MDSG-14B2

Phase 1

• Conditions: Myelodysplastic syndrome; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]

• Registration Number: CTIS2024-511651-17-00
• Lead Sponsor: Karolinska University Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-02-13
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 175

Inclusion Criteria

Signed informed consent, Age = 18 years, Subjects eligible for stem cell transplantation, Subjects having the disease myelodysplastic syndrome (MDS), mixed myelodysplastic/myeloproliferative syndrome or acute myeloid leukemia (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

Exclusion Criteria

No traceable 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: To prevent clinical events (relapse or non-relapse death) in minimal residual disease (MRD) positive subjects;Secondary Objective: MRD positive cohort: To induce molecular remission, MRD positive cohort: To evaluate the incidence and severity of graft-versus-host disease (GVHD), MRD positive cohort: To evaluate safety during pre-emptive treatment, Whole study cohort: To prevent relapse or non-relapse death, Whole study cohort: To evaluate the incidence and severity of GVHD, Whole study cohort: To prolong overall survival;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)

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):MRD positive cohort: Relapse-free survival;Secondary end point(s):MRD positive cohort: GVHD and relapse-free survival;Secondary end point(s):MRD positive cohort: Proportion of MRD positive subjects achieving MRD-negativity;Secondary end point(s):MRD positive cohort: Incidence and severity of GVHD;Secondary end point(s):MRD positive cohort: Safety in MRD positive subjects subjected to MRD-guided clinical intervention;Secondary end point(s):Whole study cohort: Relapse-free survival;Secondary end point(s):Whole study cohort: GVHD and relapse-free survival;Secondary end point(s):Whole study cohort: Incidence and severity of GVHD;Secondary end point(s):Whole study cohort: Overall survival