Trial Outcomes & Findings for Expanded Access Protocol Thymus Transplantation (NCT NCT02274662)
NCT ID: NCT02274662
Last Updated: 2025-01-16
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
11 participants
Primary outcome timeframe
1 Year
Results posted on
2025-01-16
Participant Flow
Participant milestones
| Measure |
Cultured Thymus Tissue Implantation
Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders
|
|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
7
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
Cultured Thymus Tissue Implantation
Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders
|
|---|---|
|
Overall Study
Death
|
1
|
|
Overall Study
Lost to Follow-up
|
3
|
Baseline Characteristics
Expanded Access Protocol Thymus Transplantation
Baseline characteristics by cohort
| Measure |
Cultured Thymus Tissue Implantation
n=11 Participants
Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders
|
|---|---|
|
Age, Continuous
|
1485.5 days
STANDARD_DEVIATION 1601.91 • n=99 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 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 51692
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=11 Participants
Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders
|
|---|---|
|
Survival Rate at End of 1 Year
|
90.9 percentage of participants
Interval 59.0 to 100.0
|
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 51692
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=11 Participants
Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders
|
|---|---|
|
Survival Rate at End of 2 Year
|
87.5 percentage of participants
Interval 47.0 to 100.0
|
Adverse Events
Cultured Thymus Tissue Implantation
Serious events: 8 serious events
Other events: 11 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Cultured Thymus Tissue Implantation
n=11 participants at risk
Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders
|
|---|---|
|
Infections and infestations
Device related infection
|
27.3%
3/11 • Number of events 13 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Viral upper respiratory tract infection
|
18.2%
2/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Bacterial infection
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Bacterial tracheitis
|
9.1%
1/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Clostridium difficile colitis
|
9.1%
1/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Lower respiratory tract infection bacterial
|
9.1%
1/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Respiratory tract infection bacterial
|
9.1%
1/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Tracheitis
|
9.1%
1/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Cellulitis
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Gastroenteritis adenovirus
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Lower respiratory tract infection viral
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Pneumonia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Sinusitis fungal
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Subacute endocarditis
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Viraemia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Neutropenia
|
18.2%
2/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Coombs positive haemolytic anaemia
|
18.2%
2/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
18.2%
2/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Haemolytic anaemia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Histiocytosis haematophagic
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Splenomegaly
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Diarrhoea
|
18.2%
2/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Gastrointestinal inflammation
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Haematochezia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Ileus
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Vomiting
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
General disorders
Pyrexia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
General disorders
Pneumatosis
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
9.1%
1/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Cardiac disorders
Cardio-respiratory arrest
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Immune system disorders
Graft versus host disease
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Injury, poisoning and procedural complications
Stress fracture
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Product Issues
Thrombosis in device
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Renal and urinary disorders
Acute kidney injury
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
Other adverse events
| Measure |
Cultured Thymus Tissue Implantation
n=11 participants at risk
Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders
|
|---|---|
|
Infections and infestations
Viral upper respiratory tract infection
|
18.2%
2/11 • Number of events 4 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Acarodermatitis
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Bacterial infection
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Clostridium bacteraemia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Ear infection fungal
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Gastroenteritis norovirus
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Oesophageal candidiasis
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Oral viral infection
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Otitis media chronic
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Pneumonia bacterial
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Sinusitis bacterial
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Infections and infestations
Viral rash
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma of skin
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Neutropenia
|
18.2%
2/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Immune system disorders
Cytokine release syndrome
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
27.3%
3/11 • Number of events 3 • 2 years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Acidosis
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Hypernatraemia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Metabolism and nutrition disorders
Weight gain poor
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Psychiatric disorders
Irritability
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Cardiac disorders
Bradycardia
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Cardiac disorders
Left ventricular hypertrophy
|
9.1%
1/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Vascular disorders
Hypertension
|
27.3%
3/11 • Number of events 3 • 2 years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
18.2%
2/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Diarrhoea
|
27.3%
3/11 • Number of events 4 • 2 years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Abdominal pain
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Aphthous ulcer
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Enterocolitis
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Gastrointestinal disorders
Gingival hypertrophy
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Hepatobiliary disorders
Hepatomegaly
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Rash
|
18.2%
2/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
9.1%
1/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Ecchymosis
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Eczema
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Erythema
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Musculoskeletal and connective tissue disorders
Synovitis
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Renal and urinary disorders
Acute kidney injury
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Renal and urinary disorders
Glycosuria
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Renal and urinary disorders
Proteinuria
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Renal and urinary disorders
Renal failure
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
General disorders
Pyrexia
|
45.5%
5/11 • Number of events 6 • 2 years
within 2 Years of Transplantation
|
|
General disorders
Oedema peripheral
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Investigations
Blood creatinine increased
|
18.2%
2/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Investigations
Alanine aminotransferase increased
|
9.1%
1/11 • Number of events 2 • 2 years
within 2 Years of Transplantation
|
|
Investigations
Aspartate aminotransferase increased
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Investigations
Blood iron decreased
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Investigations
Lipase increased
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Investigations
Neutrophil count decreased
|
9.1%
1/11 • Number of events 1 • 2 years
within 2 Years of Transplantation
|
|
Investigations
Weight decreased
|
9.1%
1/11 • Number of events 1 • 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