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Trial record 1 of 2 for:    nct01461837
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Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease (HaploSCD)

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ClinicalTrials.gov Identifier: NCT01461837
Recruitment Status : Recruiting
First Posted : October 28, 2011
Last Update Posted : December 18, 2017
Sponsor:
Collaborators:
Information provided by (Responsible Party):

Study Description
Brief Summary:

This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients.

Funding Source - FDA OOPD


Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: CD34 selected T-cell depleted allogeneic SCT Phase 2

Detailed Description:

The purpose of this study is to investigate host myeloimmunosuppressive conditioning followed by familial haploidentical T cell depleted allogeneic stem cell transplantation in patients with high risk Sickle Cell Disease (SCD). It is hypothesized that it will be safe and well tolerated, and result in sustained donor chimerism, acceptable engraftment and immune reconstitution. Also, that it will limit SCD related organ damage resulting in improved and/or stable neurological, neurocognitive, pulmonary and pulmonary vascular function and health related quality of life (QOL).

Patients 2-20.99 years of age with a diagnosis of high-risk SCD and with an unaffected HLA partially matched family donor and meeting eligibility criteria (inclusion and exclusion criteria) are eligible.


Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Familial Haploidentical T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease (IND 14359)
Study Start Date : January 2012
Estimated Primary Completion Date : April 2018
Estimated Study Completion Date : April 2019

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: Haplo Stem Cell Transplantation
CD34 selected T-cell depleted allogeneic SCT
Drug: CD34 selected T-cell depleted allogeneic SCT
Hydroxyurea (60 mg/kg/day) and azathioprine (3 mg/kg/day) day -59 to day -11; fludarabine (30 mg/m2) Days -17, -16, -15, -14, -13; busulfan (3.2 mg/kg/day) Days -12, -11, -10, -9; thiotepa (10 mg/kg IV) day -8; cyclophosphamide (50 mg/kg) Days -7, -6, -5, -4; TLI on day -3; rabbit ATG (2.0 mg/kg/day) day -5,-4,-3, and -2; Stem Cell infusion day 0
Other Names:
  • Familial haploidentical
  • T-cell depleted
  • allogeneic stem cell transplantation
  • high risk Sickle Cell Disease


Outcome Measures

Primary Outcome Measures :
  1. Treatment related events [ Time Frame: 1 year ]
    Death, primary or late graft rejection, or recurrence of disease and acceptable rate of hematopoietic engraftment, acute and chronic graft-versus-host disease


Secondary Outcome Measures :
  1. neurological/neurocognitive status [ Time Frame: 2 years ]
    Change from baseline in neurological/neurocognitive status

  2. Pulmonary/pulmonary vascular status [ Time Frame: 2 years ]
    Change from baseline of Pulmonary/pulmonary vascular status

  3. Health-related quality of life [ Time Frame: 2 years ]
    Change from baseline of Health-related quality of life


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Homozygous Hemoglobin S Disease, or Hemoglobin S Beta0/+ thalassemia
  • Patients must demonstrate one or more of the following Sickle Cell Disease Complications

    1. Clinically significant neurologic event (stroke) or any neurologic deficit lasting >24 hours that is accompanied by an infarct on cerebral MRI
    2. Minimum of two episodes of acute chest syndrome.
    3. Recurrent painful events (at least 3 in the 2 years prior to enrollment).
    4. Abnormal TCD study requiring starting on chronic transfusion therapy.
    5. At least one silent infarct lesion on a MRI scan of the head.
  • A familial haploidentical donor without homozygous sickle cell disease
  • Adequate organ function (renal, liver, cardiac and pulmonary function)
  • Karnofsky or Lansky (age appropriate) Performance Score ≥50%
  • Liver biopsy is optional to assess for iron overload in chronically transfused patients.

Exclusion Criteria:

  • Females who are pregnant or breast-feeding
  • SCD Patients with documented uncontrolled infection
  • SCD patients who have an unaffected HLA matched family donor willing to proceed to donation
  • Karnofsky/Lansky (age appropriate) Performance Score <50% (hemiplegia alone secondary to a previous stroke is not an exclusion)
  • Demonstrated lack of compliance with medical care.
  • Clinically significant fibrosis or cirrhosis of the liver
  • Previously received a HSCT
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01461837


Contacts
Contact: Mitchell S Cairo, MD 914-594-2150 mitchell_cairo@nymc.edu
Contact: Erin Morris, RN 714-964-5359 erin_morris@nymc.edu

Locations
United States, California
University of California Los Angeles (UCLA) Recruiting
Los Angeles, California, United States, 90095
Contact: Theodore Moore, MD    310-825-6708    tbmoore@mednet.ucla.edu   
Contact: LaMarr Taylor, CCRP    310-794-8929    LaMarrTaylor@mednet.ucla.edu   
Principal Investigator: Theodore Moore, MD         
Children's Hospital and Research Center Oakland Completed
Oakland, California, United States, 94609
United States, Illinois
Lurie Children's Hospital Completed
Chicago, Illinois, United States, 60611-2605
United States, Missouri
Washington University/St. Louis Children's Hospital Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Shalini Shenoy, MD    314-454-6018    Shenoy@kids.wustl.edu   
Contact: Megan Holtmann    314-454-6018      
Principal Investigator: Shalini Shenoy, MD         
United States, New York
New York Medical College Recruiting
Valhalla, New York, United States, 10595
Contact: Mitchell S Cairo, MD    914-594-2150    mitchell_cairo@nymc.edu   
Contact: Sandi Fabricatore, RN, PNP    914-594-2152    Fabricatores@wcmc.com   
Principal Investigator: Mitchell S Cairo, MD         
United States, Ohio
Cincinnati Children's Hospital Withdrawn
Cincinnati, Ohio, United States, 45229
United States, Wisconsin
Medical College of Wisconsin/Children's Hospital of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Julie Talano, MD    414-955-4185    jtalano@mcw.edu   
Contact: Kathy Jodarski    414-266-2681    kjodarski@chw.org   
Principal Investigator: Julie Talano, MD         
Sponsors and Collaborators
New York Medical College
Children's Hospital & Research Center Oakland
Medical College of Wisconsin
Washington University School of Medicine
Tufts Medical Center
University of California, San Francisco
University of California, Los Angeles
Miltenyi Biotec GmbH
Ann and Robert H. Lurie Children's Hospital of Chicago
Investigators
Principal Investigator: Mitchell S Cairo, MD New York Medical College
More Information

Additional Information:
Responsible Party: Mitchell Cairo, Principal Investigator, New York Medical College
ClinicalTrials.gov Identifier: NCT01461837     History of Changes
Other Study ID Numbers: NYMC526-4090
FD-R-0004090 ( Other Grant/Funding Number: FDA OOPD )
First Posted: October 28, 2011    Key Record Dates
Last Update Posted: December 18, 2017
Last Verified: December 2017

Keywords provided by Mitchell Cairo, New York Medical College:
sickle cell disease
stem cell transplantation
haploidentical

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn