Trial Outcomes & Findings for ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID (NCT NCT00598481)
NCT ID: NCT00598481
Last Updated: 2024-01-29
Results Overview
From post-treatment to up to 3 years
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
12 participants
Primary outcome timeframe
baseline to 3 years post gene therapy
Results posted on
2024-01-29
Participant Flow
The Pivotal study enrolled 12 participants.
Participant milestones
| Measure |
Gene Therapy
Infusion of autologous cluster of differentiation (CD)34+ cells transduced with retroviral vector encoding ADA after non-myeloablative conditioning with busulfan
|
|---|---|
|
Baseline
STARTED
|
12
|
|
Baseline
COMPLETED
|
12
|
|
Baseline
NOT COMPLETED
|
0
|
|
Treatment
STARTED
|
12
|
|
Treatment
COMPLETED
|
11
|
|
Treatment
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Gene Therapy
Infusion of autologous cluster of differentiation (CD)34+ cells transduced with retroviral vector encoding ADA after non-myeloablative conditioning with busulfan
|
|---|---|
|
Treatment
Physician Decision
|
1
|
Baseline Characteristics
ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
Baseline characteristics by cohort
| Measure |
Gene Therapy (Pivotal)
n=12 Participants
Infusion of autologous cluster of differentiation (CD)34+ cells transduced with retroviral vector encoding ADA after non-myeloablative conditioning with busulfan
|
|---|---|
|
Age, Categorical
<=18 years
|
12 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Age, Continuous
|
2.38 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
12 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
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
12 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
|
|
Region of Enrollment
Italy
|
12 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: baseline to 3 years post gene therapyFrom post-treatment to up to 3 years
Outcome measures
| Measure |
Gene Therapy (Pivotal)
n=12 Participants
Infusion of autologous cluster of differentiation (CD)34+ cells transduced with retroviral vector encoding ADA after non-myeloablative conditioning with busulfan
|
Severe Infections (After Gene Therapy)
This is the rate of severe infections post gene therapy, taken from 3 months post gene therapy to three years post gene therapy.
|
|---|---|---|
|
Survival
|
12 Participants
|
—
|
SECONDARY outcome
Timeframe: Before Treatment and 3-months post-treatment up to 3 yearsSevere infections were defined as those that required hospitalization or those that prolonged hospitalization. The rate of infection was estimated as number of severe infections over person-years of observation (free from severe infections) before and after treatment administration. The first 3 months after gene therapy were not considered in the post-gene therapy analysis, because all subjects were hospitalized during this period.
Outcome measures
| Measure |
Gene Therapy (Pivotal)
n=12 Participants
Infusion of autologous cluster of differentiation (CD)34+ cells transduced with retroviral vector encoding ADA after non-myeloablative conditioning with busulfan
|
Severe Infections (After Gene Therapy)
n=12 Participants
This is the rate of severe infections post gene therapy, taken from 3 months post gene therapy to three years post gene therapy.
|
|---|---|---|
|
Rate of Severe Infections
|
1.1 Severe Infections per person year
|
0.429 Severe Infections per person year
|
SECONDARY outcome
Timeframe: baseline up to 3 years post gene therapyPopulation: Only subjects with available data at each visit were included in the analysis
T-lymphocyte counts (CD3+): mean T-lymphocyte at Baseline and 3 years post gene therapy. Samples were taken from peripheral venous whole blood and tested by cytofluorometry; values are means (10\^6/L).
Outcome measures
| Measure |
Gene Therapy (Pivotal)
n=12 Participants
Infusion of autologous cluster of differentiation (CD)34+ cells transduced with retroviral vector encoding ADA after non-myeloablative conditioning with busulfan
|
Severe Infections (After Gene Therapy)
This is the rate of severe infections post gene therapy, taken from 3 months post gene therapy to three years post gene therapy.
|
|---|---|---|
|
CD3+ Cell Counts
Baseline
|
112.5 10^6 cells/L
Interval 39.4 to 321.0
|
—
|
|
CD3+ Cell Counts
Year 1
|
348.3 10^6 cells/L
Interval 194.5 to 623.8
|
—
|
|
CD3+ Cell Counts
Year 2
|
633 10^6 cells/L
Interval 482.9 to 829.8
|
—
|
|
CD3+ Cell Counts
Year 3
|
831.1 10^6 cells/L
Interval 596.7 to 1157.6
|
—
|
Adverse Events
Gene Therapy (Pivotal)
Serious events: 10 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Gene Therapy (Pivotal)
n=12 participants at risk
Infusion of autologous cluster of differentiation (CD)34+ cells transduced with retroviral vector encoding ADA after non-myeloablative conditioning with busulfan
|
|---|---|
|
Infections and infestations
Device related infection
|
41.7%
5/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Gastroenteritis
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Esptein-Barr virus infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Respiratory Tract Infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Upper Respiratory tract infection (bacterial)
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Urinary Tract Infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Varicella
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Blood and lymphatic system disorders
Neutropenia
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Blood and lymphatic system disorders
Aplastic anemia
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Blood and lymphatic system disorders
Autoimmune thrombocytopenia
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Gastrointestinal disorders
Diarrhea
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
General disorders
Pyrexia
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Hepatobiliary disorders
Autoimmune hepatitis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lipofibroma
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Vascular disorders
Hypertension
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Nervous system disorders
Guillian-Barre Syndrome
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
Other adverse events
| Measure |
Gene Therapy (Pivotal)
n=12 participants at risk
Infusion of autologous cluster of differentiation (CD)34+ cells transduced with retroviral vector encoding ADA after non-myeloablative conditioning with busulfan
|
|---|---|
|
Blood and lymphatic system disorders
Autoimmune haemolytic anemia
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Blood and lymphatic system disorders
Neutropenia
|
33.3%
4/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Blood and lymphatic system disorders
Eosinophilia
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Rhinitis
|
66.7%
8/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Device related infection
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Upper respiratory tract infection
|
41.7%
5/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Gastroenteritis
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Oral candidiasis
|
33.3%
4/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Bronchitis
|
25.0%
3/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Escherichia urinary tract infection
|
25.0%
3/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Nasopharyngitis
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Upper respiratory tract infection bacterial
|
25.0%
3/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Urinary traction infection pseudomonal
|
25.0%
3/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Varicella
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Hemophilus infection
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Otitis media
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Respiratory tract infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Gastroenteritis norovirus
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Gingivitis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Influenza
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Otitis media acute
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Pharyngitis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Pneumococcal infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Respiratory syncytial virus infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Skin infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Staphylococcal infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Streptococcal infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Tinea capitis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Urinary tract infection enterococcal
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Viral diarrhea
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Computerized tomogram thorax abnormal
|
25.0%
3/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Hepatic enzyme increased
|
41.7%
5/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Blood immunoglobulin E increased
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Electrophoresis protein abnormal
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Blood alkaline phosphatase increased
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Pseudomonas test positive
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Tympanometry abnormal
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Weight decreased
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Adenovirus test positive
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Bacterial test positive
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Clostridium test positive
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Pulmonary function test abnormal
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Serum ferritin decreased
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Spirometry abnormal
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Staphylococcus test positive
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Investigations
Total lung capacity decreased
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
4/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Interstitial lung disease
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Bronchial wall thickening
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Nasal turbinate hypertrophy
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary calcification
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary mass
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
25.0%
3/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Rash
|
25.0%
3/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Dermatitis atopic
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Drug eruption
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Eczema
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Eczema nummular
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Erythema multiforme
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Lichen planus
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Scab
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Metabolism and nutrition disorders
Decreased appetite
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Congenital, familial and genetic disorders
Phimosis
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Congenital, familial and genetic disorders
Cryptorchism
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Congenital, familial and genetic disorders
Congenital genital malformation
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Congenital, familial and genetic disorders
Micropenis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Congenital, familial and genetic disorders
Reproductive tract hypoplasia, male
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Gastrointestinal disorders
Vomiting
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Gastrointestinal disorders
Enteritis
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Gastrointestinal disorders
Dental caries
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Gastrointestinal disorders
Oral disorder
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
General disorders
Developmental delay
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
General disorders
Adverse drug reaction
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
General disorders
Device occlusion
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Nervous system disorders
Cerebral hematoma
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Nervous system disorders
Psychomotor hyperactivity
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Nervous system disorders
Speech disorder
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Hepatobiliary disorders
Hepatomegaly
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Hepatobiliary disorders
Autoimmune hepatitis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Hepatobiliary disorders
Gallbladder cholesterolosis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Hepatobiliary disorders
Hepatic fibrosis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Hepatobiliary disorders
Hepatic lesion
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Musculoskeletal and connective tissue disorders
Bone development abnormal
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Endocrine disorders
Hypothyroidism
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Injury, poisoning and procedural complications
Wound
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Psychiatric disorders
Food aversion
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Ear and labyrinth disorders
Deafness bilateral
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Ear and labyrinth disorders
Deafness
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Ear and labyrinth disorders
Middle ear inflammation
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Eye disorders
Amblyopia
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Eye disorders
Hypermetropia
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Eye disorders
Myopia
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Eye disorders
Refraction disorder
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Cardiac disorders
Pericardial effusion
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Cardiac disorders
Pulmonary valve stenosis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Cardiac disorders
Sinus arrhythmia
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Cardiac disorders
Wandering pacemaker
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Reproductive system and breast disorders
Acquired phimosis
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Reproductive system and breast disorders
Perineal erythema
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hemangioma of liver
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lipofibroma
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Pyogenic granuloma
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Immune system disorders
Anaphylactic reaction
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Renal and urinary disorders
Hypertonic bladder
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Metabolism and nutrition disorders
Acidosis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Aspergillis infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Campylobacter infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Candida infection
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Catheter site infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Clostridium difficile colitis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Clostridium difficile infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Ear infection
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Escherichia sepsis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Fungal skin infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Sinusitis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Staphylococcal sepsis
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Blood and lymphatic system disorders
Anaemia
|
33.3%
4/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Blood and lymphatic system disorders
Autoimmune thrombocytopenia
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Gastrointestinal disorders
Diarrhoea
|
33.3%
4/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
General disorders
Pyrexia
|
33.3%
4/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Epstein-Barr virus infection
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Respiratory Tract Infection, bacterial
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Infections and infestations
Urinary Tract Infection (bacterial)
|
16.7%
2/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Vascular disorders
Hypertension
|
25.0%
3/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
|
Skin and subcutaneous tissue disorders
Skin exfoliation
|
8.3%
1/12 • Adverse events were collected from pre-treatment through year 3, post gene therapy
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60