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for people ages 5–45 (full criteria)
at Oakland, California and other locations
study started
estimated completion:



This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with SCD with strokes; and (2) adults with severe SCD.

Official Title

Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients With Symptomatic Sickle Cell Disease


This study is designed as a Phase II multi-center trial to determine the feasibility of achieving a high rate of event-free survival (EFS) at 2 years post transplant using pre-conditioning hydroxyurea (HU) with a conditioning regimen that consists of a combination of Thymoglobulin/Cyclophosphamide/Fludarabine/Thiotepa with post-grafting high-dose cyclophosphamide in patients with severe SCD who have HLA-haploidentical donors. EFS is defined as survival without a qualifying event. This is a single arm study in which participants will be enrolled into one of two strata. The first stratum will be restricted to children who have stroke and 40 children will be enrolled in this stratum. The second stratum will consist of adult patients with severe sickle cell disease and 40 participants will be enrolled in this stratum.


Sickle Cell Disease Reduced Intensity Conditioning Haploidentical Bone Marrow Transplant Anemia, Sickle Cell Cyclophosphamide Fludarabine phosphate Thymoglobulin Thiotepa Fludarabine Hydroxyurea Mesna


You can join if…

Open to people ages 5–45

Adequate physical function as measured by all of the following:

  1. A Karnofsky/Lansky performance score of ≥ 60.
  2. Cardiac function: Left ventricular ejection fraction (LVEF) > 40%; or LV shortening fraction > 26% by cardiac echocardiogram or by Multi Gated Acquisition Scan (MUGA)scan.
  3. Pulmonary function: Pulse oximetry with a baseline O2 saturation of ≥ 85% and Diffusing capacity of the lung for carbon monoxide (DLCO) > 40% (corrected for hemoglobin).
  4. Renal function: Serum creatinine ≤ 1.5 x upper limit of normal for age
  5. Hepatic function: Serum conjugated (direct) bilirubin < 2x upper limit of normal for age as per local laboratory; alanine aminotransferase (ALT) and aspartate aminotransferase (AST) < 5 times upper limit of normal as per local laboratory.Participants with hyperbilirubinemia as the result of hyperhemolysis, or a severe drop in hemoglobin post blood transfusion, are not excluded.
  6. Liver MRI using a validated methodology per institutional preference (T2 or R2 or by ferriscan [R2 MRI]) for estimation of hepatic iron content is required for participants who are currently receiving ≥8 packed red blood cell transfusions per year for ≥1 year or have received ≥20 packed red blood cell transfusions (lifetime cumulative). Participants who have hepatic iron content ≥ 10 mg Fe/g liver dry weight by liver MRI must have a Gastroenterology/hepatology consultation with liver biopsy and histological examination including documentation of the absence of cirrhosis,bridging fibrosis, and active hepatitis.

  7. Participants must have a first-degree related HLA-haploidentical related donor who is willing and able to donate bone marrow. Umbilical cord blood or peripheral blood stem cell donors will not be accepted.

Inclusion Criteria for Stratum 1: Children Ages 5.00 - 14.99 years of age at enrollment

  1. Participants (Hb SS or Sß° Thalassemia) with overt stroke ischemia based on neuroimaging, clinical evidence of permanent neurological injury lasting for 24 hours, or both.

If there is clinical or radiologic evidence of a recent cerebral infarct by cerebral MRI/MRA within 30 days prior to enrollment, participants will be deferred for ≥ 6 months with repeat cerebral MRI/MRA to ensure stabilization of the neurologic event prior to proceeding to transplantation.

Inclusion Criteria for Stratum 2: Adults Ages 15.00 - 45.99 at enrollment

Participants with sickle cell anemia (Hb SS or Sß° Thalassemia) who are 15.00 - 45.99 years of age at enrollment AND who have one or more of the following:

  1. Clinically significant neurologic event (stroke) or any neurological deficit lasting >24 hours;
  2. History of two or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea);
  3. History of three or more severe vaso-occlusive pain crises per year in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e.a pain management plan and/or treatment with hydroxyurea); painful episodes related to priapism, osteonecrosis or any sickle-related complication are acceptable;
  4. Administration of regular RBC transfusion therapy, defined as receiving ≥8 packed red blood cell transfusions per year for ≥1 year in the 12 months before enrollment to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome);
  5. An echocardiographic finding of tricuspid valve regurgitant jet velocity (TRJV) ≥ 2.7 m/sec.

You CAN'T join if...

  1. Participants who have an HLA-matched sibling who is able and willing to donate bone marrow. Patients with a HLA-matched unrelated donor are not excluded.
  2. Uncontrolled bacterial, viral or fungal infection in the 6 weeks before enrollment(currently taking medication with evidence of progression of clinical symptoms or radiologic findings).
  3. Evidence of HIV infection or known HIV positive serology.
  4. Participants who have received a previous hematopoetic cell transplant (HCT).
  5. Participants who have participated in another clinical trial in which the patient received an investigational or off-label use of a drug or device within 3 months of enrollment.
  6. Females who are pregnant or breastfeeding.
  7. Participants with clinically significant, uncontrolled autoimmune disease, requiring active medical management (immunosuppressive therapy or chemotherapy), which, in the judgment of the local Principal Investigator, indicates that the patient could not tolerate transplantation.
  8. Females of child bearing potential (to include all female participants > 10 years of age, unless postmenopausal for a minimum of 1 year before the time of consent or surgically sterilized), who do not agree to practice two (2) effective methods of contraception at the same time, or do not agree to practice true abstinence when this is in line with the preferred and usual lifestyle of the subject, from the time of signing of informed consent through 12 months post-transplant.
  9. Males (even if surgical sterilized) who do not agree to practice effective barrier contraception, or who do not agree to practice true abstinence from the time of signing informed consent through 12 months post-transplant.
  10. . Anti-donor specific HLA antibodies: Positive anti-donor HLA antibody is defined as a positive crossmatch test of any titer (by complement-dependent cytotoxicity or flow cytometric testing) or the presence of anti-donor HLA antibody to the high expression loci HLA-A, -B, -C, or -DRB1 with mean fluorescence intensity >3000 by solid phase immunoassay. This will be measured before the final donor selection, and at least 180 days or less before HCT.


  • UCSF Benioff Children's Hospital Oakland accepting new patients
    Oakland, California, 94609, United States
  • Texas Transplant Institute accepting new patients
    San Antonio, Texas, 78229, United States
  • Northside Hosptial accepting new patients
    Atlanta, Georgia, 30342, United States
  • H. Lee Moffitt Cancer Center accepting new patients
    Tampa, Florida, 33612, United States


accepting new patients
Start Date
Completion Date
Medical College of Wisconsin
Blood and Marrow Transplant Clinical Trials Network Website
National Marrow Donor Program
Phase 2
Study Type
Last Updated
November 29, 2017
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