Peripheral Blood Stem Cell Transplantation From Family Haploidentical Donors in Patients With Myelodysplastic Syndrome and Acute Leukemia Under Primary Prophylaxis With Posaconazole

Phase 2

Completed

• Conditions: Graft Vs Host Disease; Transplant-Related Hematologic Malignancy; Leukemia, Acute; Fungal Infection; Myelodysplastic Syndromes
• Interventions: Drug: Conditioning treatment "Thiotepa-Treosulfan-Fludarabine"; Procedure: PBSC graft; Drug: GvHD prophylaxis; Drug: Primary antifungal prophylaxis

• Registration Number: NCT03434704
• Lead Sponsor: Ciceri Fabio

Brief 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.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-02-09
• Study First Submitted QC: 2018-02-09
• Study First Posted: 2018-02-15
• Study Start: 2018-06-18
• Primary Completion: 2019-10-13
• Study Completion: 2019-10-13
• Status Verified: 2022-07
• Last Update Submitted: 2022-07-18
• Last Update Posted: 2022-07-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

• 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.

Exclusion Criteria

• 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Single Arm Treatment	GvHD prophylaxis	Conditioning treatment "Thiotepa-Treosulfan-Fludarabine"; PBSC graft; GvHD prophylaxis; Primary antifungal prophylaxis.
Single Arm Treatment	Conditioning treatment "Thiotepa-Treosulfan-Fludarabine"	Conditioning treatment "Thiotepa-Treosulfan-Fludarabine"; PBSC graft; GvHD prophylaxis; Primary antifungal prophylaxis.
Single Arm Treatment	PBSC graft	Conditioning treatment "Thiotepa-Treosulfan-Fludarabine"; PBSC graft; GvHD prophylaxis; Primary antifungal prophylaxis.
Single Arm Treatment	Primary antifungal prophylaxis	Conditioning treatment "Thiotepa-Treosulfan-Fludarabine"; PBSC graft; GvHD prophylaxis; Primary antifungal prophylaxis.

Primary Outcome Measures

Name	Time	Method
Graft-versus-host disease-free, relapse-free survival (GRFS)	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

Secondary Outcome Measures

Name	Time	Method
Efficacy of antifungal prophylaxis strategy	85 days after transplantation	The rate of proven, probable and possible invasive fungal diseases documented within the first 85 days after transplantation

Trial Locations

Locations (1)

Ospedale San Raffaele; 🇮🇹 Milano, Lombardia, Italy