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Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases (MiniClini)

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ClinicalTrials.gov Identifier: NCT02277639
Recruitment Status : Completed
First Posted : October 29, 2014
Results First Posted : April 11, 2018
Last Update Posted : April 11, 2018
Sponsor:
Information provided by (Responsible Party):
Nancy Bunin, Children's Hospital of Philadelphia

Study Description
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Brief Summary:
This is a Phase II trial to determine the ability of a reduced intensity conditioning regimen to allow successful engraftment with CD3+ /CD19+ depleted peripheral stem cell grafts from mismatched donors. There are two conditioning regimens depending upon patient diagnosis and age.

Condition or disease Intervention/treatment Phase
Bone Marrow Failure Syndromes Immunodeficiencies Immune Dysregulation Syndromes Device: CliniMACs device Phase 2

Detailed Description:

This study will allow transplantation using a reduced intensity conditioning regimen for children with non-malignant diseases who lack a matched related or unrelated donor. Donors will be unrelated or partially matched related, depending upon urgency and availability. If each parent is haploidentical, the mother will be preferred, as there is evidence of reduced transplant related mortality and superior survival with a maternal donor. The risks of severe graft vs host disease (GVHD) and Epstein-Barr lymphoproliferative disorder will be reduced or eliminated by T and B cell depletion using the Miltenyi Clinimacs device. Patients with bone marrow failure syndromes, who are at high risk for rejection, will undergo pre-conditioning immune suppression with Thymoglobulin. It is recommended that patients with immunedysregulation syndromes receive pre-RIC alemtuzumab as this may reduce the risk on non-engraftment and hyperinflammatory states.

Post-transplant immune suppression will be used to prevent GVHD, as CD3 depletion does not deplete as completely as CD34+ selection. It will be rapidly weaned if no GVHD by day 100 to allow immune reconstitution.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I/II Study of Reduced Intensity Conditioning for Patients With Non-Malignant Diseases Using CD3+/CD19+ Depleted Unrelated Donor or Partially Matched Related Donor Peripheral Stem Cells
Study Start Date : November 2011
Actual Primary Completion Date : March 2016
Actual Study Completion Date : March 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Organ Donation

Arms and Interventions
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Arm Intervention/treatment
Experimental: Bone Marrow Failure Syndrome
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.
 Device: CliniMACs device
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
Experimental: Immunodeficiency / Dysregulation
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.
 Device: CliniMACs device
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Engraftment [ Time Frame: One Year ]
    The primary objective is to determine event free survival with durable stable engraftment of donor cells at one year.


Eligibility Criteria
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Ages Eligible for Study:   up to 22 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Bone marrow failure syndromes for which SCT is indicated, including severe aplastic anemia refractory to non transplant therapies congenital neutropenia, congenital thrombocytopenia, congenital red cell aplasia
  • Immunodeficiencies for which allogeneic hematopoietic stem cell transplant is indicated, including severe combined immunodeficiencies, Wiskott-Aldrich syndrome, IPEX syndrome, X-linked lymphoproliferative disease
  • Immune dysregulation syndromes, including refractory or recurrent hemophagocytic lymphohistiocytosis, HLH with genetic mutations, refractory multisystemic Langerhans cell histiocytosis, other MAS refractory to standard therapy
  • Organ function clearance

Exclusion Criteria:

  • Uncontrolled bacterial, viral or fungal infections
  • HLA matched related or unrelated donor able to donate mobilized peripheral stem cells.
  • Fanconi's syndrome, dyskeratosis congenita or other chromosomal fragility syndromes
  • Pregnant Females
Contacts and Locations
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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): NCT02277639


Locations
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United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Children's Hospital of Philadelphia
Investigators
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Principal Investigator: Nancy Bunin, MD Children's Hospital of Philadelphia
More Information
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Responsible Party: Nancy Bunin, BMT Medical Director, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT02277639    
Other Study ID Numbers: 11-008330
CHP 980 ( Other Identifier: Children's Hospital of Philadelphia )
First Posted: October 29, 2014    Key Record Dates
Results First Posted: April 11, 2018
Last Update Posted: April 11, 2018
Last Verified: March 2018
Keywords provided by Nancy Bunin, Children's Hospital of Philadelphia:
Transplant related mortality
Graft versus host disease
Bone marrow transplant
Additional relevant MeSH terms:
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Bone Marrow Failure Disorders
Pancytopenia
Syndrome
Disease
 Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases