Status:
TERMINATED
Serum-Free Thymus Transplantation in DiGeorge Anomaly
Lead Sponsor:
Sumitomo Pharma Switzerland GmbH
Collaborating Sponsors:
National Institutes of Health (NIH)
National Institute of Allergy and Infectious Diseases (NIAID)
Conditions:
DiGeorge Anomaly
Eligibility:
All Genders
Up to 2 years
Phase:
PHASE1
Brief Summary
The study purpose is to determine if thymus tissue cultured in a serum-free (SF) solution is a safe and effective treatment for atypical and typical complete DiGeorge anomaly. \[Funding Source - FDA O...
Detailed Description
Complete DiGeorge anomaly is a congenital disorder characterized by athymia. Without successful treatment, patients remain immunodeficient and usually die by age 2 years. In "typical" complete DiGeorg...
Eligibility Criteria
Inclusion
- Thymus Recipients Inclusion:
- Complete DiGeorge anomaly diagnosis
- Must have one of following:
- congenital heart disease
- hypocalcemia requiring replacement
- 22q11 or 10p13 hemizygous
- CHARGE
- Atypical Arm:
- Must have, or have had, rash. If rash present, skin biopsy must show T cells. If rash resolved, must have \>50/cumm T cells; \& \<50/cumm naive T cells or \<5% total
- PHA response must be \<40000 counts per minute(cpm) on immunosuppression; or, \<75000cpm off immunosuppression. PHA test must be done 2x
- CD45RA+CD62L+ CD3+ T cells must be \<50/mm3; or, \<5% of total CD3. Test must be done 2x
- Typical Arm:
- PHA response \<20 fold or \<5,000cpm
- Circulating CD3+CD45RA+CD62L+T cells \<50/mm3 or \<5% total T cells
- 2 tests of T cells \& PHA response must show similar results
- Biological Mother Inclusion:
- Must be recipient's biological mother
- Thymus Recipient Exclusion:
- Heart surgery \<4 weeks pre-transplant or within 3 months post-transplant
- Rejection by surgeon or anesthesiologist as surgical candidates
- Lack of sufficient muscle tissue to accept transplant
- Medical condition does not allow to undergo a biopsy
- HIV
- CMV(\>500 copies/ml blood by PCR on 2 tests)
- Ventilator dependence
- GVHD
- Maternal T cells \>20% of total T cells
- Prior immune reconstitution attempts (e.g., BMT, prior thymus transplant)
- Hypoparathyroidism meeting criteria for combined thymus/parathyroid transplant \& parents desiring it
- RSV or parainfluenza virus
- Enterovirus or Adenovirus in stool
- Biological Mother Exclusion:
- Unwillingness to sign consent or provide blood/buccal samples
Exclusion
Key Trial Info
Start Date :
April 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2011
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT00849888
Start Date
April 1 2008
End Date
February 1 2011
Last Update
April 1 2022
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