Trial Outcomes & Findings for Thymus Transplantation in DiGeorge Syndrome #668 (NCT NCT00576407)
NCT ID: NCT00576407
Last Updated: 2022-03-25
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
26 participants
Primary outcome timeframe
1 year post-CTTI
Results posted on
2022-03-25
Participant Flow
Participant milestones
| Measure |
Cultured Thymus Tissue Implantation
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Overall Study
STARTED
|
26
|
|
Overall Study
COMPLETED
|
18
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
Cultured Thymus Tissue Implantation
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Overall Study
Death
|
7
|
|
Overall Study
Physician Decision
|
1
|
Baseline Characteristics
Thymus Transplantation in DiGeorge Syndrome #668
Baseline characteristics by cohort
| Measure |
Cultured Thymus Tissue Implantation
n=26 Participants
Cultured Thymus Tissue Implantation is done using allogeneic cultured postnatal thymus tissue from unrelated donors.
|
|---|---|
|
Age, Categorical
<=18 years
|
26 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
153 days
STANDARD_DEVIATION 102 • n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
16 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
26 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 year post-CTTIPopulation: Of the 26 participants, 2 subjects did not have cDGA (1 SCID and 1 FoxN1) and underwent CTTI as enrollment exceptions. The FoxN1 participant is included in the efficacy analysis, thus n=25. The SCID participant is not included in the efficacy analysis as CTTI cannot lead to T cell development in SCID.
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.
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=25 Participants
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Survival at 1 Year Post-Cultured Thymus Tissue Implantation (CTTI)
|
72 % of participants who survive to 1 year
Interval 50.1 to 85.5
|
SECONDARY outcome
Timeframe: 2 years post-CTTIPopulation: Of the 26 participants, 2 subjects did not have cDGA (1 SCID and 1 FoxN1) and underwent CTTI as enrollment exceptions. The FoxN1 participant is included in the efficacy analysis, thus n=25. The SCID participant is not included in the efficacy analysis as CTTI cannot lead to T cell development in SCID.
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.
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=25 Participants
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Survival at 2 Years Post-CTTI
|
72 % of participants who survive to 2 years
Interval 50.1 to 85.5
|
SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA and FoxN1 participants for the 1 year time point if a CD3 T cell count was performed in the relevant time period.
The development of total CD3 T cells at one year as measured using flow cytometry
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=17 Participants
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Immune Reconstitution Efficacy - Total CD3 T Cells
|
770 cells/mm3
Interval 182.0 to 1698.0
|
SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA and FoxN1 participants for the 1 year time point if a CD4 T cell count was performed in the relevant time period.
The development of total CD4 T cells at one year as measured using flow cytometry
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=17 Participants
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Immune Reconstitution Efficacy - Total CD4 T Cells
|
570 cells/mm3
Interval 156.0 to 1376.0
|
SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA and FoxN1 participants for the 1 year time point if a CD8 T cell count was performed in the relevant time period.
The development of total CD8 T cells at one year as measured using flow cytometry
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=16 Participants
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Immune Reconstitution Efficacy - Total CD8 T Cells
|
122 cells/mm3
Interval 14.0 to 382.0
|
SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA and FoxN1 participants for the 1 year time point if a T cell count was performed in the relevant time period.
The development of naive CD4 T cells at one year as measured using flow cytometry
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=13 Participants
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Immune Reconstitution Efficacy - Naive CD4 T Cells
|
270 cells/mm3
Interval 48.0 to 669.0
|
SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA and FoxN1 participants for the 1 year time point if a T cell count was performed in the relevant time period.
The development of naive CD8 T cells at one year as measured using flow cytometry
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=11 Participants
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Immune Reconstitution Efficacy - Naive CD8 T Cells
|
65 cells/mm3
Interval 2.7 to 188.0
|
SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA and FoxN1 participants for the 1 year time point if testing was performed in the relevant time period.
The development of a T cell proliferative response to the mitogen phytohemagglutinin.
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=13 Participants
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Immune Reconstitution Efficacy - Response to Mitogens
|
133000 counts/minute (cpm)
Interval 53913.0 to 390546.0
|
SECONDARY outcome
Timeframe: 2 to 3 months post-CTTIPopulation: Data were only included on cDGA and FoxN1 participants if the participant had a biopsy of the thymus tissue implanted.
Evidence, on biopsy of the thymus tissue implanted in muscle, that shows the development of new T cells.
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=19 Participants
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Thymus Allograft Biopsy
Evidence of thymopoiesis
|
17 Participants
|
|
Thymus Allograft Biopsy
Evidence of rejection
|
1 Participants
|
|
Thymus Allograft Biopsy
Inconclusive for thymopoiesis
|
1 Participants
|
Adverse Events
Cultured Thymus Tissue Implantation
Serious events: 23 serious events
Other events: 26 other events
Deaths: 7 deaths
Serious adverse events
| Measure |
Cultured Thymus Tissue Implantation
n=26 participants at risk
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
Infections and infestations
Device related infection
|
38.5%
10/26 • Number of events 19 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
19.2%
5/26 • Number of events 5 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
General disorders
Pyrexia
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Staphylococcal bacteraemia
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Vascular disorders
Hypotension
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
7.7%
2/26 • Number of events 4 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Gastrointestinal disorders
Diarrhoea
|
7.7%
2/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Sepsis
|
7.7%
2/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Endocrine disorders
Hypothyroidism
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Immune system disorders
Hypersensitivity
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Enterococcal sepsis
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Gastroenteritis rotavirus
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Influenza
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Parainfluenzae virus infection
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Viral upper respiratory tract infection
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Nervous system disorders
Seizure
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory acidosis
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Pneumonia klebsiella
|
3.8%
1/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Cardiac disorders
Cyanosis
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Cardiac disorders
Right ventricular hypertension
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Gastrointestinal disorders
Diarrhoea haemorrhagic
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Gastrointestinal disorders
Haematemesis
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Gastrointestinal disorders
Vomiting
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Immune system disorders
Graft versus host disease in skin
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Bronchitis
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Croup infectious
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Cystitis escherichia
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Enterococcal bacteraemia
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Gastroenteritis adenovirus
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Gastroenteritis enteroviral
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Klebsiella sepsis
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Lower respiratory tract infection bacterial
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Pneumonia
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Pneumonia respiratory syncytial viral
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Pneumonia staphylococcal
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Pseudomonal bacteraemia
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Streptococcal bacteraemia
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Urinary tract infection viral
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Varicella
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Viraemia
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Injury, poisoning and procedural complications
Procedural haemorrhage
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Injury, poisoning and procedural complications
Splenic rupture
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Investigations
Blood creatinine increased
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Investigations
Weight decreased
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Metabolism and nutrition disorders
Feeding intolerance
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Nervous system disorders
Central nervous system haemorrhage
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Nervous system disorders
Cranial nerve disorder
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Nervous system disorders
Febrile convulsion
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Nervous system disorders
Hydrocephalus
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Nervous system disorders
Hypertonia
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Nervous system disorders
Hypocalcaemic seizure
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Nervous system disorders
Infantile spasms
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Nervous system disorders
Motor dysfunction
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Renal and urinary disorders
Glomerulonephritis minimal lesion
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Alveolar lung disease
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Diaphragmatic paralysis
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory arrest
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Skin and subcutaneous tissue disorders
Stevens-Johnson syndrome
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Vascular disorders
Superior vena cava syndrome
|
3.8%
1/26 • Number of events 1 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
Other adverse events
| Measure |
Cultured Thymus Tissue Implantation
n=26 participants at risk
Cultured Thymus Tissue Implantation (previously described as transplantation) is done using allogeneic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
General disorders
Pyrexia
|
57.7%
15/26 • Number of events 26 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Skin and subcutaneous tissue disorders
Rash
|
34.6%
9/26 • Number of events 17 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Device related infection
|
19.2%
5/26 • Number of events 11 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Oropharyngeal candidiasis
|
19.2%
5/26 • Number of events 6 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Investigations
Alanine aminotransferase increased
|
19.2%
5/26 • Number of events 6 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Renal and urinary disorders
Hydronephrosis
|
19.2%
5/26 • Number of events 6 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Hepatobiliary disorders
Hepatomegaly
|
19.2%
5/26 • Number of events 5 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
19.2%
5/26 • Number of events 5 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Staphylococcal bacteraemia
|
15.4%
4/26 • Number of events 8 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Investigations
Aspartate aminotransferase increased
|
15.4%
4/26 • Number of events 5 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
15.4%
4/26 • Number of events 5 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
15.4%
4/26 • Number of events 4 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Endocrine disorders
Hypothyroidism
|
15.4%
4/26 • Number of events 4 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Gastrointestinal disorders
Diarrhoea
|
15.4%
4/26 • Number of events 4 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Clostridium difficile colitis
|
15.4%
4/26 • Number of events 4 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Eye infection staphylococcal
|
15.4%
4/26 • Number of events 4 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
15.4%
4/26 • Number of events 4 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Gastrointestinal disorders
Vomiting
|
11.5%
3/26 • Number of events 4 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Urinary tract infection enterococcal
|
11.5%
3/26 • Number of events 4 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Blood and lymphatic system disorders
Eosinophilia
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Gastrointestinal disorders
Umbilical hernia
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Immune system disorders
Hypersensitivity
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Ear infection
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Eye infection bacterial
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Gastrointestinal viral infection
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Otitis media
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Urinary tract infection fungal
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Vascular disorders
Hypertension
|
11.5%
3/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Cystitis klebsiella
|
7.7%
2/26 • Number of events 4 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Candiduria
|
7.7%
2/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
7.7%
2/26 • Number of events 3 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Blood and lymphatic system disorders
Anaemia
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Blood and lymphatic system disorders
Disseminated intravascular coagulation
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Blood and lymphatic system disorders
Splenomegaly
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Cardiac disorders
Bundle branch block right
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Congenital, familial and genetic disorders
Hydrocele
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Gastrointestinal disorders
Constipation
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Gastrointestinal disorders
Pancreatic insufficiency
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
General disorders
Generalised oedema
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Enterococcal bacteraemia
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Lower respiratory tract infection bacterial
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Infections and infestations
Pneumonia staphylococcal
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Injury, poisoning and procedural complications
Stoma site haemorrhage
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Injury, poisoning and procedural complications
Wound dehiscence
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Investigations
Activated partial thromboplastin time prolonged
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Investigations
Occult blood positive
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Investigations
Prothrombin time prolonged
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Metabolism and nutrition disorders
Feeding intolerance
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Renal and urinary disorders
Nephrocalcinosis
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Renal and urinary disorders
Proteinuria
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Respiratory, thoracic and mediastinal disorders
Rales
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Skin and subcutaneous tissue disorders
Eczema
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Skin and subcutaneous tissue disorders
Skin exfoliation
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
7.7%
2/26 • Number of events 2 • 2 years post-CTTI
Adverse events reporting on all participants (cDGA, FoxN1, and SCID) who underwent cultured thymus tissue implantation (previously described as transplantation).
|
Additional Information
M. Louise Markert, MD, PhD Professor of Pediatrics and Immunology
Duke University Medical Center
Phone: 919-684-6263
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place