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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT01220531
Date of registration: 22/09/2010
Prospective Registration: Yes
Primary sponsor: M. Louise Markert
Public title: Thymus Transplantation Safety-Efficacy
Scientific title: Safety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836
Date of first enrolment: September 2010
Target sample size:
Recruitment status: Available
URL:  https://clinicaltrials.gov/show/NCT01220531
Study type:  Expanded Access
Study design:   
Phase: 
Countries of recruitment
United States
Contacts
Name:     M. Louise Markert, M.D., Ph.D
Address: 
Telephone: 919-684-6263
Email: marke001@mc.duke.edu
Affiliation: 
Name:     M. Louise Markert, M.D., Ph.D
Address: 
Telephone: 919-684-6263
Email: marke001@mc.duke.edu
Affiliation: 
Name:     M. Louise Markert, M.D., Ph.D
Address: 
Telephone:
Email:
Affiliation:  Duke University Medical Center, Pediatrics, Allergy & Immunology
Key inclusion & exclusion criteria

Transplant Inclusion:

- Must have 1 of following: 22q11 or 10p13 hemizygosity; hypocalcemia requiring
replacement; congenital heart disease; or CHARGE syndrome or CHD7 mutation

- Complete DiGeorge: <50 CD3+ T cells/cumm or <50 CD3+ T cells/cumm that are CD62L+
CD45RA+, or <5% of CD3+ cells are CD62L+ CD45RA+

- Atypical DiGeorge subjects must have, or have had, a rash.

Group 1

•Typical cDGA whose T cells have a PHA response < 5,000 cpm and < 20 fold PHA response.

Group 2

•Typical cDGA whose T cells have a PHA response >5,000 cpm and <50,000 cpm and >20 fold PHA
response

Group 3

- Typical cDGA whose T cells have PHA response >50,000 cpm

- Typical cDGA with maternal engraftment

- Atypical cDGA whose T cells have PHA response <40,000 cpm when on immunosuppression or
<75,000 cpm to PHA when not on immunosuppression

- Atypical cDGA with group 3 PHA response & maternal engraftment

Group 4

- Atypical cDGA with PHA responses >75,000cpm while on no immunosuppression or PHA
responses >40,000cpm while on immunosuppression

- Atypical cDGA with maternal engraftment and group 4 PHA response

Transplant Exclusion:

- Heart surgery <4 wks pre-transplantation

- Heart surgery anticipated w/in 3 months after proposed transplantation

- Rejection by surgeon or anesthesiologist as surgical candidate

- Lack of sufficient muscle tissue to accept transplant of 2,000 mm2/m2 body surface
area

- HIV infection

- Prior attempts at immune reconstitution, such as bone marrow transplant or previous
thymus transplant

- CMV on 2 tests for Groups 2, 3, and 4



Age minimum: N/A
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Complete DiGeorge Anomaly
Complete DiGeorge Syndrome
DiGeorge Anomaly
DiGeorge Syndrome
Intervention(s)
Biological: Thymus Tissue for Transplantation
Drug: Basiliximab
Drug: Cyclosporine
Drug: Methylprednisolone or Prednisolone
Drug: Mycophenolate mofetil
Drug: Rabbit anti-thymocyte globulin
Drug: Tacrolimus
Procedure: Blood Draw
Primary Outcome(s)
Secondary Outcome(s)
Secondary ID(s)
2R01AI047040-11A2
5K12HD043494-09
Pro00025966
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Enzyvant Therapeutics GmbH
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
National Institute of Allergy and Infectious Diseases (NIAID)
National Institutes of Health (NIH)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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