Trial Outcomes & Findings for Thymus Transplantation Safety-Efficacy (NCT NCT01220531)
NCT ID: NCT01220531
Last Updated: 2025-01-16
Results Overview
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
29 participants
Primary outcome timeframe
1 year
Results posted on
2025-01-16
Participant Flow
Participant milestones
| Measure |
Cultured Thymus Tissue
Subjects receive thymus transplant.
|
|---|---|
|
Overall Study
STARTED
|
29
|
|
Overall Study
COMPLETED
|
21
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
Cultured Thymus Tissue
Subjects receive thymus transplant.
|
|---|---|
|
Overall Study
Death
|
7
|
|
Overall Study
Lost to Follow-up
|
1
|
Baseline Characteristics
Thymus Transplantation Safety-Efficacy
Baseline characteristics by cohort
| Measure |
Cultured Thymus Tissue
n=29 Participants
Subjects receive thymus transplant.
|
|---|---|
|
Age, Continuous
|
449.7 Days
STANDARD_DEVIATION 226.27 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
19 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
21 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
21 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 yearPopulation: Efficacy Analysis Set (EAS): EAS set includes all subjects with athymia associated with complete DiGeorge anomaly or FoxN1 deficiency, who had no prior hematopoietic cell transplant (HCT) and were treated with cultured postnatal thymus tissue in study 25966
Outcome measures
| Measure |
Cultured Thymus Tissue
n=29 Participants
Subjects receive thymus transplant.
|
|---|---|
|
Survival Rate at End of 1 Year
|
79.3 percentage of participants
Interval 60.0 to 92.0
|
PRIMARY outcome
Timeframe: 2 yearPopulation: Efficacy Analysis Set (EAS): EAS set includes all subjects with athymia associated with complete DiGeorge anomaly or FoxN1 deficiency, who had no prior hematopoietic cell transplant (HCT) and were treated with cultured postnatal thymus tissue in study 25966
Outcome measures
| Measure |
Cultured Thymus Tissue
n=29 Participants
Subjects receive thymus transplant.
|
|---|---|
|
Survival Rate at End of 2 Year
|
75 percentage of participants
Interval 55.0 to 89.0
|
Adverse Events
Cultured Thymus Tissue
Serious events: 24 serious events
Other events: 29 other events
Deaths: 7 deaths
Serious adverse events
| Measure |
Cultured Thymus Tissue
n=29 participants at risk
Subjects receive thymus transplant.
|
|---|---|
|
Infections and infestations
Device related infection
|
48.3%
14/29 • Number of events 33 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Lower respiratory tract infection bacterial
|
10.3%
3/29 • Number of events 4 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Pneumonia
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Klebsiella bacteraemia
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Cellulitis
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Cytomegalovirus viraemia
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
24.1%
7/29 • Number of events 8 • 2 Years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
13.8%
4/29 • Number of events 4 • 2 Years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
10.3%
3/29 • Number of events 5 • 2 Years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
General disorders
Pyrexia
|
24.1%
7/29 • Number of events 8 • 2 Years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Pancreatitis
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Diarrhoea
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Renal and urinary disorders
Renal failure
|
13.8%
4/29 • Number of events 4 • 2 Years
within 2 Years of Transplantation
|
|
Renal and urinary disorders
Proteinuria
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Immune system disorders
Graft versus host disease
|
13.8%
4/29 • Number of events 4 • 2 Years
within 2 Years of Transplantation
|
Other adverse events
| Measure |
Cultured Thymus Tissue
n=29 participants at risk
Subjects receive thymus transplant.
|
|---|---|
|
Infections and infestations
Viral upper respiratory tract infection
|
24.1%
7/29 • Number of events 7 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Device related infection
|
20.7%
6/29 • Number of events 8 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Oropharyngeal candidiasis
|
20.7%
6/29 • Number of events 7 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Lower respiratory tract infection bacterial
|
13.8%
4/29 • Number of events 9 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Pneumonia
|
13.8%
4/29 • Number of events 4 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Enterobacter pneumonia
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Fungal skin infection
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Gastroenteritis norovirus
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Upper respiratory tract infection
|
10.3%
3/29 • Number of events 4 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Urinary tract infection bacterial
|
10.3%
3/29 • Number of events 7 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Bacterial tracheitis
|
6.9%
2/29 • Number of events 4 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Klebsiella bacteraemia
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Oral candidiasis
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Otitis media
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Sinusitis bacterial
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Staphylococcal skin infection
|
6.9%
2/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Stoma site infection
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Infections and infestations
Viraemia
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Anaemia
|
20.7%
6/29 • Number of events 7 • 2 Years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
20.7%
6/29 • Number of events 6 • 2 Years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Neutropenia
|
17.2%
5/29 • Number of events 5 • 2 Years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Splenomegaly
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Immune system disorders
Cytokine release syndrome
|
17.2%
5/29 • Number of events 5 • 2 Years
within 2 Years of Transplantation
|
|
Endocrine disorders
Hypothyroidism
|
13.8%
4/29 • Number of events 4 • 2 Years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
13.8%
4/29 • Number of events 4 • 2 Years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Acidosis
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Hypernatraemia
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Hypercalcaemia
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Cardiac disorders
Cardiomegaly
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Vascular disorders
Hypertension
|
20.7%
6/29 • Number of events 6 • 2 Years
within 2 Years of Transplantation
|
|
Vascular disorders
Thrombosis
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Diarrhoea
|
31.0%
9/29 • Number of events 9 • 2 Years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Haematochezia
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Vomiting
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Ileus
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Hepatobiliary disorders
Hepatomegaly
|
20.7%
6/29 • Number of events 7 • 2 Years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Rash
|
44.8%
13/29 • Number of events 19 • 2 Years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
10.3%
3/29 • Number of events 5 • 2 Years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Renal and urinary disorders
Acute kidney injury
|
10.3%
3/29 • Number of events 3 • 2 Years
within 2 Years of Transplantation
|
|
General disorders
Pyrexia
|
41.4%
12/29 • Number of events 17 • 2 Years
within 2 Years of Transplantation
|
|
General disorders
Granuloma
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
|
Investigations
Aspartate aminotransferase increased
|
20.7%
6/29 • Number of events 7 • 2 Years
within 2 Years of Transplantation
|
|
Investigations
Alanine aminotransferase increased
|
17.2%
5/29 • Number of events 7 • 2 Years
within 2 Years of Transplantation
|
|
Investigations
Blood creatinine increased
|
6.9%
2/29 • Number of events 2 • 2 Years
within 2 Years of Transplantation
|
Additional Information
Director Clinical Operations
Sumitomo Pharma America, Inc.
Phone: +1 (508) 481-6700
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee In the event the Study is part of a multi-center study, the first publication of the results of the Study shall be made in conjunction with the results of other participating study sites as a multi-center publication; provided however, if a multi-center publication is not forthcoming within twenty-four (24) months following completion of the Study at all sites, Institution and Investigator shall be free to publish.
- Publication restrictions are in place
Restriction type: OTHER