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

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2016 by New York Medical College
Sponsor:
Collaborators:
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
Children's Hospital Medical Center, Cincinnati
Ann and Robert H. Lurie Children's Hospital of Chicago
Information provided by (Responsible Party):
Mitchell Cairo, New York Medical College
ClinicalTrials.gov Identifier:
NCT01461837
First received: October 19, 2011
Last updated: March 10, 2016
Last verified: March 2016

October 19, 2011
March 10, 2016
January 2012
April 2017   (final data collection date for primary outcome measure)
Treatment related events [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
Death, primary or late graft rejection, or recurrence of disease and acceptable rate of hematopoietic engraftment, acute and chronic graft-versus-host disease
Same as current
Complete list of historical versions of study NCT01461837 on ClinicalTrials.gov Archive Site
  • neurological/neurocognitive status [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Change from baseline in neurological/neurocognitive status
  • Pulmonary/pulmonary vascular status [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Change from baseline of Pulmonary/pulmonary vascular status
  • Health-related quality of life [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Change from baseline of Health-related quality of life
Same as current
Not Provided
Not Provided
 
Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease
Familial Haploidentical T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease (IND 14359)

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

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.

Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Sickle Cell Disease
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
Experimental: Haplo Stem Cell Transplantation
CD34 selected T-cell depleted allogeneic SCT
Intervention: Drug: CD34 selected T-cell depleted allogeneic SCT
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
35
April 2018
April 2017   (final data collection date for primary outcome measure)

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
Both
2 Years to 20 Years   (Child, Adult)
No
Contact: Mitchell S Cairo, MD 914-594-2150 mitchell_cairo@nymc.edu
Contact: Erin Morris, RN 714-964-5359 erin_morris@nymc.edu
United States
 
NCT01461837
NYMC526-4090, FD-R-0004090
Yes
Not Provided
Not Provided
Mitchell Cairo, New York Medical College
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
  • Children's Hospital Medical Center, Cincinnati
  • Ann and Robert H. Lurie Children's Hospital of Chicago
Principal Investigator: Mitchell S Cairo, MD New York Medical College
New York Medical College
March 2016

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