A study using subject-specific minimal residual disease markers to adopt treatment after allogeneic stem cell transplantation for subjects with myelodysplastic syndrome - MDS

Phase 1

Conditions: Myelodysplastic syndrome (MDS)
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2021-003809-22-NO

Lead Sponsor: Karolinska University Hospital

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 200

Inclusion Criteria

Signed informed consent
Age = 18 years
Subjects eligible for stem cell transplantation (SCT)
Subjects having the disease MDS, mixed myelodysplastic/myeloproliferative syndrome or AML with myelodysplasia related dysplasia and 20-29% marrow blasts
All female subjects of childbearing potential have to have negative pregnancy test within 2 weeks prior to inclusion to the study

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 100
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 100

Exclusion Criteria

No genetic aberration identified either in screening next generation sequencing panel or next generation sequencing panel performed at diagnosis
Uncontrolled hypertension, heart, liver, kidney related or other uncontrolled medical or psychiatric disorders
Mental inability, reluctance or language difficulties that results in difficulty understanding the meaning of study participation

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary study objective is to prevent clinical events (relapse or non-relapse death) in minimal residual disease (MRD) positive subjects ;Secondary Objective: MRD positive cohort: <br> To induce molecular remission<br> To evaluate the incidence and severity of graft-versus-host disease (GVHD)<br>To evaluate safety during pre-emptive treatment <br>Whole study cohort: <br>To prevent relapse or non-relapse death <br>To evaluate the incidence and severity of GVHD<br>To prolong overall survival <br>;Primary end point(s): The percentage of clinical events defined as relapse or non-relapse death at 12 months from verified MRD positivity (NMDSG14B Part 2 compared to Part 1);Timepoint(s) of evaluation of this end point: 12 months from MRD positivity

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): MRD positive cohort: <br>Relapse-free survival<br>Proportion of MRD positive subjects achieving MRD-negativity <br>Incidence and severity of GVHD<br>Safety in MRD positive subjects subjected to MRD-guided clinical intervention <br>Whole study cohort:<br>Relapse-free survival <br>Incidence and severity of GVHD<br>Overall survival<br>;Timepoint(s) of evaluation of this end point: Weeks: 6, 12, 18, 24; months: 9, 12, 15, 18, 21 and 2 years after SCT