Allogeneic HSCT in MDS Patients Based on Risk According to R-IPSS

• Sponsor: University Of Perugia (Other)
• Overall Status: Recruiting
• CT.gov ID: NCT04801563
• Collaborator: (none)
• Enrollment: 80
• Location: 1
• Arm: 1
• Anticipated Duration (Months): 72
• Patients Per Site Per Month: 1.1

Study Details

Study Description

Brief Summary

An "intention-to-treat" study to evaluate the impact of allogeneic HSCT with Total Marrow and Lymphoid irradiation (TMLI), followed by Treg/Tcon adoptive immunotherapy, on overall survival in patients affected by Myelodysplastic Syndrome (MDS), according to IPSS-R.

Condition or Disease	Intervention/Treatment	Phase
MDS and Allogeneic Stem Cell Transplantation	Other: MDS and AlloHSCT	N/A

Detailed Description

MDS patients with Intermediate, High, or Very-High R-IPSS risk will be enrolled. Allogeneic stem cell transplantation with regulatory and conventional T cell adoptive immunotherapy will be proposed to eligible High and Very-High risk MDS patients. Intermediate risk MDS patients will be assessed for risk modification every six months and moved to transplant in case of increased risk. Intermediate risk MDS patients will be also evaluated for the presence of molecular alterations (TP53, ASXL1, RUNX1). Overall survival of the entire cohort will be assessed as primary endpoint. Incidence of Treatment Related Mortality, acute Graft versus Host Disease, chronic Graft versus Host Disease, Relapse will be also assessed.

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 80 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Allogeneic Transplantation in Patients With Myelodysplastic Syndrome Based on Risk According to Revised-International Prognostic Scoring System (R-IPSS)
• Actual Study Start Date: Sep 20, 2019
• Anticipated Primary Completion Date: Sep 19, 2025
• Anticipated Study Completion Date: Sep 19, 2025

Arms and Interventions

Arm	Intervention/Treatment
Other: SINGLE ARM	Other: MDS and AlloHSCT; Allogeneic stem cell transplantation, based on TMLI and followed by infusion of donor regulatory T cells and conventional T cells and purified donor CD34+ hematopoietic stem cells

Outcome Measures

Primary Outcome Measures

1. overall survival [4 years]

   We will evaluate overall survival in patients with intermediate/High/very-High risk MDS

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 70 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Patients affected by Myelodysplastic Syndrome with IPSS-R INT, HIGH or VERY HIGH;

• Age <71 years;

• Signature of the informed consent.

Exclusion Criteria:

• Patients affected by Myelodysplastic Syndrome with IPSS-R VERY LOW or LOW;

• Patients affected by Myelodysplastic Syndrome/Myeloproliferative Neoplasm;

• Age >70 years;

• No signature of the informed consent

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Perugia	Perugia	PG	Italy	06132

Sponsors and Collaborators

• University Of Perugia

Investigators

• Principal Investigator: Andrea Velardi, MD, University Of Perugia

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Andrea Velardi, FULL PROFESSOR, University Of Perugia
• ClinicalTrials.gov Identifier: NCT04801563
• Other Study ID Numbers: 03/19
• First Posted: Mar 17, 2021
• Last Update Posted: Mar 17, 2021
• Last Verified: Mar 1, 2021

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Study Results

No Results Posted as of Mar 17, 2021