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

NCT02277639 | Completed Phase 2 Results DMC

• 2 other identifiers: 11-008330CHP 980
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

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.

Conditions

Bone Marrow Failure Syndromes; Immunodeficiencies; Immune Dysregulation Syndromes

Keywords

Transplant related mortality; Graft versus host disease; Bone marrow transplant

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Engraftment

  The primary objective is to determine event free survival with durable stable engraftment of donor cells at one year.

  One Year

Study Arms (2)

Bone Marrow Failure Syndrome

EXPERIMENTAL

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

Immunodeficiency / Dysregulation

EXPERIMENTAL

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

Interventions

CliniMACs device DEVICE

Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.

Bone Marrow Failure Syndrome; Immunodeficiency / Dysregulation

Eligibility Criteria

• Age: Up to 22 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead

Study Sites (1)

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Bone Marrow Failure Disorders; Immunologic Deficiency Syndromes; Graft vs Host Disease

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Immune System Diseases

Results Point of Contact

• Title: Nancy Bunin, MD
• Organization: Children's Hospital of Philadelphia

buninn@email.chop.edu

215-590-2255

Study Officials

• Nancy Bunin, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: BMT Medical Director

Model Details: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.

Study Record Dates

• First Submitted: October 22, 2014
• First Posted: October 29, 2014
• Study Start: November 1, 2011
• Primary Completion: March 1, 2016
• Study Completion: March 1, 2016
• Last Updated: April 11, 2018
• Results First Posted: April 11, 2018

Record last verified: 2018-03

Locations

US (1)