Transplant-Related Hematologic MalignancyPhase 2SIR-POSANCT03434704
Peripheral Blood Stem Cell Transplantation From Family Haploidentical Donors in Patients With Myelodysplastic Syndrome and Acute Leukemia Under Primary Prophylaxis With Posaconazole
A Phase 2 study testing Conditioning treatment "Thiotepa-Treosulfan-Fludarabine", PBSC graft and GvHD prophylaxis in people with Transplant-Related Hematologic Malignancy and Leukemia, Acute, run by Ciceri Fabio at 1 site in Italy.
Trial summary
SIR-POSA is a phase II trial of peripheral blood stem cell (PBSC) transplantation from a partially compatible family (Haplo) donor in patients with a blood tumor (myelodysplastic syndrome (MDS) and acute leukemia) treated for the prevention of primary fungal infections with posaconazole. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transplant-Related Hematologic Malignancy, Leukemia, Acute, Myelodysplastic Syndromes
- What is being tested?
- Conditioning treatment "Thiotepa-Treosulfan-Fludarabine", PBSC graft and GvHD prophylaxis
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteItaly
- Study dates
- 18 Jun 2018to 13 Oct 2019
- Phase
- Phase 2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of myelodysplastic syndromes or acute leukemia
- Activation of an alternative donor search by the Italian Bone Marrow Donor Registry (IBMDR) with absence of a 10/10 Human Leukocyte Antigens (HLA) matched unrelated donor
- Age >18
- Unavailability of a HLA-matched related donor (MRD)
- Performance status : Eastern Cooperative Oncology Group (ECOG) <3
- Written and signed informed consent
- Life expectancy not severely limited by concomitant illness.
Who cannot join
- Women of child-bearing potential who do not agree to abstinence or, if sexually active, do not agree to the use of highly effective method of birth control such as condoms, implants, injectables, combined oral contraceptives, intrauterine devices (IUDs), vasectomised partner on treatment and for at least 6 months thereafter.
- Pregnant or nursing (lactating) women.
- Known allergies, hypersensitivity, or intolerance to any experimental drugs.
- Any active, uncontrolled infection.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase II Trial of Allogeneic Peripheral Blood Stem Cell Transplantation From Family Haploidentical Donors in Patients With Myelodysplastic Syndrome and Acute Leukemia Under Primary Antifungal Prophylaxis With Posaconazole.
SIR-POSA is a phase II trial of peripheral blood stem cell (PBSC) transplantation from a partially compatible family (Haplo) donor in patients with a blood tumor (myelodysplastic syndrome (MDS) and acute leukemia) treated for the prevention of primary fungal infections with posaconazole.
The aim is evaluate the composite end-point graft-versus-host disease-free, relapse-free survival (GRFS) in these patients and evaluate the feasibility and efficacy of posaconazole oral tablets as primary antifungal prophylaxis.
- Conditions
- Transplant-Related Hematologic Malignancy, Leukemia, Acute, Myelodysplastic Syndromes, Graft Vs Host Disease, Fungal Infection
- Being tested
- Conditioning treatment "Thiotepa-Treosulfan-Fludarabine", PBSC graft, GvHD prophylaxis, Primary antifungal prophylaxis
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes, Graft vs Host Disease, Mycoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Conditioning treatment "Thiotepa-Treosulfan-Fludarabine"Drug
Also called TTF
Thiotepa iv 5 mg/kg/bid (total dose TD 10 mg/kg) day -3,-2; Treosulfan iv 14g/mq/day (TD 42 g/mq) as a single daily dose day -6, -5, -4; Fludarabine iv 30 mg/m2 (TD 150 mg/m2) day -6, -5, -4, -3, -2. Thiotepa TD 5 mg/kg and treosulfan TD 36 g/mq dose reduction according to age > 65 years.
PBSC graftProcedure or surgery
(target 4-8 × 106 CD34+ cells/kg patient body weight)
GvHD prophylaxisDrug
Sirolimus orally, monitored two times a week to maintain a target therapeutic plasma level of 8-15 ng/ml from day +5.
Mycophenolate 15 mg/kg bid i.v. or per os day +5 through +28. Cyclophosphamide 50 mg/kg i.v. day +3+4. Mesna: >80% of the cyclophosphamide dose in 3 divided doses from day +3 daily through day +5.
Primary antifungal prophylaxisDrug
Posaconazole delayed-release tablet [available in 100 mg tablets]: 300 mg twice daily on the first day followed by a maintenance dose of 300 mg once a day, starting on the day 0 to day 85.
| Study group | Role | What they receive |
|---|---|---|
| Single Arm Treatment | Experimental | Conditioning treatment "Thiotepa-Treosulfan-Fludarabine"; PBSC graft; GvHD prophylaxis; Primary antifungal prophylaxis. Receives: Drug: Conditioning treatment "Thiotepa-Treosulfan-Fludarabine"; Procedure: PBSC graft; Drug: GvHD prophylaxis; Drug: Primary antifungal prophylaxis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Graft-versus-host disease-free, relapse-free survival (GRFS)
GRFS events is defined as the first event among grade III-IV acute Graft vs. Host Disease (GVHD), severe chronic GVHD, relapse and death from any cause
Measured: 2 years
Secondary outcomes (1)
Efficacy of antifungal prophylaxis strategy
The rate of proven, probable and possible invasive fungal diseases documented within the first 85 days after transplantation
Measured: 85 days after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2018
- Study start18 Jun 2018
- Primary completion13 Oct 2019
- Study completion13 Oct 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transplant-Related Hematologic MalignancyStudies 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ciceri Fabio
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Ciceri Fabio, IRCCS San Raffaele
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03434704
- Sponsor's protocol ID
- 2017-004423-78
- Other IDs
- 2017
- Acronym
- SIR-POSA
- Status last verified
- Jul 2022
- First submitted
- 9 Feb 2018
- Last updated
- 19 Jul 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of Allogeneic Peripheral Blood Stem Cell Transplantation From Family Haploidentical Donors in Patients With Myelodysplastic Syndrome and Acute Leukemia Under Primary Antifungal Prophylaxis With Posaconazole.
- ClinicalTrials.gov ID
- NCT03434704
- Acronym / sponsor ID
- SIR-POSA / 2017-004423-78
- Conditions
- Transplant-Related Hematologic Malignancy, Leukemia, Acute, Myelodysplastic Syndromes, Graft Vs Host Disease, Fungal Infection
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Ciceri FabioUniversity, hospital or other
- Study start
- 18 Jun 2018 (actual)
- Primary completion
- 13 Oct 2019 (actual)
- Study completion
- 13 Oct 2019 (actual)
- First posted
- 15 Feb 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.
SIR-POSA is a phase II trial of peripheral blood stem cell (PBSC) transplantation from a partially compatible family (Haplo) donor in patients with a blood tumor (myelodysplastic syndrome (MDS) and acute leukemia) treated for the prevention of primary fungal infections with posaconazole.
The aim is evaluate the composite end-point graft-versus-host disease-free, relapse-free survival (GRFS) in these patients and evaluate the feasibility and efficacy of posaconazole oral tablets as primary antifungal prophylaxis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm Treatment | Experimental | Drug: Conditioning treatment "Thiotepa-Treosulfan-Fludarabine", Procedure: PBSC graft, Drug: GvHD prophylaxis, Drug: Primary antifungal prophylaxisConditioning treatment "Thiotepa-Treosulfan-Fludarabine"; PBSC graft; GvHD prophylaxis; Primary antifungal prophylaxis. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosis of myelodysplastic syndromes or acute leukemia
- Activation of an alternative donor search by the Italian Bone Marrow Donor Registry (IBMDR) with absence of a 10/10 Human Leukocyte Antigens (HLA) matched unrelated donor
- Age >18
- Unavailability of a HLA-matched related donor (MRD)
- Performance status : Eastern Cooperative Oncology Group (ECOG) <3
Key exclusion criteria (4 listed)
- Women of child-bearing potential who do not agree to abstinence or, if sexually active, do not agree to the use of highly effective method of birth control such as condoms, implants, injectables, combined oral contraceptives, intrauterine devices (IUDs), vasectomised partner on treatment and for at least 6 months thereafter.
- Pregnant or nursing (lactating) women.
- Known allergies, hypersensitivity, or intolerance to any experimental drugs.
- Any active, uncontrolled infection.
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 |
|---|---|---|
| Conditioning treatment "Thiotepa-Treosulfan-Fludarabine"Also: TTFThiotepa iv 5 mg/kg/bid (total dose TD 10 mg/kg) day -3,-2; Treosulfan iv 14g/mq/day (TD 42 g/mq) as a single daily dose day -6, -5, -4; Fludarabine iv 30 mg/m2 (TD 150 mg/m2) day -6, -5, -4, -3, -2. Thiotepa TD 5 mg/kg and treosulfan TD 36 g/mq dose reduction according to age > 65 years. | Drug | Single Arm Treatment |
| PBSC graft(target 4-8 × 106 CD34+ cells/kg patient body weight) | Procedure or surgery | Single Arm Treatment |
| GvHD prophylaxisSirolimus orally, monitored two times a week to maintain a target therapeutic plasma level of 8-15 ng/ml from day +5. Mycophenolate 15 mg/kg bid i.v. or per os day +5 through +28. Cyclophosphamide 50 mg/kg i.v. day +3+4. Mesna: >80% of the cyclophosphamide dose in 3 divided doses from day +3 daily through day +5. | Drug | Single Arm Treatment |
| Primary antifungal prophylaxisPosaconazole delayed-release tablet [available in 100 mg tablets]: 300 mg twice daily on the first day followed by a maintenance dose of 300 mg once a day, starting on the day 0 to day 85. | Drug | Single Arm Treatment |
Primary outcome
Graft-versus-host disease-free, relapse-free survival (GRFS)
Time frame: 2 years
GRFS events is defined as the first event among grade III-IV acute Graft vs. Host Disease (GVHD), severe chronic GVHD, relapse and death from any cause
1 secondary outcome
Efficacy of antifungal prophylaxis strategy
Time frame: 85 days after transplantation
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jul 2022.
Related trials for Transplant-Related Hematologic Malignancy
RecruitingPhase 2
CMV-MVA Triplex Vaccination in HLA-Matched Related Stem Cell Donors for the Prevention of CMV Infection in Patients Undergoing Hematopoietic Stem Cell Transplant
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
RecruitingPhase 2
GM-CSF With Post-Transplant Cyclophosphamide
Transplant-Related Hematologic Malignancy
Not yet recruiting
Effects of Resistance Training on Recovery During Hematopoietic Stem Cell Transplantation
Hematologic Malignancies, Hematopoietic Stem Cell Transplantation
Not yet recruitingPhase 2
CD34+ Transplants for Myeloid Malignancies
Leukemia, Myeloid, Acute, Leukemia, Lymphocytic, Acute
RecruitingPhase 2
Optimizing GVHD Prophylaxis After Allogeneic Hematopoietic Cell Transplantation
Hematological Malignancies, Graft-versus-Host Disease (GVHD)
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.
The record lists no contact. Use the official record to reach the sponsor.
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 Conditioning treatment "Thiotepa-Treosulfan-Fludarabine", PBSC graft and GvHD prophylaxis in people with Transplant-Related Hematologic Malignancy and Leukemia, Acute, run by Ciceri Fabio at 1 site 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 7 inclusion and 4 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 19 Jul 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 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.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Graft-versus-host disease-free, relapse-free survival (GRFS) (time frame: 2 years). 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 19 Jul 2022.