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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 : March 13, 2019
Sponsor:
Collaborators:
UCSF Benioff Children’s Hospital 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
Lurie Children's Hospital of Chicago
Information provided by (Responsible Party):
Mitchell Cairo, New York Medical College

Tracking Information
First Submitted Date  ICMJE October 19, 2011
First Posted Date  ICMJE October 28, 2011
Last Update Posted Date March 13, 2019
Study Start Date  ICMJE January 2012
Estimated Primary Completion Date June 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 26, 2011)
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
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01461837 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 26, 2011)
  • neurological/neurocognitive status [ Time Frame: 2 years ]
    Change from baseline in neurological/neurocognitive status
  • Pulmonary/pulmonary vascular status [ Time Frame: 2 years ]
    Change from baseline of Pulmonary/pulmonary vascular status
  • Health-related quality of life [ Time Frame: 2 years ]
    Change from baseline of Health-related quality of life
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease
Official Title  ICMJE Familial Haploidentical T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease (IND 14359)
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

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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE 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
Study Arms  ICMJE Experimental: Haplo Stem Cell Transplantation
CD34 selected T-cell depleted allogeneic SCT
Intervention: Drug: CD34 selected T-cell depleted allogeneic SCT
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 20, 2016)
20
Original Estimated Enrollment  ICMJE
 (submitted: October 26, 2011)
35
Estimated Study Completion Date  ICMJE December 2019
Estimated Primary Completion Date June 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 20 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Mitchell S Cairo, MD 914-594-2150 mitchell_cairo@nymc.edu
Contact: Erin Morris, RN 714-964-5359 erin_morris@nymc.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01461837
Other Study ID Numbers  ICMJE NYMC526-4090
FD-R-0004090 ( Other Grant/Funding Number: FDA OOPD )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Mitchell Cairo, New York Medical College
Study Sponsor  ICMJE New York Medical College
Collaborators  ICMJE
  • UCSF Benioff Children’s Hospital 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
  • Lurie Children's Hospital of Chicago
Investigators  ICMJE
Principal Investigator: Mitchell S Cairo, MD New York Medical College
PRS Account New York Medical College
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP