Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies

Sponsor
M.D. Anderson Cancer Center
Study ID
NCT04776850
Phase
EARLY_PHASE1
Status
Withdrawn

Conditions

  • Beta Thalassemia Major
  • Sickle Beta 0 Thalassemia
  • Sickle Beta Plus Thalassemia
  • Sickle Beta Thalassemia
  • Sickle Cell Disease
  • Sickle Cell-SS Disease

Eligibility Criteria

Sex
ALL
Age
2 Years - 30 Years
Healthy Volunteers
Not accepted

Interventions

  • Bortezomib — DRUG
    Given IV
  • Busulfan — DRUG
    Given IV
  • Cyclophosphamide — DRUG
    Given IV
  • Dexamethasone — DRUG
    Given IV
  • Fludarabine Phosphate — DRUG
    Given IV
  • Hematopoietic Cell Transplantation — PROCEDURE
    Undergo HCT
  • Lapine T-Lymphocyte Immune Globulin — BIOLOGICAL
    Given IV
  • Mycophenolate Mofetil — DRUG
    Given IV or PO
  • Plasmapheresis — PROCEDURE
    Undergo plasmapheresis
  • Rituximab — BIOLOGICAL
    Given IV
  • Tacrolimus — DRUG
    Given IV or PO

Study Details

This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies). PTIS helps prepare the body for the transplant and lowers the risk of developing graft versus host disease (GVHD). Hematopoietic cells are found in the bone marrow and produce blood cells. Hematopoietic cell transplantation (HCT) injects healthy hematopoietic cells into the body to support blood cell production. PTIS and HCT may help to control severe hemoglobinopathies.

Key Dates

Start date
Dec 29, 2020
Status verified
Nov 2022
Primary completion
Dec 5, 2022
Completion
Dec 5, 2022

Study Design

Enrollment
0 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment (PTIS, HCT)
    See Detailed Description.

Primary Outcome Measure

Event-free survival (EFS) [ Time Frame: At 2 years post-hematopoietic cell transplantation (HCT) ]

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