Allogeneic stem cell transplantation for patients with myelodysplastic syndrome based on risk assessment
Allogeneic Transplantation in Patients With Myelodysplastic Syndrome Based on Risk According to Revised-International Prognostic Scoring System (R-IPSS)
This study is testing if a special type of stem cell transplant combined with targeted treatments can help people with high-risk myelodysplastic syndrome live longer and healthier.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 80 (estimated) |
| Ages | N/A to 70 Years |
| Sex | All |
| Sponsor | University Of Perugia Academic / other |
| Drugs / interventions | immunotherapy |
| Locations | 1 site (Perugia, PG) |
| Trial ID | NCT04801563 on ClinicalTrials.gov |
What this trial studies
This study evaluates the effectiveness of allogeneic hematopoietic stem cell transplantation (HSCT) combined with Total Marrow and Lymphoid Irradiation (TMLI) and Treg/Tcon adoptive immunotherapy in improving overall survival for patients with Myelodysplastic Syndrome (MDS) categorized as Intermediate, High, or Very-High risk according to the Revised International Prognostic Scoring System (R-IPSS). Eligible patients will undergo regular risk assessments, and those with Intermediate risk may be considered for transplantation if their risk increases. The primary endpoint is overall survival, while secondary endpoints include treatment-related mortality and the incidence of graft versus host disease. This approach aims to tailor treatment based on individual risk profiles.
Who should consider this trial
Good fit: Ideal candidates are patients aged 70 years or younger with Intermediate, High, or Very-High risk MDS.
Not a fit: Patients with Very Low or Low risk MDS or those over 70 years old may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could significantly improve survival rates for patients with high-risk MDS.
How similar studies have performed: While similar approaches have been explored, this specific combination of therapies in high-risk MDS patients is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Perugia, PG
- University of Perugia — Perugia, Pg, Italy (Recruiting)
Study contacts
- Principal investigator: Andrea Velardi, MD — University Of Perugia
- Study coordinator: Antonio Pierini, MD
- Email: antonio.pierini@unipg.it
- Phone: +390755784147
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.