NCT05293509

Pharmacologic Pretransplant Immunosuppression (PTIS) + Reduced Toxicity Conditioning (RTC) Allogeneic Stem Cell Transplantation in Inherited Hematologic Disorders

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

To assess the outcomes of NRM when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC) and an allogeneic stem cell transplant (allo-SCT) and post-transplant graft-versus-host disease prophylaxis based on post-transplant cyclophosphamide (PT-Cy) in patients with inherited blood disorders.

Detailed description

Objectives Primary: To estimate the 100-day non-relapse mortality (NRM) rate when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC) and an allogeneic stem cell transplant (allo-SCT) and post-transplant graft-versus-host disease prophylaxis based on post-transplant cyclophosphamide (PT-Cy) in patients with inherited blood disorders. Secondary outcomes include the following: i. Immune reconstitution ii. Infectious complications iii. Quality of life (QOL) at 3 months,100 days, and 1 year post-transplant iv. OS, EFS, and GRFS v. Incidence of aGVHD at day 100. vi. Rate of chronic GVHD within the first-year post transplantation. vii. Rate of Graft failure

Conditions

• Stem Cell Transplantation

Interventions

Type	Name	Description
DRUG	Fludarabine	40 mg/m2/day i.v.- by vein
DRUG	Dexamethasone	25 mg/m2/day i.v.-by vein
DRUG	Cyclophosphamide	100 mg/m2 IV-by vein
DRUG	Bortezomib	Four doses of bortezomib at a dose of 1.3 mg/m2 -injection under the skin
DRUG	Rituximab	Four doses of rituximab at a dose of 375 mg/m2- by vein
DRUG	Busulfan	110 mg/m2 i.v-by vein
DRUG	Cyclophosphamide (Cy)	by vein
DRUG	Tacrolimus (or cyclosporine)	by vein
DRUG	Mycophenolate mofetil (MMF)	given by PO
DRUG	Rabbit ATG	by vein

Timeline

• Start date: 2022-03-02
• Primary completion: 2023-09-18
• Completion: 2023-09-18
• First posted: 2022-03-24
• Last updated: 2023-09-21

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study