A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease

Part of paid clinical trials in Columbus, Ohio.

Sponsor
Nationwide Children's Hospital
Study ID
NCT01279616
Phase
PHASE2
Status
Terminated

Conditions

Eligibility Criteria

Sex
ALL
Age
N/A - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • Fludarabine monophosphate — DRUG
    180 mg/m2 over 6 days.
  • Rituximab — DRUG
    375 mg/m2 on day -13 and day -3
  • Busulfan — DRUG
    AUC 1000-1200 microM.mt
  • ATG — DRUG
    2.5 mg/kg for 3 days
  • Cyclophosphamide — DRUG
    50 mg/kg on day +3
  • Mycophenolate mofetil — DRUG
    15 mg/kg q 8 hours
  • Tacrolimus — DRUG
    0.03 mg/kg /d

Study Details

Majority of patients who are eligible for allogeneic HSCT for cure of severe sickle cell disease lack a matched family donor. This study aims for cure of sickle cell disease by performing unrelated donor (outside family) allogeneic HSCT. Donors or unrelated cord blood units will be selected from the NMDP database. It is designed to estimate the safety of a novel reduced toxicity, yet an immunosuppressive and myeloablative preparative regimen. This is meant for patients \<21 years old who have severe complications from sickle cell and do not have matched sibling donors in the family to undergo stem cell transplant. Patients will undergo transplant using unrelated donor stem cells after receiving the protocol therapy. They will be followed for 1 year to monitor for engraftment of donor cells and complications like graft versus host disease (GVHD), infections and death.

Key Dates

Start date
Sep 30, 2010
Status verified
Apr 2019
Primary completion
Jan 31, 2015
Completion
Jan 31, 2015

Study Design

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

Arms

  • Experimental: Hematopoietic Stem Cell Transplant
    Stem cell infusion on Day 0.

Primary Outcome Measure

Event-free Survival [ Time Frame: 1 year ]

Locations (1)

FacilityCityStateZIPSite coordinators
Nationwide Children's HospitalColumbusOhio43205-

Find similar trials in Columbus, OH

By condition
Sickle cell disease
By research site
Nationwide Children's Hospital· Columbus, OH

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