Adult Hodgkin LymphomaPhase 1NCT02728700
Sirolimus and Mycophenolate Mofetil in Preventing GVHD in Patients With Hematologic Malignancies Undergoing HSCT
A Phase 1 study testing Allogeneic Hematopoietic Stem Cell Transplantation, Laboratory Biomarker Analysis and Mycophenolate Mofetil in people with Adult Hodgkin Lymphoma and Adult Myelodysplastic Syndrome, run by Stanford University at 1 site in United States.
Trial summary
This pilot phase I/II trial studies the side effects and how well sirolimus and mycophenolate mofetil work in preventing graft versus host disease (GvHD) in patients with hematologic malignancies undergoing hematopoietic stem cell transplant (HSCT). Read more about this study
- Who is it for?
- Ages 3 to 30 years
- Conditions the study is for
- Adult Hodgkin Lymphoma, Adult Myelodysplastic Syndrome, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- What is being tested?
- Allogeneic Hematopoietic Stem Cell Transplantation, Laboratory Biomarker Analysis and Mycophenolate Mofetil
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2016to Jul 2018
- 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 3 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects must have one of the following disease categories:
- Acute myeloid leukemia (AML) beyond 2nd remission or relapsed/refractory disease
- Acute lymphoblastic leukemia (ALL) beyond 2nd remission or relapsed/refractory disease
- Chronic myeloid leukemia (CML) beyond 2nd chronic phase or in blast crises
- Myelodysplastic syndrome (MDS)
- Myeloproliferative disorders including myeloid metaplasia and myelofibrosis
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- High risk non-Hodgkin's lymphoma (NHL) in first remission
- Relapsed or refractory NHL
- Hodgkin's lymphoma (HL) beyond first remission
- Performance status by Karnofsky of >= 70% or Lansky > 70% for patients < 16 years of age
- Human leukocyte antigen (HLA) mismatched related or unrelated donor identified 8/10 or 9/10
- Willingness to take oral medications during the transplantation period
- Willingness and ability to sign a written informed consent (assent if applicable)
Who cannot join
- Prior myeloablative allogeneic or autologous HSCT
- Human immunodeficiency virus (HIV) infection
- Pregnant or lactating females
- Evidence of uncontrolled active infection
- Down syndrome
- Serum creatinine (CR) < 1.5mg/dl or 24 hour CR clearance < 50 ml/min
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- Direct bilirubin > 2 x upper limit of normal (ULN)
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 x ULN
- Carbon monoxide diffusing capability test (DLCO) > 60% predicted and in children- room air oxygen saturation > 92%
- Left ventricular ejection fraction < 45% and in children-shortening fraction < 26%
- Fasting cholesterol > 300 mg/dl or triglycerides > 300 while on lipid lowering agents
- Patients who have received an investigational drug within 30 days of enrollment in study
- Patients with prior malignancies except basal cell carcinoma or treated carcinoma in-situ; cancer treated with curative intent > 5 years will be allowed; cancer treatment with curative intent =< 5 years will not be allowed
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University, School of Medicine
Palo Alto, California, 94304
About this study
Official title: Pilot Safety and Feasibility Trial of Mycophenolate and Sirolimus for Prevention of GVHD in Mismatched Unrelated and Related Donor Hematopoietic Stem Cell Transplantation for Hematologic Malignancies
This pilot phase I/II trial studies the side effects and how well sirolimus and mycophenolate mofetil work in preventing graft versus host disease (GvHD) in patients with hematologic malignancies undergoing hematopoietic stem cell transplant (HSCT). Biological therapies, such as sirolimus and mycophenolate mofetil, use substances made from living organisms that may stimulate or suppress the immune system in different ways and stop tumor cells from growing. Giving sirolimus and mycophenolate mofetil after hematopoietic stem cell transplant may be better in preventing graft-versus-host disease.
Read the detailed description
PRIMARY OBJECTIVES:
- Evaluate the safety and feasibility of administering sirolimus and mycophenolate mofetil (MMF) as prophylaxis of grade III-IV acute graft versus host disease (aGvHD) in patients undergoing mismatched unrelated and related donor hematopoietic stem cell transplant (HSCT).
OUTLINE:
Patients receive sirolimus orally (PO) starting on day -3, 3 times a week during hospitalization and then once a week for up to 6 months. Patients undergo HSCT on day 0. Patients also receive mycophenolate mofetil intravenously (IV) or PO three times a day (TID) on days 1-180. Treatment continues in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at days 30, 60, 100, 180, 270, and 365, and then yearly thereafter.
- Conditions
- Adult Hodgkin Lymphoma, Adult Myelodysplastic Syndrome, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Childhood Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Childhood Hodgkin Lymphoma, Childhood Myelodysplastic Syndrome
- Being tested
- Allogeneic Hematopoietic Stem Cell Transplantation, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Sirolimus
- Medical subject headings
- Hodgkin Disease, Anemia, Refractory, with Excess of Blasts, Blast Crisis, Leukemia, Myeloid, Chronic-Phase, Primary Myelofibrosis, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Lymphoma, Non-Hodgkin
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 stem cell transplantation, HSC, HSCT
Undergo HSCT
Laboratory Biomarker AnalysisOther
Correlative studies
Also called Cellcept, MMF
Given IV
SirolimusDrug
Also called AY 22989, RAPA, Rapamune, RAPAMYCIN
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Treatment (sirolimus, HSCT, MMF) | Experimental | Patients receive sirolimus PO starting on day -3, 3 times a week during hospitalization and then once a week for up to 6 months. Patients undergo HSCT on day 0. Patients also receive mycophenolate mofetil IV or PO TID on days 1-180. Treatment continues in the absence of disease progression or unacceptable toxicity. Receives: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Other: Laboratory Biomarker Analysis; Drug: Mycophenolate Mofetil; Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of acute GvHD grade 3 & 4, according to the Glucksberg staging criteria
Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum.
Measured: Up to 60 days post-transplant
Incidence of acute GvHD grade 3 & 4, according to the Glucksberg staging criteria
Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum.
Measured: Up to 100 days post-transplant
Secondary outcomes (5)
Incidence of thrombotic microangiopathy defined according to the bone marrow transplant clinical trials network toxicity committee
Defined as: red blood cell fragmentation and at least two schistocytes per high-power field on peripheral smear; concurrent increased serum lactate dehydrogenase measurement above institutional baseline; concurrent doubling of serum creatinine or 50% increase in creatinine clearance from baseline and/or neurological dysfunction without other explanations; and negative direct and indirect Coombs. Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum.
Measured: Up to 100 days
Incidence of venous-occlusive disease (VOD) using Modified Seattle Criteria
Severe VOD will be considered a dose limiting toxicity. Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum.
Measured: Up to 100 days
Severity of mucositis determined by National Cancer Institute Common Terminology Criteria for Adverse Events version 4.03
Grade III and IV will be considered dose limiting toxicities. Statistical analysis results will be reported using summary tables, figures, and data listings. Categorical variables will be summarized by numbers and percentages of subjects in corresponding categories.
Measured: Up to 100 days
Time to neutrophil engraftment defined as first of 3 consecutive days with the absolute neutrophil count is > 500/ul in the peripheral blood
Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum.
Measured: Baseline to up to 100 days
Time to platelet engraftment defined as the first day of a minimum of three consecutive measurements on different days when platelet count > 50,000/mm^3 and patient is transfusion-independent for a minimum of 7 days
Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum.
Measured: Baseline to up to 100 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2016 (estimated)
- Study startFeb 2016
- Primary completionApr 2017
- Study completionJul 2018
Why it stopped: Accrual factor
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Adult Hodgkin LymphomaStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe 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
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Rajni Agarwal-Hashmi, Principal investigator, Stanford Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02728700
- Sponsor's protocol ID
- IRB-34973
- Other IDs
- NCI-2016-00387, PEDSBMT295
- Status last verified
- Aug 2018
- First submitted
- 30 Mar 2016
- Last updated
- 12 Sep 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Safety and Feasibility Trial of Mycophenolate and Sirolimus for Prevention of GVHD in Mismatched Unrelated and Related Donor Hematopoietic Stem Cell Transplantation for Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT02728700
- Acronym / sponsor ID
- IRB-34973
- Conditions
- Adult Hodgkin Lymphoma, Adult Myelodysplastic Syndrome, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Childhood Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Childhood Hodgkin Lymphoma, Childhood Myelodysplastic Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Accrual factor
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- Feb 2016
- Primary completion
- Apr 2017 (actual)
- Study completion
- Jul 2018 (actual)
- First posted
- 5 Apr 2016 (estimated)
- 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 pilot phase I/II trial studies the side effects and how well sirolimus and mycophenolate mofetil work in preventing graft versus host disease (GvHD) in patients with hematologic malignancies undergoing hematopoietic stem cell transplant (HSCT). Biological therapies, such as sirolimus and mycophenolate mofetil, use substances made from living organisms that may stimulate or suppress the immune system in different ways and stop tumor cells from growing. Giving sirolimus and mycophenolate mofetil after hematopoietic stem cell transplant may be better in preventing graft-versus-host disease.
Detailed description
PRIMARY OBJECTIVES:
- Evaluate the safety and feasibility of administering sirolimus and mycophenolate mofetil (MMF) as prophylaxis of grade III-IV acute graft versus host disease (aGvHD) in patients undergoing mismatched unrelated and related donor hematopoietic stem cell transplant (HSCT).
OUTLINE:
Patients receive sirolimus orally (PO) starting on day -3, 3 times a week during hospitalization and then once a week for up to 6 months. Patients undergo HSCT on day 0. Patients also receive mycophenolate mofetil intravenously (IV) or PO three times a day (TID) on days 1-180. Treatment continues in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at days 30, 60, 100, 180, 270, and 365, and then yearly thereafter.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (sirolimus, HSCT, MMF) | Experimental | Procedure: Allogeneic Hematopoietic Stem Cell Transplantation, Other: Laboratory Biomarker Analysis, Drug: Mycophenolate Mofetil, Drug: SirolimusPatients receive sirolimus PO starting on day -3, 3 times a week during hospitalization and then once a week for up to 6 months. Patients undergo HSCT on day 0. Patients also receive mycophenolate mofetil IV or PO TID on days 1-180. Treatment continues in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 3 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- Subjects must have one of the following disease categories:
- Performance status by Karnofsky of >= 70% or Lansky > 70% for patients < 16 years of age
- Human leukocyte antigen (HLA) mismatched related or unrelated donor identified 8/10 or 9/10
- Willingness to take oral medications during the transplantation period
- Willingness and ability to sign a written informed consent (assent if applicable)
Key exclusion criteria (13 listed)
- Prior myeloablative allogeneic or autologous HSCT
- Human immunodeficiency virus (HIV) infection
- Pregnant or lactating females
- Evidence of uncontrolled active infection
- Down syndrome
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 stem cell transplantation, HSC, HSCTUndergo HSCT | Procedure or surgery | Treatment (sirolimus, HSCT, MMF) |
| Laboratory Biomarker AnalysisCorrelative studies | Other | Treatment (sirolimus, HSCT, MMF) |
| Mycophenolate MofetilAlso: Cellcept, MMFGiven IV | Drug | Treatment (sirolimus, HSCT, MMF) |
| SirolimusAlso: AY 22989, RAPA, Rapamune, RAPAMYCINGiven PO | Drug | Treatment (sirolimus, HSCT, MMF) |
Primary outcome
Incidence of acute GvHD grade 3 & 4, according to the Glucksberg staging criteria
Time frame: Up to 60 days post-transplant
Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum.
Incidence of acute GvHD grade 3 & 4, according to the Glucksberg staging criteria
Time frame: Up to 100 days post-transplant
Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum.
5 secondary outcomes
Incidence of thrombotic microangiopathy defined according to the bone marrow transplant clinical trials network toxicity committee
Time frame: Up to 100 days
Incidence of venous-occlusive disease (VOD) using Modified Seattle Criteria
Time frame: Up to 100 days
Severity of mucositis determined by National Cancer Institute Common Terminology Criteria for Adverse Events version 4.03
Time frame: Up to 100 days
Time to neutrophil engraftment defined as first of 3 consecutive days with the absolute neutrophil count is > 500/ul in the peripheral blood
Time frame: Baseline to up to 100 days
Time to platelet engraftment defined as the first day of a minimum of three consecutive measurements on different days when platelet count > 50,000/mm^3 and patient is transfusion-independent for a minimum of 7 days
Time frame: Baseline to up to 100 days
Results and safety
The study was stopped early (reason given: Accrual factor). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Sep 2018.
- Individual participant dataSharing planned: No
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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.
Rajni Agarwal-Hashmi principal investigator
Stanford Cancer Institute
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, Laboratory Biomarker Analysis and Mycophenolate Mofetil in people with Adult Hodgkin Lymphoma and Adult Myelodysplastic Syndrome, run by Stanford University 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 13 inclusion and 13 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: stopped early, as of the sponsor's last update on 12 Sep 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 stopped early (reason given: Accrual factor). Results are not available in the source record.
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?
Incidence of acute GvHD grade 3 & 4, according to the Glucksberg staging criteria (time frame: Up to 60 days post-transplant). The study lists 2 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 12 Sep 2018.