Thymus Transplantation With Immunosuppression

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00579709
• Date of registration: 20/12/2007
• Prospective Registration: No
• Primary sponsor: M. Louise Markert
• Public title: Thymus Transplantation With Immunosuppression 884
• Scientific title: Thymus Transplantation With Immunosuppression, #884
• Date of first enrolment: July 2002
• Target sample size: 15
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00579709
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: M. Louise Markert, MD, PhD
• Affiliation: Duke University Medical Center, Pediatrics, Allergy & Immunology

Key inclusion & exclusion criteria

Transplant Inclusion

- No age limit

- Thyroid studies must be done and if abnormal, must be on therapy

DiGeorge diagnosis - must have 1 symptom from the following list:

- Heart defect

- Hypocalcemia requiring replacement

- 22q11 hemizygosity

- 10p13 hemizygosity

- CHARGE association

- Abnormal ears plus mother with diabetes (type I, type II, or gestational)

Atypical Diagnosis:

- Must have, or have had, a rash. If rash present, biopsy of rash must show T cells in
skin. If rash & adenopathy resolved, must still have oligoclonal T cells.

- Within 1 month of tx must have PHA response >20 fold above background or >5,000 cpm,
whichever is higher, or response can be < this.

- Circulating CD3+ T cells >50/mm3 but CD45RA+CD62L+CD3+ T cells <50/mm or <5% of CD3
count, whichever is higher (must be done 2x)

- Immunoscope with >40% oligoclonal TCRBV families. A 2nd test per sponsor discretion if
T cell numbers increase or activation status changes.

- If TREC done pre-tx must have TRECs <100 per 100,000 CD3+ cells.

Typical Diagnosis:

- Circulating CD3+ CD45RA+ CD62L+ T cells and <50/mm3 or <5% of total T cells

- PHA response >20 fold above background or >5,000 cpm, whichever is higher.

- 2 studies must show similar immunological findings qualify for this study.

- TRECs, if done, should be <100/100,000 CD3 cells

Transplant Exclusion:

- Heart surgery <4 weeks pre-tx date

- Heart surgery anticipated w/in 3 months of proposed tx

- Rejection by surgeon or anesthesiologist as surgical candidates

- Lack of sufficient muscle tissue to accept 0.2 grams/kg transplant



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Complete DiGeorge Syndrome

Complete DiGeorge Anomaly

DiGeorge Syndrome

DiGeorge Anomaly

Intervention(s)

Biological: Thymus Tissue for Transplantation

Primary Outcome(s)

Safety & tolerability of Thymoglobulin and cyclosporine followed by thymus transplantation: Survival at 1 year post-transplantation. [Time Frame: 1 year post-transplantation]

Secondary Outcome(s)

Allograft biopsy used to evaluate graft rejection [Time Frame: 2 to 4 months post-transplant]

CD3 count [Time Frame: 10 - 14 months post-transplantation]

Use of additional post transplant immunosuppression after that listed in the protocol. [Time Frame: The post thymus transplantation period]

CD8 count [Time Frame: 10-14 months after thymus transplantation]

Thymopoiesis [Time Frame: 2-4 months after thymus transplantation]

CD4 count [Time Frame: 10-14 months after thymus transplantation]

naive CD4 count [Time Frame: 10-14 months after thymus transplantation]

naive CD8 count [Time Frame: 10-14 months after thymus transplantation]

Secondary ID(s)

R56 Bridge R01AI4704011A1

#884

2R01AI047040-11A2

R01AI054843

3R56AI047040-11A1S1

Pro00013734

5K12HD043494-09

R01AI047040

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Enzyvant Therapeutics GmbH

National Institute of Allergy and Infectious Diseases (NIAID)

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

National Institutes of Health (NIH)