the same condition, open and under way.
- More trials run by City of Hope Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- City of Hope Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Karamjeet Sandhu, Principal investigator, City of Hope Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03531281
- Sponsor's protocol ID
- 17399
- Other IDs
- NCI-2018-00053, 17399
- Status last verified
- Dec 2018
- First submitted
- 26 Jan 2018
- Last updated
- 7 Dec 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Pilot Study of Human Lysozyme Goat Milk in Recipients of Standard Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT03531281
- Acronym / sponsor ID
- 17399
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Administratively withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- City of Hope Medical CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 30 Dec 2018 (estimated)
- Primary completion
- 30 Dec 2022 (estimated)
- Study completion
- 30 Dec 2022 (estimated)
- First posted
- 21 May 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This randomized pilot phase I trial studies the side effects of human lysozyme goat milk in treating patients with blood cancer undergoing a donor stem cell transplant. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving human lysozyme goat milk to patients undergoing a donor stem cell transplant may stop this from happening.
Detailed description
PRIMARY OBJECTIVES:
- To evaluate the safety and feasibility of human lysozyme goat milk (hLZ) treatment by assessing type, frequency, severity, attribution, time course and duration of adverse events, including diarrhea, bloodstream/intestinal infections.
II. To evaluate the safety and feasibility of hLZ treatment by assessing patient compliance, the patients' ability to drink the specified volume (250 ml 3 x/day) of hLZ during the treatment period.
SECONDARY OBJECTIVES:
- 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.
- 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 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 arms.
ARM I:
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 infusion 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.
ARM II:
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 infusion 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 completion of study treatment, patients are followed up for up to 2 years.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I (goat milk, transplant conditioning/prophylaxis) | Experimental | Procedure: Allogeneic Hematopoietic Stem Cell Transplantation, Drug: Cyclophosphamide, Drug: Etoposide, Drug: Goat Milk, Other: Laboratory Biomarker Analysis, Biological: Palifermin, Drug: Sirolimus, Drug: Tacrolimus, Radiation: Total-Body IrradiationCONDITIONING: 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 infusion 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. |
| Arm II (transplant conditioning/prophylaxis) | Active comparator | Procedure: Allogeneic Hematopoietic Stem Cell Transplantation, Drug: Cyclophosphamide, Drug: Etoposide, Other: Laboratory Biomarker Analysis, Biological: Palifermin, Drug: Sirolimus, Drug: Tacrolimus, Radiation: Total-Body IrradiationCONDITIONING: 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 infusion 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. |
Who may qualify
- Ages
- 12 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (14 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Allogeneic Hematopoietic Stem Cell TransplantationAlso: Allogeneic Hematopoietic Cell Transplantation, Allogeneic Stem Cell Transplantation, HSC, HSCTUndergo allo-HCT | Procedure or surgery | Arm I (goat milk, transplant conditioning/prophylaxis), Arm II (transplant conditioning/prophylaxis) |
| CyclophosphamideAlso: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, CiclofosfamidaGiven IV | Drug | Arm I (goat milk, transplant conditioning/prophylaxis), Arm II (transplant conditioning/prophylaxis) |
| EtoposideAlso: Demethyl Epipodophyllotoxin Ethylidine Glucoside, EPEG, Lastet, ToposarGiven IV | Drug | Arm I (goat milk, transplant conditioning/prophylaxis), Arm II (transplant conditioning/prophylaxis) |
| Goat MilkGiven human lysozyme goat milk PO | Drug | Arm I (goat milk, transplant conditioning/prophylaxis) |
| Laboratory Biomarker AnalysisCorrelative studies | Other | Arm I (goat milk, transplant conditioning/prophylaxis), Arm II (transplant conditioning/prophylaxis) |
| PaliferminAlso: Growth Factor, Recombinant Human Keratinocyte, Kepivance, Keratinocyte Growth Factor, Recombinant Human, Recombinant Human Keratinocyte Growth FactorGiven IV | Biological | Arm I (goat milk, transplant conditioning/prophylaxis), Arm II (transplant conditioning/prophylaxis) |
| SirolimusAlso: AY 22989, RAPA, Rapamune, RapamycinGiven PO | Drug | Arm I (goat milk, transplant conditioning/prophylaxis), Arm II (transplant conditioning/prophylaxis) |
| TacrolimusAlso: FK 506, Fujimycin, Hecoria, PrografGiven IV and PO | Drug | Arm I (goat milk, transplant conditioning/prophylaxis), Arm II (transplant conditioning/prophylaxis) |
| Total-Body IrradiationAlso: Total Body Irradiation, Whole-Body IrradiationUndergo FTBI | Radiation | Arm I (goat milk, transplant conditioning/prophylaxis), Arm II (transplant conditioning/prophylaxis) |
Primary outcome
Feasibility of drinking human lysozyme goat milk (hLZ)
Time frame: Up to +28 days post-transplant or date of discharge
Feasibility of drinking hLZ will be evaluated by assessment of patients' ability to drink the specified volume (250 ml 3 x per day) of hLZ during the safety lead-in phase.
Unacceptable toxicity
Time frame: Up to 28 days post-transplant or date of discharge
The modified Bearman Scale will be used to define unacceptable toxicity events. Unacceptable toxicity in a given patient is defined as either of the following that are considered at least possibly related to drinking hLZ milk: GI toxicity grade III or IV per Bearman scale or inability to consume hLZ milk for >7 days.
Adverse events
Time frame: Up to 100 days post-transplant
Incidence and severity of adverse events will be reported according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version (v)4.03. Observed toxicities will be summarized in terms of type (organ affected or laboratory determination), severity, time of onset, duration, probable association with the study treatment and reversibility or outcome.
Volume of hLZ consumed
Time frame: Up to 28 days post-transplant or date of discharge
Tolerability is defined as the ability to consume >= 150 ml/day over the treatment period.
15 secondary outcomes
Cumulative incidence (CI) of chronic GVHD
Time frame: At 6 months
Cumulative incidence (CI) of chronic GVHD
Time frame: At 1 year
Cumulative incidence (CI) of chronic GVHD
Time frame: At 2 years
CI of non-relapse mortality (NRM)
Time frame: At 100 days
CI of NRM
Time frame: At 1 year
CI of NRM
Time frame: At 2 years
CI of relapse/progression
Time frame: At 100 days
CI of relapse/progression
Time frame: At 1 year
CI of relapse/progression
Time frame: At 2 years
Gut microbiome diversity
Time frame: Day - 8 +/- 3, Day 0, Day +7, Day +14, Day +21, Day +28
CI of acute graft versus host disease (aGVHD)
Time frame: At 100 days
Incidence of bloodstream infections
Time frame: Up to 100 days
Overall survival (OS)
Time frame: At 100 days
Overall survival (OS)
Time frame: At 1 year
Overall survival (OS)
Time frame: At 2 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Dec 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Karamjeet Sandhu principal investigator
City of Hope Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide and Etoposide in people with Hematopoietic and Lymphoid Cell Neoplasm and Hematopoietic Cell Transplantation Recipient, run by City of Hope Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 7 Dec 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Feasibility of drinking human lysozyme goat milk (hLZ) (time frame: Up to +28 days post-transplant or date of discharge). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Dec 2018.