LeukemiaPhase 2NCT02065154
Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis
A Phase 2 study testing Cyclophosphamide in people with Leukemia and Lymphoma, run by University of Alabama at Birmingham at 1 site in United States.
Trial summary
The main purpose of this study is to assess the effects of cyclophosphamide (cytoxan) in the post transplant setting to prevent onset of acute graft-versus-host disease (GVHD). The primary objective is to determine the incidence of grade II-IV acute GVHD following Allogeneic (allo) Hematopoeitic Cell Transplant (HCT)… Read more about this study
- Who is it for?
- Ages 19 to 65 years
- Conditions the study is for
- Leukemia, Lymphoma, Myelodysplastic Syndrome
- What is being tested?
- Cyclophosphamide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Aug 2013to Apr 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 19 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Disease Criteria: patients must meet diagnostic criteria of acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myeloid leukemia (CML), chronic lymphocytic leukemia (CLL), non-Hodgkin lymphoma (NHL), Hodgkin lymphoma (HL), myelodysplastic syndrome (MDS), myelofibrosis, or severe aplastic anemia. Patients will be allowed on study if they are deemed eligible for allo HCT regardless of remission status.
- Age Criteria: 19 to 65 years in age.
- Organ Function Criteria: All organ function testing should be done within 28 days of study registration.
- Cardiac: Left ventricular ejection fraction (LVEF) ≥ 50% by MUGA (Multi Gated Acquisition) scan or echocardiogram.
- Pulmonary: FEV1 (Forced expiratory volume in 1 second) and FVC (Forced vital capacity) ≥ 50% predicted, DLCO (diffusing capacity of the lung for carbon monoxide) (corrected for hemoglobin) ≥ 50% of predicted.
- Renal: The estimated creatinine clearance (CrCl) must be equal or greater than 60 mL/min/1.73 m2 as calculated by the Cockcroft-Gault Formula:
Show all 14Show fewer
- CrCl=(140-age) x weight(kg) x 0.85 (if female)/72 x serum creatinine (mg/dL)
- Hepatic:
- Serum bilirubin 1.5 upper limit of normal (ULN)
- Aspartate transaminase (AST)/alanine transaminase (ALT) 2.5 ULN
- Alkaline phosphatase 2.5 ULN
- Performance status: Karnofsky ≥ 70%.,
- Patient must be informed of the investigational nature of this study in accordance with institutional and federal guidelines and have the ability to provide written informed consent prior to initiation of any study-related procedures, and ability, in the opinion of the principal investigator, to comply with all the requirements of the study.
- Patient has a suitable and willing HLA-8/8 matched or 6/8 mismatched (at one allele) unrelated donor identified.
Who cannot join
- Non-compliant to medications.
- No appropriate caregivers identified.
- HIV1 (Human Immunodeficiency Virus-1) or HIV2 positive
- Uncontrolled medical or psychiatric disorders.
- Uncontrolled infections, defined as positive blood cultures within 72 hours of study entry, or evidence of progressive infection by imaging studies such as chest CT scan within 14 days of registration.
- Active central nervous system (CNS) leukemia.
- Preceding allogeneic HSCT.
- Pregnancy or Breastfeeding.
Trial locations and contacts
1 site in 1 country.
United States1 site
UAB Bone Marrow Transplantation and Cellular Therapy Program
Birmingham, Alabama, 35249
About this study
Official title: Phase II Clinical Trial of the Use of Post-Transplant Cyclophosphamide for Graft Versus Host Disease (GvHD) Prophylaxis Following Matched Unrelated Donor (MUD) and Mismatched Unrelated Donor (MMUD)Hematopoietic Stem Cell Transplant (HSCT)
The main purpose of this study is to assess the effects of cyclophosphamide (cytoxan) in the post transplant setting to prevent onset of acute graft-versus-host disease (GVHD). The primary objective is to determine the incidence of grade II-IV acute GVHD following Allogeneic (allo) Hematopoeitic Cell Transplant (HCT) using post-transplant cyclophosphamide (cytoxan) for patients with human leukocyte antigen (HLA) matched unrelated (MUD) and mismatched unrelated (MMUD) donors. Other objectives for this study will be the determination of disease-free survival (DFS) and overall survival (OS) following allo HCT and assess the safety of post-transplant cyclophosphamide (cytoxan) for MUD and MMUD transplantation. Disease recurrence and time to recurrence in patients receiving post-transplant cyclophosphamide compared to historical control without post-transplant cyclophosphamide (cytoxan) will also be evaluated. Other objectives will be to determine the time of onset, severity, responsiveness to treatment, organs involved of acute and chronic GVHD as well as observation of Immune Reconstitution over time.
Read the detailed description
he main purpose of this study is to assess the effects of cyclophosphamide (cytoxan) in the post transplant setting to prevent onset of acute graft-versus-host disease (GVHD). The primary objective is to determine the incidence of grade II-IV acute GVHD following Allogeneic (allo) Hematopoeitic Cell Transplant (HCT) using post-transplant cyclophosphamide (cytoxan) for patients with human leukocyte antigen (HLA) matched unrelated (MUD) and mismatched unrelated (MMUD) donors. Other objectives for this study will be the determination of disease-free survival (DFS) and overall survival (OS) following allo HCT and assess the safety of post-transplant cyclophosphamide (cytoxan) for MUD and MMUD transplantation. Disease recurrence and time to recurrence in patients receiving post-transplant cyclophosphamide compared to historical control without post-transplant cyclophosphamide (cytoxan) will also be evaluated. Other objectives will be to determine the time of onset, severity, responsiveness to treatment, organs involved of acute and chronic GVHD as well as observation of Immune Reconstitution over time.
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndrome, Myelofibrosis, Severe Aplastic Anemia, Allogeneic Transplant
- Being tested
- Cyclophosphamide
- Keywords
- acute myeloid leukemia, AML, acute lymphoblastic leukemia, ALL, chronic myeloid leukemia, CML, chronic lymphocytic leukemia, CLL, non-Hodgkin lymphoma, NHL, Hodgkin lymphoma, HL, myelodysplastic syndrome, MDS, myelofibrosis, severe aplastic anemia
- Medical subject headings
- Leukemia, Lymphoma, Myelodysplastic Syndromes, Primary Myelofibrosis, Anemia, Aplastic, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
Also called Cytoxan
| Study group | Role | What they receive |
|---|---|---|
| Cyclophosphamide (Cytoxan) | Experimental | Cyclophosphamide (Cytoxan) Receives: Drug: Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Grade II-IV Acute GVHD
To calculate the percentage of patients developing graft versus host disease, grade II-IV, in the first 100 days after transplant
Measured: Till 100 days post transplant
Secondary outcomes (4)
Overall Survival
What percentage of participants were alive at a certain time point after transplant
Measured: 2 Year Post Transplant
Disease-free Survival
What percentage of participants did not have relapse of disease after transplant
Measured: 1 Year Post-transplant
Regimen Related Toxicity
Number of toxicities experienced by the patients in the study
Measured: 100 Days Post Transplant
Relapse Rate
What percentage of participants relapsed
Measured: 2 years post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2014 (estimated)
- Study start27 Aug 2013
- Primary completionApr 2022
- Study completionApr 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by University of Alabama at BirminghamThe 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 2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Omer Jamy, University of Alabama at Birmingham
- Investigators
- Racquel D Innis-Shelton, MD, Principal investigator, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02065154
- Sponsor's protocol ID
- UAB 1286
- Status last verified
- Sep 2022
- First submitted
- 5 Nov 2013
- Last updated
- 30 Sep 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Jamy O, Innis-Shelton R, Bal S, Paluri R, Salzman D, Di Stasi A, Costa L, Meredith R, Lamb L, Minagawa K, Mineishi S, Saad A. Phase II clinical trial of one dose of post-transplant cyclophosphamide for graft versus host disease prevention following myeloablative, peripheral blood stem cell, matched-unrelated donor transplantation. Am J Hematol. 2021 Oct 1;96(10):E396-E398. doi: 10.1002/ajh.26296. Epub 2021 Jul 31. No abstract available.
PubMed 34288026
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Clinical Trial of the Use of Post-Transplant Cyclophosphamide for Graft Versus Host Disease (GvHD) Prophylaxis Following Matched Unrelated Donor (MUD) and Mismatched Unrelated Donor (MMUD)Hematopoietic Stem Cell Transplant (HSCT)
- ClinicalTrials.gov ID
- NCT02065154
- Acronym / sponsor ID
- UAB 1286
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndrome, Myelofibrosis, Severe Aplastic Anemia, Allogeneic Transplant
- Keywords
- acute myeloid leukemia, AML, acute lymphoblastic leukemia, ALL, chronic myeloid leukemia, CML, chronic lymphocytic leukemia, CLL
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Study start
- 27 Aug 2013 (actual)
- Primary completion
- Apr 2022 (actual)
- Study completion
- Apr 2022 (actual)
- First posted
- 17 Feb 2014 (estimated)
- Results
- Posted 15 Jun 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main purpose of this study is to assess the effects of cyclophosphamide (cytoxan) in the post transplant setting to prevent onset of acute graft-versus-host disease (GVHD). The primary objective is to determine the incidence of grade II-IV acute GVHD following Allogeneic (allo) Hematopoeitic Cell Transplant (HCT) using post-transplant cyclophosphamide (cytoxan) for patients with human leukocyte antigen (HLA) matched unrelated (MUD) and mismatched unrelated (MMUD) donors. Other objectives for this study will be the determination of disease-free survival (DFS) and overall survival (OS) following allo HCT and assess the safety of post-transplant cyclophosphamide (cytoxan) for MUD and MMUD transplantation. Disease recurrence and time to recurrence in patients receiving post-transplant cyclophosphamide compared to historical control without post-transplant cyclophosphamide (cytoxan) will also be evaluated. Other objectives will be to determine the time of onset, severity, responsiveness to treatment, organs involved of acute and chronic GVHD as well as observation of Immune Reconstitution over time.
Detailed description
he main purpose of this study is to assess the effects of cyclophosphamide (cytoxan) in the post transplant setting to prevent onset of acute graft-versus-host disease (GVHD). The primary objective is to determine the incidence of grade II-IV acute GVHD following Allogeneic (allo) Hematopoeitic Cell Transplant (HCT) using post-transplant cyclophosphamide (cytoxan) for patients with human leukocyte antigen (HLA) matched unrelated (MUD) and mismatched unrelated (MMUD) donors. Other objectives for this study will be the determination of disease-free survival (DFS) and overall survival (OS) following allo HCT and assess the safety of post-transplant cyclophosphamide (cytoxan) for MUD and MMUD transplantation. Disease recurrence and time to recurrence in patients receiving post-transplant cyclophosphamide compared to historical control without post-transplant cyclophosphamide (cytoxan) will also be evaluated. Other objectives will be to determine the time of onset, severity, responsiveness to treatment, organs involved of acute and chronic GVHD as well as observation of Immune Reconstitution over time.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cyclophosphamide (Cytoxan) | Experimental | Drug: CyclophosphamideCyclophosphamide (Cytoxan) |
Who may qualify
- Ages
- 19 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Disease Criteria: patients must meet diagnostic criteria of acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myeloid leukemia (CML), chronic lymphocytic leukemia (CLL), non-Hodgkin lymphoma (NHL), Hodgkin lymphoma (HL), myelodysplastic syndrome (MDS), myelofibrosis, or severe aplastic anemia. Patients will be allowed on study if they are deemed eligible for allo HCT regardless of remission status.
- Age Criteria: 19 to 65 years in age.
- Organ Function Criteria: All organ function testing should be done within 28 days of study registration.
- Cardiac: Left ventricular ejection fraction (LVEF) ≥ 50% by MUGA (Multi Gated Acquisition) scan or echocardiogram.
- Pulmonary: FEV1 (Forced expiratory volume in 1 second) and FVC (Forced vital capacity) ≥ 50% predicted, DLCO (diffusing capacity of the lung for carbon monoxide) (corrected for hemoglobin) ≥ 50% of predicted.
Key exclusion criteria (8 listed)
- Non-compliant to medications.
- No appropriate caregivers identified.
- HIV1 (Human Immunodeficiency Virus-1) or HIV2 positive
- Uncontrolled medical or psychiatric disorders.
- Uncontrolled infections, defined as positive blood cultures within 72 hours of study entry, or evidence of progressive infection by imaging studies such as chest CT scan within 14 days of registration.
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 |
|---|---|---|
| CyclophosphamideAlso: Cytoxan | Drug | Cyclophosphamide (Cytoxan) |
Primary outcome
Grade II-IV Acute GVHD
Time frame: Till 100 days post transplant
To calculate the percentage of patients developing graft versus host disease, grade II-IV, in the first 100 days after transplant
4 secondary outcomes
Overall Survival
Time frame: 2 Year Post Transplant
Disease-free Survival
Time frame: 1 Year Post-transplant
Regimen Related Toxicity
Time frame: 100 Days Post Transplant
Relapse Rate
Time frame: 2 years post-transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34288026Jamy O, Innis-Shelton R, Bal S, Paluri R, Salzman D, Di Stasi A, Costa L, Meredith R, Lamb L, Minagawa K, Mineishi S, Saad A. Phase II clinical trial of one dose of post-transplant cyclophosphamide for graft versus host disease prevention following myeloablative, peripheral blood stem cell, matched-unrelated donor transplantation. Am J Hematol. 2021 Oct 1;96(10):E396-E398. doi: 10.1002/ajh.26296. Epub 2021 Jul 31. No abstract available.
- UAB Bone Marrow Transplantation and Cell Therapy ProgramLink provided by the sponsor.
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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.
Racquel D Innis-Shelton, MD principal investigator
University of Alabama at Birmingham
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cyclophosphamide in people with Leukemia and Lymphoma, run by University of Alabama at Birmingham 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: finished, as of the sponsor's last update on 30 Sep 2022. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Jun 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Grade II-IV Acute GVHD (time frame: Till 100 days post transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02065154, which also lists 1 linked publication.
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 30 Sep 2022.