Hematological MalignanciesNCT02300571
Observational Study of the Combination of Post-transplant High Dose Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Acute Graft-versus-Host Disease in Patients Eligible to Allogeneic Hematopoietic Stem Cell Transplant
An observational study of Hematological Malignancies, run by Fondazione del Piemonte per l'Oncologia at 2 sites in Italy.
Trial summary
This observational study is proposed to observe the effect of high-dose, post-transplantation cyclophosphamide after a T cell-replete, HLA-matched PBSC graft from an HLA-identical or mismatched donor. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Hematological Malignancies
- How many people
- 60 participants
- Where
- 2 sitesItaly
- Study dates
- Sep 2013to Jan 2018
- Phase
- Observational
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with Hematological Malignancies Eligible to Allogeneic Hematopoietic Stem Cell Transplant using Peripheral Blood Stem Cells (PBSC) from unrelated or related, HLA-identical or partially mismatched donors
Who may be able to join
- Patient is scheduled for transplant of 'mobilized' peripheral blood stem cells (PBSC) from a genotypically HLA-unrelated or related identical or partially mismatched stem cell donor.
- Patient is ≥ 18 years and ≤ 65 years of age.
- Diagnosis of malignancy. Patients will be divided on the basis of their disease in low risk and high risk patients.
- High risk diseases: AML > CR1, ALL > CR1, CML in CP #2, AP or BP, non-Hodgkin's lymphoma > CR2, Hodgkin's lymphoma > CR2, other patient with refractory malignancy Low risk: multiple myeloma (all patients), AML in CR1, myelodysplastic syndrome beyond RA (including CMML) and ALL in CR1.
- Patient or legal guardian has signed/dated the informed consent form.
- Female patients must have a negative pregnancy test (blood or urine) unless they are prepuberal or surgically sterile.
Show all 9Show fewer
- Estimated Creatinine Clearance ≥ 60 mL/min at time of consent.
- Total bilirubin is ≤ 1.5 times the upper limit of normal at time of consent.
- SGOT and SGPT are ≤ 2.0 times the upper limit of normal at time of consent.
Who cannot join
- Patient > 65 years of age
- Patient has not signed/dated the informed consent form.
- Patient is receiving a T-cell depleted hematopoietic stem cell graft.
- Pregnant or lactating women
- Patient has an acute pulmonary infection suspected on the basis of abnormal chest x-ray.
- Patient has an active systemic infection not controlled with anti-microbial therapy.
- Patient is a known carrier of any of the Human Immune Deficiency Viruses (HIV-1 or others).
Trial locations and contacts
2 sites in 1 country.
Italy2 sites, 2 recruiting
- Recruiting
Fondazione del Piemonte per l'Oncologia
Candiolo, 10060
Luisa Gioeni, PharmD, +39011993 ext. 3959, luisa.gioeni@ircc.it
Fabrizio Carnevale Schianca, MD
- Not yet recruiting
Ospedale Regina Margherita
Torino, 10126
Massimo Berger, MD, +39011.313 ext. 5360, massimoberger@gmail.com
Franca Fagioli, MD
About this study
Official title: An Observational Retrospective/Prospective Study of the Combination of Post-transplant High Dose Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Acute Graft-versus-Host Disease in Patients Eligible to Allogeneic Hematopoietic Stem Cell Transplant Using Peripheral Blood Stem Cells (PBSC) From Unrelated or Related, HLA-identical or Partially Mismatched Donors
This observational study is proposed to observe the effect of high-dose, post-transplantation cyclophosphamide after a T cell-replete, HLA-matched PBSC graft from an HLA-identical or mismatched donor.
Read the detailed description
Allogeneic hematopoietic cell transplantation (HCT) remains the only curative approach for many hematological malignancies. In allogeneic HCT the donor immune system through the donor lymphocytes exerts both a beneficial and detrimental effect. Graft versus host disease (GVHD) represents the major complication and cause of mortality of allogeneic HCT. The principal aim that clinical transplant research must accomplish in the next years is to elaborate a transplant strategy devoid of any GVHD but still capable of generating, through donor lymphocytes, the graft versus tumor effect (GVT). The most used GVHD prophylaxis regimen remains the association of a calcineurin-inhibitor (CNIs) for six months and four low-doses of methotrexate (MTX) but the long length prophylaxis impacts on the process of post-transplant immune reconstitution slowing it down and exposing patients to a high risk of developing severe infections. The use of post-transplant cyclophosphamide looks the most promising among the new approaches to GVHD control. The study design is an observational retrospective/prospective Study in Patients Eligible to Allogeneic Hematopoietic Stem Cell Transplant using Peripheral Blood Stem Cells (PBSC) from unrelated or related, HLA-identical or partially mismatched donors. In case of unrelated donor, donor selection will be done accordingly to Italian Bone Marrow Donor Registry (IBMDR). This protocol and the treatment plan outlined below are limited to the plan or GVHD prevention.
The treatment plan for all patients including pre-conditioning therapy, TBI/chemotherapy, central nervous system prophylaxis and other planned therapies, is described in the primary transplant protocols which the patient has been assigned by the investigational site.
- Conditions
- Hematological Malignancies
- Medical subject headings
- Hematologic Neoplasms
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of the observed GVHD rate and infections
Evaluations through day 100 after transplantation will be performed with:
- Complete blood count (CBC), including differential and platelet count per standard practice guidelines at the performance site.
- Blood chemistries: including sodium, potassium, chloride, bicarbonate (HCO3) or total carbon dioxide (CO2), glucose, blood urea nitrogen (BUN), creatinine, calcium, magnesium, phosphorus, total bilirubin, total protein, albumin, serum glutamic oxaloacetic transaminase (SGOT), lactic dehydrogenase (LDH), alkaline phosphatase per standard practice guidelines at the performance site.
Tacrolimus whole blood concentrations weekly starting on day 6. CMV surveillance, Aspergillus surveillance will be performed per standard practice guidelines at the performance site.
Evaluations after 100 days post-transplant will be completed per standard practice guidelines at each performance site.
Measured: 100 day
Secondary outcomes (2)
Overall survival
To determine the overall survival at 1 years (OS)
Measured: 1 years
Progression-free survival
To determine progression-free survival at 1 years (PFS)
Measured: 1 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2014 (estimated)
- Study startSep 2013
- Primary completionDec 2017 (estimated)
- Study completionJan 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hematological MalignanciesStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Other
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fondazione del Piemonte per l'Oncologia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Massimo Aglietta, MD, Principal investigator, Fondazione del Piemonte per l'OncologiaFranca Fagioli, MD, Study chair, Ospedale Regina MargheritaFabrizio Carnevale-Schianca, MD, Study chair, Fondazione del Piemonte per l'Oncologia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02300571
- Sponsor's protocol ID
- HDCTX 2013
- Status last verified
- Aug 2017
- First submitted
- 21 Nov 2014
- Last updated
- 5 Sep 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Carnevale-Schianca F, Caravelli D, Gallo S, Becco P, Paruzzo L, Poletto S, Polo A, Mangioni M, Salierno M, Berger M, Pessolano R, Saglio F, Gottardi D, Rota-Scalabrini D, Grignani G, Fizzotti M, Ferrero I, Frascione PMM, D'Ambrosio L, Gaidano V, Gammaitoni L, Sangiolo D, Saglietto A, Vassallo E, Cignetti A, Aglietta M, Fagioli F. Post-Transplant Cyclophosphamide and Tacrolimus-Mycophenolate Mofetil Combination Governs GVHD and Immunosuppression Need, Reducing Late Toxicities in Allogeneic Peripheral Blood Hematopoietic Cell Transplantation from HLA-Matched Donors. J Clin Med. 2021 Mar 11;10(6):1173. doi: 10.3390/jcm10061173.
PubMed 33799685Carnevale-Schianca F, Caravelli D, Gallo S, Coha V, D'Ambrosio L, Vassallo E, Fizzotti M, Nesi F, Gioeni L, Berger M, Polo A, Gammaitoni L, Becco P, Giraudo L, Mangioni M, Sangiolo D, Grignani G, Rota-Scalabrini D, Sottile A, Fagioli F, Aglietta M. Post-Transplant Cyclophosphamide and Tacrolimus-Mycophenolate Mofetil Combination Prevents Graft-versus-Host Disease in Allogeneic Peripheral Blood Hematopoietic Cell Transplantation from HLA-Matched Donors. Biol Blood Marrow Transplant. 2017 Mar;23(3):459-466. doi: 10.1016/j.bbmt.2016.12.636. Epub 2016 Dec 27.
PubMed 28039079
Study at a glance
From the registry record, last updated by the sponsor on 5 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Retrospective/Prospective Study of the Combination of Post-transplant High Dose Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Acute Graft-versus-Host Disease in Patients Eligible to Allogeneic Hematopoietic Stem Cell Transplant Using Peripheral Blood Stem Cells (PBSC) From Unrelated or Related, HLA-identical or Partially Mismatched Donors
- ClinicalTrials.gov ID
- NCT02300571
- Acronym / sponsor ID
- HDCTX 2013
- Conditions
- Hematological Malignancies
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Fondazione del Piemonte per l'OncologiaUniversity, hospital or other
- Study start
- Sep 2013
- Primary completion
- Dec 2017 (estimated)
- Study completion
- Jan 2018 (estimated)
- First posted
- 25 Nov 2014 (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 observational study is proposed to observe the effect of high-dose, post-transplantation cyclophosphamide after a T cell-replete, HLA-matched PBSC graft from an HLA-identical or mismatched donor.
Detailed description
Allogeneic hematopoietic cell transplantation (HCT) remains the only curative approach for many hematological malignancies. In allogeneic HCT the donor immune system through the donor lymphocytes exerts both a beneficial and detrimental effect. Graft versus host disease (GVHD) represents the major complication and cause of mortality of allogeneic HCT. The principal aim that clinical transplant research must accomplish in the next years is to elaborate a transplant strategy devoid of any GVHD but still capable of generating, through donor lymphocytes, the graft versus tumor effect (GVT). The most used GVHD prophylaxis regimen remains the association of a calcineurin-inhibitor (CNIs) for six months and four low-doses of methotrexate (MTX) but the long length prophylaxis impacts on the process of post-transplant immune reconstitution slowing it down and exposing patients to a high risk of developing severe infections. The use of post-transplant cyclophosphamide looks the most promising among the new approaches to GVHD control. The study design is an observational retrospective/prospective Study in Patients Eligible to Allogeneic Hematopoietic Stem Cell Transplant using Peripheral Blood Stem Cells (PBSC) from unrelated or related, HLA-identical or partially mismatched donors. In case of unrelated donor, donor selection will be done accordingly to Italian Bone Marrow Donor Registry (IBMDR). This protocol and the treatment plan outlined below are limited to the plan or GVHD prevention.
The treatment plan for all patients including pre-conditioning therapy, TBI/chemotherapy, central nervous system prophylaxis and other planned therapies, is described in the primary transplant protocols which the patient has been assigned by the investigational site.
Study design
- Observational model
- Case only
- Time perspective
- Other
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with Hematological Malignancies Eligible to Allogeneic Hematopoietic Stem Cell Transplant using Peripheral Blood Stem Cells (PBSC) from unrelated or related, HLA-identical or partially mismatched donors
Key inclusion criteria (9 listed)
- Patient is scheduled for transplant of 'mobilized' peripheral blood stem cells (PBSC) from a genotypically HLA-unrelated or related identical or partially mismatched stem cell donor.
- Patient is ≥ 18 years and ≤ 65 years of age.
- Diagnosis of malignancy. Patients will be divided on the basis of their disease in low risk and high risk patients.
- High risk diseases: AML > CR1, ALL > CR1, CML in CP #2, AP or BP, non-Hodgkin's lymphoma > CR2, Hodgkin's lymphoma > CR2, other patient with refractory malignancy Low risk: multiple myeloma (all patients), AML in CR1, myelodysplastic syndrome beyond RA (including CMML) and ALL in CR1.
- Patient or legal guardian has signed/dated the informed consent form.
Key exclusion criteria (7 listed)
- Patient > 65 years of age
- Patient has not signed/dated the informed consent form.
- Patient is receiving a T-cell depleted hematopoietic stem cell graft.
- Pregnant or lactating women
- Patient has an acute pulmonary infection suspected on the basis of abnormal chest x-ray.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Incidence of the observed GVHD rate and infections
Time frame: 100 day
Evaluations through day 100 after transplantation will be performed with: 1. Complete blood count (CBC), including differential and platelet count per standard practice guidelines at the performance site. 2. Blood chemistries: including sodium, potassium, chloride, bicarbonate (HCO3) or total carbon dioxide (CO2), glucose, blood urea nitrogen (BUN), creatinine, calcium, magnesium, phosphorus, total bilirubin, total protein, albumin, serum glutamic oxaloacetic transaminase (SGOT), lactic dehydrogenase (LDH), alkaline phosphatase per standard practice guidelines at the performance site. Tacrolimus whole blood concentrations weekly starting on day 6. CMV surveillance, Aspergillus surveillance will be performed per standard practice guidelines at the performance site. Evaluations after 100 days post-transplant will be completed per standard practice guidelines at each performance site.
2 secondary outcomes
Overall survival
Time frame: 1 years
Progression-free survival
Time frame: 1 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Sep 2017.
- Derived from this study: PubMed 33799685Carnevale-Schianca F, Caravelli D, Gallo S, Becco P, Paruzzo L, Poletto S, Polo A, Mangioni M, Salierno M, Berger M, Pessolano R, Saglio F, Gottardi D, Rota-Scalabrini D, Grignani G, Fizzotti M, Ferrero I, Frascione PMM, D'Ambrosio L, Gaidano V, Gammaitoni L, Sangiolo D, Saglietto A, Vassallo E, Cignetti A, Aglietta M, Fagioli F. Post-Transplant Cyclophosphamide and Tacrolimus-Mycophenolate Mofetil Combination Governs GVHD and Immunosuppression Need, Reducing Late Toxicities in Allogeneic Peripheral Blood Hematopoietic Cell Transplantation from HLA-Matched Donors. J Clin Med. 2021 Mar 11;10(6):1173. doi: 10.3390/jcm10061173.
- Derived from this study: PubMed 28039079Carnevale-Schianca F, Caravelli D, Gallo S, Coha V, D'Ambrosio L, Vassallo E, Fizzotti M, Nesi F, Gioeni L, Berger M, Polo A, Gammaitoni L, Becco P, Giraudo L, Mangioni M, Sangiolo D, Grignani G, Rota-Scalabrini D, Sottile A, Fagioli F, Aglietta M. Post-Transplant Cyclophosphamide and Tacrolimus-Mycophenolate Mofetil Combination Prevents Graft-versus-Host Disease in Allogeneic Peripheral Blood Hematopoietic Cell Transplantation from HLA-Matched Donors. Biol Blood Marrow Transplant. 2017 Mar;23(3):459-466. doi: 10.1016/j.bbmt.2016.12.636. Epub 2016 Dec 27.
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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.
Massimo Aglietta, MD principal investigator
Fondazione del Piemonte per l'Oncologia
Franca Fagioli, MD study chair
Ospedale Regina Margherita
Fabrizio Carnevale-Schianca, MD study chair
Fondazione del Piemonte per l'Oncologia
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hematological Malignancies, run by Fondazione del Piemonte per l'Oncologia at 2 sites in Italy.
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 9 inclusion and 7 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: status not confirmed, as of the sponsor's last update on 5 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Italy. 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Fabrizio Carnevale-Schianca, MD (+39011993 ext. 3623, fabrizio.carnevale@ircc.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of the observed GVHD rate and infections (time frame: 100 day). 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 NCT02300571, which also lists 2 linked publications.
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 5 Sep 2017.