Allo HSCT Using RIC and PTCy for Hematological Diseases
Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning (RIC) With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases
1 other identifier
interventional
56
1 country
1
Brief Summary
This is a Phase II study following subjects proceeding with our Institutional non-myeloablative cyclophosphamide/ fludarabine/total body irradiation (TBI) preparative regimen followed by a related, unrelated, or partially matched family donor stem cell infusion using post-transplant cyclophosphamide (PTCy), sirolimus and MMF GVHD prophylaxis.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started May 2023
Longer than P75 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 21, 2023
CompletedFirst Posted
Study publicly available on registry
April 10, 2023
CompletedStudy Start
First participant enrolled
May 1, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 22, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 22, 2028
July 1, 2025
June 1, 2025
4.5 years
February 21, 2023
June 27, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Evaluate rates of acute graft-versus-host disease (GVHD)
Number of participants with GVHD grades 2-4 after one year post transplant.
12 months
Evaluate rates of chronic graft-versus-host disease (GVHD)
Number of participants with chronic GVHD after one year post transplant.
12 months
Secondary Outcomes (3)
Observe rates of relapse (RR)
100 days
Overall Survival (OS)
72 months
Observe transplant related mortality (TRM)
12 months
Study Arms (1)
Cy/Flu/TBI + Post transplant CY
EXPERIMENTALInterventions
On day 0, a target dose of 5 x 106 CD34 cells/kg will be infused.
300 mg/day from day -7 to day 0. Allopurinol 150mg/m2/day for pediatric participants.
30 mg/m2 IV over 1 hour. Administered on day -6 to day -2.
Administered as a 2 hour IV infusion on day -6, +3, and +4.
On day 0, a target dose of 3 x 108 nucleated cells/kg recipient weight will be infused.
The dose of TBI will be 200 cGy given in a single fraction on day -1.
All participants begin +5 to day +60. Loading dose on day +5 of 5 mg/m2/day orally once (max dose of 8 mg). Maintenance dose 2.5 mg/m2 orally daily to maintain a level of 8-12 ng/ml (max dose of 4 mg).
All patients begin day +5 through day +35. 3 gram/day IV/PO for patients who are ≥ 40 kg divided in 2 doses. In obese patients (\>125% IBW) 15 mg/kg every 12 hours may be considered. Pediatric patients will receive MMF at the dose of 15 mg/kg/dose (maximum of 1 gram per dose) every 8 hours.
Eligibility Criteria
You may qualify if:
- Age 0 to 75 years of age with Karnofsky score ≥ 70% (≥ 16 years) or Lansky score ≥ 50 (\< 16 years).
- /6 or 6/6 related donor, OR a 7-8/8 HLA-A, B, C, DRB1 allele match, OR a haplotype (at least 5/10) matched related donor. Donors will be requested to provide PBSCs although bone marrow is acceptable according to donor preference.
- Acute Myeloid Leukemia (AML) and related precursor neoplasms:
- nd or greater complete remission (CR); first complete remission (CR1) in patients \> 60 years old; CR1 in ≤ 60 years old that is NOT considered as favorable-risk.
- Favorable risk AML is defined as having one of the following:
- t(8,21) without cKIT mutation
- inv(16) or t(16;16) without cKIT mutation
- Normal karyotype with mutated NPM1 and wild type FLT-ITD (unless persistently NPM1 positive by PCR following two cycles of chemotherapy)
- Normal karyotype with double mutated CEBPA
- Acute prolymphocytic leukemia (APL) in first molecular remission at the end of consolidation
- Acute lymphoblastic Leukemia (ALL) /lymphoma:
- CR2 or greater, CR1 unable to tolerate consolidation chemotherapy due to chemotherapy-related toxicities; CR1 high-risk ALL.
- High risk ALL is defined as having one of the following:
- Evidence of high risk cytogenetics, e.g. t(9;22), t(1;19), t(4;11), other MLL rearrangements, IKZF1
- years of age or older at diagnosis
- +24 more criteria
You may not qualify if:
- Pregnant or breast feeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a negative pregnancy test prior to starting therapy.
- Untreated active infection
- Active central nervous system malignancy
- CML in blast crisis
- Intermediate or high grade NHL, mantle cell NHL, and Hodgkin disease that is progressive on salvage therapy. Stable disease is acceptable to move forward provided it is non-bulky.
- Less than 3 months since prior myeloablative transplant
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, 55455, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Mark Juckett
University of Minnesota Masonic Cancer Center
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 21, 2023
First Posted
April 10, 2023
Study Start
May 1, 2023
Primary Completion (Estimated)
October 22, 2027
Study Completion (Estimated)
October 22, 2028
Last Updated
July 1, 2025
Record last verified: 2025-06