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

View this study on Beta.ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Nancy Bunin, Children's Hospital of Philadelphia

Study Type	Interventional
Study Design	Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Conditions	Bone Marrow Failure Syndromes Immunodeficiencies Immune Dysregulation Syndromes
Intervention	Device: CliniMACs device
Enrollment	2

Participant Flow

Recruitment Details
Pre-assignment Details


Arm/Group Title	Bone Marrow Failure Syndrome	Immunodeficiency / Dysregulation
Arm/Group Description	Reduced intensity conditioning with...	Reduced intensity conditioning with...
Arm/Group Description	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. CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.	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. CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
Period Title: Overall Study
Started	0	2
Completed	0	2
Not Completed	0	0

Baseline Characteristics

Arm/Group Title		Bone Marrow Failure Syndrome	Immunodeficiency / Dysregulation	Total
Arm/Group Description		Reduced intensity conditioning with...	Reduced intensity conditioning with...	Total of all reporting groups
Arm/Group Description		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. CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.	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. CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.	Total of all reporting groups
Overall Number of Baseline Participants		0	2	2
Baseline Analysis Population Description		[Not Specified]
Baseline Analysis Population Description		[Not Specified]
Age, Continuous Median (Full Range) Unit of measure: Years
	Number Analyzed	0 participants	2 participants	2 participants
			9.5 (4 to 15)	9.5 (4 to 15)
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	0 participants	2 participants	2 participants
	Female		1 50.0%	1 50.0%
	Male		1 50.0%	1 50.0%

Outcome Measures

1.Primary Outcome


Title	Number of Participants With Engraftment
Description	The primary objective is to determine event free survival with durable...
Description	The primary objective is to determine event free survival with durable stable engraftment of donor cells at one year.
Time Frame	One Year

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Bone Marrow Failure Syndrome	Immunodeficiency / Dysregulation
Arm/Group Description:	Reduced intensity conditioning with...	Reduced intensity conditioning with...
Arm/Group Description:	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. CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.	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. CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
Overall Number of Participants Analyzed	0	2
Measure Type: Count of Participants Unit of Measure: Participants
		2 100.0%

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]

Arm/Group Title	Bone Marrow Failure Syndrome	Immunodeficiency / Dysregulation
Arm/Group Description	Reduced intensity conditioning with...	Reduced intensity conditioning with...
Arm/Group Description	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. CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.	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. CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
All-Cause Mortality
	Bone Marrow Failure Syndrome	Immunodeficiency / Dysregulation
	Affected / at Risk (%)	Affected / at Risk (%)
Total	0/0	0/2 (0.00%)
Serious Adverse Events Serious Adverse Events
	Bone Marrow Failure Syndrome	Immunodeficiency / Dysregulation
	Affected / at Risk (%)	Affected / at Risk (%)
Total	0/0	0/2 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Bone Marrow Failure Syndrome	Immunodeficiency / Dysregulation
	Affected / at Risk (%)	Affected / at Risk (%)
Total	0/0	0/2 (0.00%)

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	Nancy Bunin, MD
Organization:	Children's Hospital of Philadelphia
Phone:	215-590-2255
EMail:	buninn@email.chop.edu

Layout table for additonal information

Responsible Party:	Nancy Bunin, 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 Submitted:	October 22, 2014
First Posted:	October 29, 2014
Results First Submitted:	June 26, 2017
Results First Posted:	April 11, 2018
Last Update Posted:	April 11, 2018

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