DiGeorge AnomalyPhase 2NCT00576836

Thymus Transplantation Dose in DiGeorge #932

A Phase 2 study testing Cultured Thymus Tissue Implantation (CTTI) and Cultured Thymus Tissue Implantation with Parathyroid Transplantation in people with DiGeorge Anomaly and DiGeorge Syndrome, run by Sumitomo Pharma Switzerland GmbH at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

One purpose of this study is to determine whether the amount of cultured thymus tissue implanted into DiGeorge anomaly infants has any effect on the immune outcome. Another purpose of this study is to determine whether parental parathyroid transplantation (in addition to cultured thymus tissue implantation (CTTI) can… Read more about this study

Who is it for?: Any age
Conditions the study is for: DiGeorge Anomaly, DiGeorge Syndrome, Complete DiGeorge Anomaly
What is being tested?: Cultured Thymus Tissue Implantation (CTTI) and Cultured Thymus Tissue Implantation with Parathyroid Transplantation
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 7 participants
Where: 1 siteUnited States
Study dates: 2 Sep 2004to 31 Dec 2019
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

None listed.

Who cannot join

Parents do not meet enrollment criteria.
Parent(s) decline to be parathyroid donor(s).
Parental Parathyroid Donor Inclusion:
> 18 years old
Answers all questionnaire items and meets safety screening criteria
Normal serum calcium

Show all 39Show fewer

Normal PTH function
HLA typing consistent with parentage
Parent chosen for donation will share HLA-DR allele in thymus donor; if not applicable, then either parent will be selected (if meet all other criteria).
Must not be on anticoagulation or can come off for donation/transplantation
Parental Parathyroid Donor Exclusion:
Donor is only living involved parent or caretaker of the recipient
Hypoparathyroidism - low parathyroid hormone (PTH) in presence of low serum calcium and high serum phosphate
Hyperparathyroidism (or history of) - elevated PTH in presence of high serum calcium and low serum phosphate
History of cancer
Evidence of any of following: HIV-1, HIV-2, HTLV-1, HTLV-2, syphilis, hepatitis B, hepatitis C, West Nile virus, or Trypanosoma Cruzi (Chagas disease)
Elevated AST, ALT, alkaline phosphatase > 3 times upper limit of normal
History including receipt of a xenograft or risk factors for SARS, Mad Cow - Disease or smallpox. Note: if parent has Mad Cow Disease risk factors (but not active disease), parent(s) may give permission for transplantation.
CMV positive urine
Positive CMV IgM antibodies
Positive IgM anti-EBV VCA
On blood thinners and cannot stop for the parathyroid donation
Elevated PT or PTT (> ULN)
Platelets < 100,000
Positive Toxoplasma IgM
The donor will receive a history and physical; may be excluded based on PI's medical judgment
Hemoglobin < 9 g/dl
Infectious lesion on head or neck
Goiter on ultrasound
Abnormal fiberoptic laryngoscopy of vocal cords
Pregnancy
Positive HSV IgG is not an exclusion; however, post transplantation prophylaxis is needed
Positive VZV IgG is not an exclusion; however, post transplantation prophylaxis is needed
Medical concern of otolaryngologist
Concern by medical psychologist or social worker. Parents are interviewed together and separately regarding following areas: medical history; health habits; substance use; relationships and support; education/work history; mental status/psychological history; readiness for donation.
Questionnaire (safety screening) responses can lead to exclusion.
Biological Mother of DiGeorge Subject Inclusion Criteria:
Competent to provide consent
Willing to provide blood for testing (No other inclusion/exclusion for mother)

Trial locations and contacts

1 site in 1 country.

United States1 site

Duke University Medical Center
Durham, North Carolina, 27710

Other trials in United States

About this study

Official title: Dose Study of Thymus Transplantation in DiGeorge Anomaly, IND 9836, #932.1

Read the detailed description

DiGeorge anomaly is a congenital disorder in which infants are born with defects of the thymus, heart, and parathyroid gland. Complete DiGeorge Anomaly is usually fatal within the first two years of life. This trial evaluates the role of cultured thymus tissue dose in cultured thymus tissue implantation (CTTI) in complete (typical) DiGeorge anomaly infants, and continues safety assessments.

DiGeorge infants who have successful CTTIs but remain with hypoparathyroidism must go to the clinic for frequent calcium levels and to the hospital for calcium infusions; these infants are at risk for seizures from low calcium. Approximately ½ of infants with profound hypoparathyroidism will develop nephrocalcinosis. This protocol had a parental parathyroid transplant arm for complete DiGeorge infants with athymia and profound hypoparathyroidism.

Conditions: DiGeorge Anomaly, DiGeorge Syndrome, Complete DiGeorge Anomaly, Complete DiGeorge Syndrome
Being tested: Cultured Thymus Tissue Implantation (CTTI), Cultured Thymus Tissue Implantation with Parathyroid Transplantation
Keywords: Thymus Transplantation, DiGeorge Syndrome, DiGeorge Anomaly, Athymia, Parathyroid Transplantation, Hypocalcemia, Hypoparathyroidism, Low T cell numbers, Immunoreconstitution, Immunodeficiency, Complete DiGeorge, Complete DiGeorge Anomaly, Typical DiGeorge, Cultured Thymus Tissue Implantation (CTTI)
Medical subject headings: DiGeorge Syndrome, Thymic aplasia, Hypocalcemia, Hypoparathyroidism, Immunologic Deficiency Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Cultured Thymus Tissue Implantation (CTTI)Biological
Also called Thymus Tissue for Transplantation, CTTI
Thymus tissue (from unrelated donor), thymus donor, and thymus donor's birth mother screened for safety. CTTI was done under general anesthesia. Cultured thymus tissue was implanted into quadriceps. Thymus dose at least 4grams/m2 body surface area (0.2 grams/kg body weight) and not >18 grams/m2 body surface area (1.0 grams/kg body weight). At time of CTTI, skin biopsy was obtained to look for preexisting T cells. 2-3 months post-CTTI allograft biopsy was done to evaluate for thymopoiesis \& graft rejection. At time of biopsy, skin biopsy done to look for T cell clonal populations. (Allograft biopsy not done if subject medically unstable.) Post-CTTI, subjects followed by immune evaluations, using blood samples.
Cultured Thymus Tissue Implantation with Parathyroid TransplantationOther
Also called Thymus and Parathyroid Transplant, CTTI and Parathyroid Transplant
Parental parathyroid donors screened for eligibility and safety. If both parents meet eligibility criteria, the parathyroid will be harvested from parent who shares the most Human Leukocyte Antigens (HLA) alleles with thymus donor. Parathyroid harvest \& transplant preferably done at same time as CTTI. (If parathyroid transplant cannot be done at same time, then it is done within 3-8 weeks of CTTI.) Parathyroid harvest done under general anesthesia. One parathyroid gland is minced \& placed in quadriceps muscle; there is no dose in mg. No biopsy done of the parathyroid. Parathyroid donors are monitored as outpatients until recipients' discharge. Recipients' calcium and PTH levels are monitored indefinitely.

Study groups in NCT00576836
Study group	Role	What they receive
Cultured Thymus Tissue Implantation w Parathyroid Transplant	Experimental	Cultured Thymus Tissue Implantation With Parathyroid Tissue Transplantation. Subjects who were enrolled in this arm underwent cultured thymus tissue implantation with parathyroid transplantation, if eligible. No specific dose was assigned. The thymus tissue dose was the number of grams of cultured thymus tissue divided by the weight of the recipient in kg or per square meter of body surface area of the recipient. There was a one time administration of the cultured thymus tissue and parathyroid tissue. Receives: Other: Cultured Thymus Tissue Implantation with Parathyroid Transplantation
Cultured Thymus Tissue Implantation	Experimental	Cultured Thymus Tissue Implantation. Subjects who were enrolled in this arm underwent cultured thymus tissue implantation (CTTI) only. No specific dose was assigned. The thymus tissue dose was the number of grams of cultured thymus tissue divided by the weight of the recipient in kg or per square meter of body surface area of the recipient. There was a one time administration of the cultured thymus tissue. . Receives: Biological: Cultured Thymus Tissue Implantation (CTTI)

What is being measured

The primary outcome is the main question the study is designed to answer.

Survival at 1 Year Post-CTTI
Survival at 1 year post CTTI was assessed using the Kaplan Meier Estimated Survival. This mathematical function estimates the survival for a certain length of time.
Measured: 1 year post-CTTI

Secondary outcomes (8)

Survival at 2 Years Post-CTTI
Survival at 2 years post CTTI was assessed using the Kaplan Meier Estimated Survival. This mathematical function estimates the survival for a certain length of time.
Measured: 2 years post-CTTI
Immune Reconstitution Efficacy - CD3 T Cells
The development of total CD3 T cells at one year as measured using flow cytometry
Measured: 1 year post-CTTI
Immune Reconstitution Efficacy - CD4 T Cells
The development of total CD4 T cells at one year as measured using flow cytometry
Measured: 1 year post-CTTI
Immune Reconstitution Efficacy - CD8 T Cells
The development of total CD8 T cells at one year as measured using flow cytometry
Measured: 1 year post-CTTI
Immune Reconstitution Efficacy - Naive CD4 T Cells
The development of naive CD4 T cells at one year as measured using flow cytometry
Measured: 1 year post-CTTI
Immune Reconstitution Efficacy - Naive CD8 T Cells
The development of naïve CD8 T cells at one year as measured using flow cytometry.
Measured: 1 year post-CTTI
Immune Reconstitution Efficacy - Response to Mitogens
The development of a T cell proliferative response to the mitogen phytohemagglutinin.
Measured: 1 year post-CTTI
Thymus Allograft Biopsy
Evidence, on biopsy of the thymus tissue implanted in the recipient muscle, that shows the development of new T cells.
Measured: 2 to 3 months post-CTTI

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

2 Sep 200431 Dec 2019

First posted19 Dec 2007 (estimated)
Study start2 Sep 2004
Primary completionNov 2010
Study completion31 Dec 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (14)Papers the sponsor has linked to this study.
Other trials for DiGeorge AnomalyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 7 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00576836
Sponsor's protocol ID: Pro00016144
Other IDs: FDA-FD-R-002606, 2R01AI047040-11A2, R56 Bridge R01AI4704011A1, 5K12HD043494-09, R01AI054843, R01AI047040, 3R56AI047040-11A1S1, 932
Status last verified: Mar 2022
First submitted: 17 Dec 2007
Last updated: 25 Mar 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Chinn IK, Devlin BH, Li YJ, Markert ML. Long-term tolerance to allogeneic thymus transplants in complete DiGeorge anomaly. Clin Immunol. 2008 Mar;126(3):277-81. doi: 10.1016/j.clim.2007.11.009. Epub 2007 Dec 26.
PubMed 18155964
Markert ML, Sarzotti M, Ozaki DA, Sempowski GD, Rhein ME, Hale LP, Le Deist F, Alexieff MJ, Li J, Hauser ER, Haynes BF, Rice HE, Skinner MA, Mahaffey SM, Jaggers J, Stein LD, Mill MR. Thymus transplantation in complete DiGeorge syndrome: immunologic and safety evaluations in 12 patients. Blood. 2003 Aug 1;102(3):1121-30. doi: 10.1182/blood-2002-08-2545. Epub 2003 Apr 17.
PubMed 12702512
Chinn IK, Milner JD, Scheinberg P, Douek DC, Markert ML. Thymus transplantation restores the repertoires of forkhead box protein 3 (FoxP3)+ and FoxP3- T cells in complete DiGeorge anomaly. Clin Exp Immunol. 2013 Jul;173(1):140-9. doi: 10.1111/cei.12088.
PubMed 23607606
Chinn IK, Olson JA, Skinner MA, McCarthy EA, Gupton SE, Chen DF, Bonilla FA, Roberts RL, Kanariou MG, Devlin BH, Markert ML. Mechanisms of tolerance to parental parathyroid tissue when combined with human allogeneic thymus transplantation. J Allergy Clin Immunol. 2010 Oct;126(4):814-820.e8. doi: 10.1016/j.jaci.2010.07.016. Epub 2010 Sep 15.
PubMed 20832849
Markert ML, Devlin BH, Alexieff MJ, Li J, McCarthy EA, Gupton SE, Chinn IK, Hale LP, Kepler TB, He M, Sarzotti M, Skinner MA, Rice HE, Hoehner JC. Review of 54 patients with complete DiGeorge anomaly enrolled in protocols for thymus transplantation: outcome of 44 consecutive transplants. Blood. 2007 May 15;109(10):4539-47. doi: 10.1182/blood-2006-10-048652. Epub 2007 Feb 6.
PubMed 17284531
Markert ML, Devlin BH, McCarthy EA. Thymus transplantation. Clin Immunol. 2010 May;135(2):236-46. doi: 10.1016/j.clim.2010.02.007. Epub 2010 Mar 16.
PubMed 20236866
Markert ML, Devlin BH, Chinn IK, McCarthy EA, Li YJ. Factors affecting success of thymus transplantation for complete DiGeorge anomaly. Am J Transplant. 2008 Aug;8(8):1729-36. doi: 10.1111/j.1600-6143.2008.02301.x. Epub 2008 Jun 28.
PubMed 18557726
Markert ML and Devlin BH. Thymic reconstitution (in Rich RR, Shearer WT, Fleischer T, Schroeder HW, Weyand CM, Frew A, eds., Clinical Immunology 3rd edn., Elsevier, Edinburgh) p 1253-1262, 2008.
Markert ML, Li J, Devlin BH, Hoehner JC, Rice HE, Skinner MA, Li YJ, Hale LP. Use of allograft biopsies to assess thymopoiesis after thymus transplantation. J Immunol. 2008 May 1;180(9):6354-64. doi: 10.4049/jimmunol.180.9.6354.
PubMed 18424759
Hudson LL, Louise Markert M, Devlin BH, Haynes BF, Sempowski GD. Human T cell reconstitution in DiGeorge syndrome and HIV-1 infection. Semin Immunol. 2007 Oct;19(5):297-309. doi: 10.1016/j.smim.2007.10.002. Epub 2007 Nov 26.
PubMed 18035553
Markert ML, Devlin BH, McCarthy EA, Chinn IK, Hale LP. Thymus Transplantation in Thymus Gland Pathology: Clinical, Diagnostic, and Therapeutic Features. Eds Lavinin C, Moran CA, Morandi U, Schoenhuber R. Springer-Verlag Italia, Milan, 2008, pp 255-267.
Markert ML, Devlin BH, Chinn IK, McCarthy EA. Thymus transplantation in complete DiGeorge anomaly. Immunol Res. 2009;44(1-3):61-70. doi: 10.1007/s12026-008-8082-5.
PubMed 19066739
Li B, Li J, Devlin BH, Markert ML. Thymic microenvironment reconstitution after postnatal human thymus transplantation. Clin Immunol. 2011 Sep;140(3):244-59. doi: 10.1016/j.clim.2011.04.004. Epub 2011 Apr 16.
PubMed 21565561
Ciupe SM, Devlin BH, Markert ML, Kepler TB. Quantification of total T-cell receptor diversity by flow cytometry and spectratyping. BMC Immunol. 2013 Aug 6;14:35. doi: 10.1186/1471-2172-14-35.
PubMed 23914737

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All DiGeorge Anomaly trials

Study at a glance

From the registry record, last updated by the sponsor on 25 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Dose Study of Thymus Transplantation in DiGeorge Anomaly, IND 9836, #932.1
ClinicalTrials.gov ID: NCT00576836
Acronym / sponsor ID: Pro00016144
Conditions: DiGeorge Anomaly, DiGeorge Syndrome, Complete DiGeorge Anomaly, Complete DiGeorge Syndrome
Keywords: Thymus Transplantation, DiGeorge Syndrome, DiGeorge Anomaly, Athymia, Parathyroid Transplantation, Hypocalcemia, Hypoparathyroidism, Low T cell numbers
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 7 participants (actual)
Lead sponsor: Sumitomo Pharma Switzerland GmbHIndustry (pharma, biotech, devices)
Collaborators: National Institutes of Health (NIH), National Institute of Allergy and Infectious Diseases (NIAID), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Study start: 2 Sep 2004 (actual)
Primary completion: Nov 2010 (actual)
Study completion: 31 Dec 2019 (actual)
First posted: 19 Dec 2007 (estimated)
Results: Posted 17 Feb 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Cultured Thymus Tissue Implantation w Parathyroid Transplant	Experimental	Other: Cultured Thymus Tissue Implantation with Parathyroid TransplantationCultured Thymus Tissue Implantation With Parathyroid Tissue Transplantation. Subjects who were enrolled in this arm underwent cultured thymus tissue implantation with parathyroid transplantation, if eligible. No specific dose was assigned. The thymus tissue dose was the number of grams of cultured thymus tissue divided by the weight of the recipient in kg or per square meter of body surface area of the recipient. There was a one time administration of the cultured thymus tissue and parathyroid tissue.
Cultured Thymus Tissue Implantation	Experimental	Biological: Cultured Thymus Tissue Implantation (CTTI)Cultured Thymus Tissue Implantation. Subjects who were enrolled in this arm underwent cultured thymus tissue implantation (CTTI) only. No specific dose was assigned. The thymus tissue dose was the number of grams of cultured thymus tissue divided by the weight of the recipient in kg or per square meter of body surface area of the recipient. There was a one time administration of the cultured thymus tissue. .

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (0 listed)

Key exclusion criteria (39 listed)

Parents do not meet enrollment criteria.
Parent(s) decline to be parathyroid donor(s).
> 18 years old
Answers all questionnaire items and meets safety screening criteria
Normal serum calcium

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Cultured Thymus Tissue Implantation (CTTI)Also: Thymus Tissue for Transplantation, CTTIThymus tissue (from unrelated donor), thymus donor, and thymus donor's birth mother screened for safety. CTTI was done under general anesthesia. Cultured thymus tissue was implanted into quadriceps. Thymus dose at least 4grams/m2 body surface area (0.2 grams/kg body weight) and not >18 grams/m2 body surface area (1.0 grams/kg body weight). At time of CTTI, skin biopsy was obtained to look for preexisting T cells. 2-3 months post-CTTI allograft biopsy was done to evaluate for thymopoiesis \& graft rejection. At time of biopsy, skin biopsy done to look for T cell clonal populations. (Allograft biopsy not done if subject medically unstable.) Post-CTTI, subjects followed by immune evaluations, using blood samples.	Biological	Cultured Thymus Tissue Implantation
Cultured Thymus Tissue Implantation with Parathyroid TransplantationAlso: Thymus and Parathyroid Transplant, CTTI and Parathyroid TransplantParental parathyroid donors screened for eligibility and safety. If both parents meet eligibility criteria, the parathyroid will be harvested from parent who shares the most Human Leukocyte Antigens (HLA) alleles with thymus donor. Parathyroid harvest \& transplant preferably done at same time as CTTI. (If parathyroid transplant cannot be done at same time, then it is done within 3-8 weeks of CTTI.) Parathyroid harvest done under general anesthesia. One parathyroid gland is minced \& placed in quadriceps muscle; there is no dose in mg. No biopsy done of the parathyroid. Parathyroid donors are monitored as outpatients until recipients' discharge. Recipients' calcium and PTH levels are monitored indefinitely.	Other	Cultured Thymus Tissue Implantation w Parathyroid Transplant

Primary outcome

Survival at 1 Year Post-CTTI
Time frame: 1 year post-CTTI
Survival at 1 year post CTTI was assessed using the Kaplan Meier Estimated Survival. This mathematical function estimates the survival for a certain length of time.

8 secondary outcomes

Survival at 2 Years Post-CTTI
Time frame: 2 years post-CTTI
Immune Reconstitution Efficacy - CD3 T Cells
Time frame: 1 year post-CTTI
Immune Reconstitution Efficacy - CD4 T Cells
Time frame: 1 year post-CTTI
Immune Reconstitution Efficacy - CD8 T Cells
Time frame: 1 year post-CTTI
Immune Reconstitution Efficacy - Naive CD4 T Cells
Time frame: 1 year post-CTTI
Immune Reconstitution Efficacy - Naive CD8 T Cells
Time frame: 1 year post-CTTI
Immune Reconstitution Efficacy - Response to Mitogens
Time frame: 1 year post-CTTI
Thymus Allograft Biopsy
Time frame: 2 to 3 months post-CTTI

Results and safety

Results posted to the registry on 17 Feb 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Cultured Thymus Tissue Implantation w Parathyroid Transplant	0	0
Cultured Thymus Tissue Implantation	7	5

Primary outcome: Survival at 1 Year Post-CTTI

Time frame: 1 year post-CTTI. Number (95% Confidence Interval). Unit: % of participants who survive to 1 year

Measure	Cultured Thymus Tissue Implantation n = 6
Value	83 (27.3 to 97.5)

Population analysed: Analysis includes cDGA participants. After CTTI, 1 subject was determined to have SCID and not cDGA. Efficacy analysis as reported is on cDGA, without the SCID subject as CTTI cannot lead to T cell development in SCID. No subjects were enrolled into Arm 1. No subjects received parathyroid transplant. Therefore, results are reported for Arm 2 only.

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: Two years post-CTTI. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cultured Thymus Tissue Implantation	6 of 7 (85.7%)	7 of 7 (100%)	1 of 7

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 18155964Chinn IK, Devlin BH, Li YJ, Markert ML. Long-term tolerance to allogeneic thymus transplants in complete DiGeorge anomaly. Clin Immunol. 2008 Mar;126(3):277-81. doi: 10.1016/j.clim.2007.11.009. Epub 2007 Dec 26.
Background: PubMed 12702512Markert ML, Sarzotti M, Ozaki DA, Sempowski GD, Rhein ME, Hale LP, Le Deist F, Alexieff MJ, Li J, Hauser ER, Haynes BF, Rice HE, Skinner MA, Mahaffey SM, Jaggers J, Stein LD, Mill MR. Thymus transplantation in complete DiGeorge syndrome: immunologic and safety evaluations in 12 patients. Blood. 2003 Aug 1;102(3):1121-30. doi: 10.1182/blood-2002-08-2545. Epub 2003 Apr 17.
Background: PubMed 23607606Chinn IK, Milner JD, Scheinberg P, Douek DC, Markert ML. Thymus transplantation restores the repertoires of forkhead box protein 3 (FoxP3)+ and FoxP3- T cells in complete DiGeorge anomaly. Clin Exp Immunol. 2013 Jul;173(1):140-9. doi: 10.1111/cei.12088.
Background: PubMed 20832849Chinn IK, Olson JA, Skinner MA, McCarthy EA, Gupton SE, Chen DF, Bonilla FA, Roberts RL, Kanariou MG, Devlin BH, Markert ML. Mechanisms of tolerance to parental parathyroid tissue when combined with human allogeneic thymus transplantation. J Allergy Clin Immunol. 2010 Oct;126(4):814-820.e8. doi: 10.1016/j.jaci.2010.07.016. Epub 2010 Sep 15.
Results publication: PubMed 17284531Markert ML, Devlin BH, Alexieff MJ, Li J, McCarthy EA, Gupton SE, Chinn IK, Hale LP, Kepler TB, He M, Sarzotti M, Skinner MA, Rice HE, Hoehner JC. Review of 54 patients with complete DiGeorge anomaly enrolled in protocols for thymus transplantation: outcome of 44 consecutive transplants. Blood. 2007 May 15;109(10):4539-47. doi: 10.1182/blood-2006-10-048652. Epub 2007 Feb 6.
Results publication: PubMed 20236866Markert ML, Devlin BH, McCarthy EA. Thymus transplantation. Clin Immunol. 2010 May;135(2):236-46. doi: 10.1016/j.clim.2010.02.007. Epub 2010 Mar 16.
Results publication: PubMed 18557726Markert ML, Devlin BH, Chinn IK, McCarthy EA, Li YJ. Factors affecting success of thymus transplantation for complete DiGeorge anomaly. Am J Transplant. 2008 Aug;8(8):1729-36. doi: 10.1111/j.1600-6143.2008.02301.x. Epub 2008 Jun 28.
Results publicationMarkert ML and Devlin BH. Thymic reconstitution (in Rich RR, Shearer WT, Fleischer T, Schroeder HW, Weyand CM, Frew A, eds., Clinical Immunology 3rd edn., Elsevier, Edinburgh) p 1253-1262, 2008.
Results publication: PubMed 18424759Markert ML, Li J, Devlin BH, Hoehner JC, Rice HE, Skinner MA, Li YJ, Hale LP. Use of allograft biopsies to assess thymopoiesis after thymus transplantation. J Immunol. 2008 May 1;180(9):6354-64. doi: 10.4049/jimmunol.180.9.6354.
Results publication: PubMed 18035553Hudson LL, Louise Markert M, Devlin BH, Haynes BF, Sempowski GD. Human T cell reconstitution in DiGeorge syndrome and HIV-1 infection. Semin Immunol. 2007 Oct;19(5):297-309. doi: 10.1016/j.smim.2007.10.002. Epub 2007 Nov 26.
Results publicationMarkert ML, Devlin BH, McCarthy EA, Chinn IK, Hale LP. Thymus Transplantation in Thymus Gland Pathology: Clinical, Diagnostic, and Therapeutic Features. Eds Lavinin C, Moran CA, Morandi U, Schoenhuber R. Springer-Verlag Italia, Milan, 2008, pp 255-267.
Results publication: PubMed 19066739Markert ML, Devlin BH, Chinn IK, McCarthy EA. Thymus transplantation in complete DiGeorge anomaly. Immunol Res. 2009;44(1-3):61-70. doi: 10.1007/s12026-008-8082-5.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

M. Louise Markert, MD, PhD principal investigator

Duke University Medical Center, Pediatrics, Allergy & Immunology

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 0 inclusion and 39 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 25 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Feb 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Survival at 1 Year Post-CTTI (time frame: 1 year post-CTTI). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00576836, which also lists 14 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2022.