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

Conditions

• Stem Cell Transplantation

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date

Mar 2, 2022

Status verified

Sep 2023

Primary completion

Sep 18, 2023

Completion

Sep 18, 2023

Study Design

Enrollment

0 participants (actual)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Phase I: Sequential Pharmacological PTIS
• Experimental: Phase II: RTC Regimen and GVHD Prophylaxis Based on Post-Cy

Primary Outcome Measure

To determine the 100-day non-relapse mortality (NRM) rate when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC [ Time Frame: through study completion, an average of 1 year ]

Locations (1)

Find similar trials in Houston, TX

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