Trial Outcomes & Findings for Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene (NCT NCT01852071)
NCT ID: NCT01852071
Last Updated: 2022-08-03
Results Overview
Overall survival is defined as the percentage of subjects alive at 12 months post- treatment with OTL-101 or HSCT
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
46 participants
Primary outcome timeframe
12 months
Results posted on
2022-08-03
Participant Flow
Participant milestones
| Measure |
Gene Therapy
Infusion of autologous CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV)
Busulfan: Busulfan is used for non-myeloablative conditioning
Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
|
Historical Control Group
Historical data from patients with Severe Combined Immunodeficiency Due to ADA Deficiency (ADA-SCID) who were treated with Hematopoietic Stem Cell Transplantation (HSCT): Historical data from a database of ADA-SCID patients treated with allogeneic HSCT from Great Ormond Street Hospital (GOSH) and Duke University Children's Hospital were collected as comparator group.
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|---|---|---|
|
Overall Study
STARTED
|
20
|
26
|
|
Overall Study
COMPLETED
|
20
|
26
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
Baseline characteristics by cohort
| Measure |
Gene Therapy
n=20 Participants
Infusion of autologous CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV)
Busulfan: Busulfan is used for non-myeloablative conditioning
Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
|
Historical Control Group
n=26 Participants
Historical data from patients with Severe Combined Immunodeficiency Due to ADA Deficiency (ADA-SCID) who were treated with Hematopoietic Stem Cell Transplantation (HSCT): Historical data from a database of ADA-SCID patients treated with allogeneic HSCT from Great Ormond Street Hospital (GOSH) and Duke University Children's Hospital were collected as comparator group.
|
Total
n=46 Participants
Total of all reporting groups
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|---|---|---|---|
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Race/Ethnicity, Customized
African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
6 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Native American or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
10 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Unknown
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Not Reported
|
0 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Age, Categorical
<=18 years
|
20 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
46 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex/Gender, Customized
Sex, n · Female
|
11 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Sex/Gender, Customized
Sex, n · Male
|
9 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex/Gender, Customized
Sex, n · Not Reported
|
0 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Region of Enrollment
United States
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20 participants
n=5 Participants
|
10 participants
n=7 Participants
|
30 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
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0 participants
n=5 Participants
|
16 participants
n=7 Participants
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16 participants
n=5 Participants
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PRIMARY outcome
Timeframe: 12 monthsOverall survival is defined as the percentage of subjects alive at 12 months post- treatment with OTL-101 or HSCT
Outcome measures
| Measure |
OTL-101 Gene Therapy
n=20 Participants
The primary efficacy population consists of all subjects treated with OTL-101 at University of California, Los Angeles/National Institutes of Health (UCLA/NIH)
|
HSCT Controls Without MRD
n=14 Participants
The primary comparator population from the HSCT historical control group consists of ADA-SCID patients without a medically eligible human leukocyte antigen (HLA)-identical sibling or family donor and treated with HSCT at either GOSH or Duke University between 2000 and 2016.
|
HSCT Controls With MRD
n=12 Participants
ADA-SCID patients with an Matched Related Donor (MRD) treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
All HSCT Control Group
n=26 Participants
The complete HSCT historical control group, which consists of ADA-SCID patients with any type of donor treated with HSCT at either GOSH or Duke University from 2000 to 2016
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|---|---|---|---|---|
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Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
|
100 percentage of participants
Interval 83.16 to 100.0
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85.71 percentage of participants
Interval 57.19 to 98.22
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100 percentage of participants
Interval 73.54 to 100.0
|
92.31 percentage of participants
Interval 74.87 to 99.05
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PRIMARY outcome
Timeframe: 12 monthsEvent-free survival is defined as the percentage of subjects alive with no "event", an "event" being the resumption of PEG-ADA ERT or the need for a rescue allogeneic Hematopoietic Stem Cell Transplant (HSCT), or death.
Outcome measures
| Measure |
OTL-101 Gene Therapy
n=20 Participants
The primary efficacy population consists of all subjects treated with OTL-101 at University of California, Los Angeles/National Institutes of Health (UCLA/NIH)
|
HSCT Controls Without MRD
n=14 Participants
The primary comparator population from the HSCT historical control group consists of ADA-SCID patients without a medically eligible human leukocyte antigen (HLA)-identical sibling or family donor and treated with HSCT at either GOSH or Duke University between 2000 and 2016.
|
HSCT Controls With MRD
n=12 Participants
ADA-SCID patients with an Matched Related Donor (MRD) treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
All HSCT Control Group
n=26 Participants
The complete HSCT historical control group, which consists of ADA-SCID patients with any type of donor treated with HSCT at either GOSH or Duke University from 2000 to 2016
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|---|---|---|---|---|
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Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
|
100 percentage of participants
Interval 83.16 to 100.0
|
64.29 percentage of participants
Interval 35.14 to 87.24
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100 percentage of participants
Interval 73.54 to 100.0
|
80.77 percentage of participants
Interval 60.65 to 93.45
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SECONDARY outcome
Timeframe: 24 monthsOS is defined as the percentage of subjects alive at 24 months post- treatment with OTL-101 or HSCT
Outcome measures
| Measure |
OTL-101 Gene Therapy
n=20 Participants
The primary efficacy population consists of all subjects treated with OTL-101 at University of California, Los Angeles/National Institutes of Health (UCLA/NIH)
|
HSCT Controls Without MRD
n=14 Participants
The primary comparator population from the HSCT historical control group consists of ADA-SCID patients without a medically eligible human leukocyte antigen (HLA)-identical sibling or family donor and treated with HSCT at either GOSH or Duke University between 2000 and 2016.
|
HSCT Controls With MRD
n=12 Participants
ADA-SCID patients with an Matched Related Donor (MRD) treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
All HSCT Control Group
n=26 Participants
The complete HSCT historical control group, which consists of ADA-SCID patients with any type of donor treated with HSCT at either GOSH or Duke University from 2000 to 2016
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|---|---|---|---|---|
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OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)
|
100 percentage of participants
Interval 83.16 to 100.0
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85.71 percentage of participants
Interval 57.19 to 98.22
|
90.91 percentage of participants
Interval 58.72 to 99.77
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88.00 percentage of participants
Interval 68.78 to 97.45
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SECONDARY outcome
Timeframe: 24 monthsEvent-free survival is defined as the percentage of subjects alive with no "event", an "event" being the resumption of PEG-ADA ERT or the need for a rescue allogenic Hematopoietic Stem Cell Transplant (HSCT), or death.
Outcome measures
| Measure |
OTL-101 Gene Therapy
n=20 Participants
The primary efficacy population consists of all subjects treated with OTL-101 at University of California, Los Angeles/National Institutes of Health (UCLA/NIH)
|
HSCT Controls Without MRD
n=14 Participants
The primary comparator population from the HSCT historical control group consists of ADA-SCID patients without a medically eligible human leukocyte antigen (HLA)-identical sibling or family donor and treated with HSCT at either GOSH or Duke University between 2000 and 2016.
|
HSCT Controls With MRD
n=12 Participants
ADA-SCID patients with an Matched Related Donor (MRD) treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
All HSCT Control Group
n=26 Participants
The complete HSCT historical control group, which consists of ADA-SCID patients with any type of donor treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
|---|---|---|---|---|
|
EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)
|
100 percentage of participants
Interval 83.16 to 100.0
|
50.00 percentage of participants
Interval 23.04 to 76.96
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63.64 percentage of participants
Interval 30.79 to 89.07
|
56.00 percentage of participants
Interval 34.93 to 75.6
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SECONDARY outcome
Timeframe: 24 monthsPopulation: Historical control groups/arms are not included in the analysis population for this outcome measure as these subjects did not receive gene therapy, and so measurement of VCN levels is not relevant
Vector copy number in the PB granulocyte fraction that was T cell depleted, is a surrogate for amount of engrafted genetically modified Hematopoietic stem cell (HSC) that are producing granulocytes every 3-5 days. VCN analysis was performed by Droplet Digital PCR (ddPCR) on DNA extracted from peripheral blood granulocytes.
Outcome measures
| Measure |
OTL-101 Gene Therapy
n=18 Participants
The primary efficacy population consists of all subjects treated with OTL-101 at University of California, Los Angeles/National Institutes of Health (UCLA/NIH)
|
HSCT Controls Without MRD
The primary comparator population from the HSCT historical control group consists of ADA-SCID patients without a medically eligible human leukocyte antigen (HLA)-identical sibling or family donor and treated with HSCT at either GOSH or Duke University between 2000 and 2016.
|
HSCT Controls With MRD
ADA-SCID patients with an Matched Related Donor (MRD) treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
All HSCT Control Group
The complete HSCT historical control group, which consists of ADA-SCID patients with any type of donor treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
|---|---|---|---|---|
|
Vector Copy Number (VCN) in Peripheral Blood (PB) Granulocytes.
|
0.093 copies/cell
Interval 0.04 to 2.5
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Historical control groups/arms are not included in the analysis population for this outcome measure as these subjects did not receive gene therapy, and so measurement of VCN levels is not relevant
PBMC VCN is a measure of the accumulation of peripheral blood leukocytes arising from engrafted, genetically modified HSC. VCN analysis was performed by ddPCR on DNA extracted from PBMC.
Outcome measures
| Measure |
OTL-101 Gene Therapy
n=18 Participants
The primary efficacy population consists of all subjects treated with OTL-101 at University of California, Los Angeles/National Institutes of Health (UCLA/NIH)
|
HSCT Controls Without MRD
The primary comparator population from the HSCT historical control group consists of ADA-SCID patients without a medically eligible human leukocyte antigen (HLA)-identical sibling or family donor and treated with HSCT at either GOSH or Duke University between 2000 and 2016.
|
HSCT Controls With MRD
ADA-SCID patients with an Matched Related Donor (MRD) treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
All HSCT Control Group
The complete HSCT historical control group, which consists of ADA-SCID patients with any type of donor treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
|---|---|---|---|---|
|
VCN in Peripheral Blood Mononuclear Cells (PBMCs)
|
0.972 copies/cell
Interval 0.13 to 1.86
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: 24 monthsADA enzyme activity measured to assess the amount of functional gene product produced from the normal ADA transgene delivered by EFS-ADA LV; persistence of ADA enzyme activity over time demonstrates successful engraftment and differentiation of genetically modified HSC.
Outcome measures
| Measure |
OTL-101 Gene Therapy
n=14 Participants
The primary efficacy population consists of all subjects treated with OTL-101 at University of California, Los Angeles/National Institutes of Health (UCLA/NIH)
|
HSCT Controls Without MRD
n=4 Participants
The primary comparator population from the HSCT historical control group consists of ADA-SCID patients without a medically eligible human leukocyte antigen (HLA)-identical sibling or family donor and treated with HSCT at either GOSH or Duke University between 2000 and 2016.
|
HSCT Controls With MRD
n=3 Participants
ADA-SCID patients with an Matched Related Donor (MRD) treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
All HSCT Control Group
n=7 Participants
The complete HSCT historical control group, which consists of ADA-SCID patients with any type of donor treated with HSCT at either GOSH or Duke University from 2000 to 2016
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|---|---|---|---|---|
|
ADA Activity in Erythrocytes
|
105.50 nmol/h/mg
Interval 34.1 to 268.9
|
48.500 nmol/h/mg
Interval 0.0 to 123.0
|
1.000 nmol/h/mg
Interval 1.0 to 2.0
|
2.000 nmol/h/mg
Interval 0.0 to 123.0
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: HSCT Controls With MRD arm is not included in the analysis population for this outcome measure as dAXP data was not reported between 17-30 months post-HSCT for any patients in this arm.
Decreased dAXP levels coincide with increased ADA enzyme activity, detoxification was used to demonstrate functional ADA enzyme production from the introduced ADA transgene. The threshold for detoxification was \<100 μmol/L.
Outcome measures
| Measure |
OTL-101 Gene Therapy
n=14 Participants
The primary efficacy population consists of all subjects treated with OTL-101 at University of California, Los Angeles/National Institutes of Health (UCLA/NIH)
|
HSCT Controls Without MRD
n=5 Participants
The primary comparator population from the HSCT historical control group consists of ADA-SCID patients without a medically eligible human leukocyte antigen (HLA)-identical sibling or family donor and treated with HSCT at either GOSH or Duke University between 2000 and 2016.
|
HSCT Controls With MRD
n=5 Participants
ADA-SCID patients with an Matched Related Donor (MRD) treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
All HSCT Control Group
The complete HSCT historical control group, which consists of ADA-SCID patients with any type of donor treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
|---|---|---|---|---|
|
Reduction in Deoxyadenosine Nucleotide (dAXP) in Erythrocytes
|
0.0330 umol/mL
Interval 0.007 to 0.101
|
0.0360 umol/mL
Interval 0.004 to 0.256
|
0.0360 umol/mL
Interval 0.004 to 0.256
|
—
|
SECONDARY outcome
Timeframe: 24 monthsImmune reconstitution was assessed by change in CD3+ T Cell counts at baseline to Month 24.
Outcome measures
| Measure |
OTL-101 Gene Therapy
n=14 Participants
The primary efficacy population consists of all subjects treated with OTL-101 at University of California, Los Angeles/National Institutes of Health (UCLA/NIH)
|
HSCT Controls Without MRD
n=5 Participants
The primary comparator population from the HSCT historical control group consists of ADA-SCID patients without a medically eligible human leukocyte antigen (HLA)-identical sibling or family donor and treated with HSCT at either GOSH or Duke University between 2000 and 2016.
|
HSCT Controls With MRD
n=1 Participants
ADA-SCID patients with an Matched Related Donor (MRD) treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
All HSCT Control Group
n=6 Participants
The complete HSCT historical control group, which consists of ADA-SCID patients with any type of donor treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
|---|---|---|---|---|
|
Change From Baseline in CD3+ T Cell Counts (2 Years)
|
569.0 cells/μL
Interval -426.0 to 2211.0
|
340.0 cells/μL
Interval -23.0 to 1243.0
|
538.0 cells/μL
Interval 538.0 to 538.0
|
395.5 cells/μL
Interval -23.0 to 1243.0
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Historical control groups/arms are not included in the analysis population for this outcome measure as these subjects did not receive gene therapy, and so measurement of VISA is not relevant
Vector Integration Site Analysis (VISA) allowed determination of the distribution of vector integration sites in each subject's genome, as well as the relative clonal abundance. VISA was to be considered abnormal for a subject if, in 2 or more instances during the course of follow-up, a single integration site was found to represent \>30% of the total integration sites detected.
Outcome measures
| Measure |
OTL-101 Gene Therapy
n=19 Participants
The primary efficacy population consists of all subjects treated with OTL-101 at University of California, Los Angeles/National Institutes of Health (UCLA/NIH)
|
HSCT Controls Without MRD
The primary comparator population from the HSCT historical control group consists of ADA-SCID patients without a medically eligible human leukocyte antigen (HLA)-identical sibling or family donor and treated with HSCT at either GOSH or Duke University between 2000 and 2016.
|
HSCT Controls With MRD
ADA-SCID patients with an Matched Related Donor (MRD) treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
All HSCT Control Group
The complete HSCT historical control group, which consists of ADA-SCID patients with any type of donor treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
|---|---|---|---|---|
|
Number of Single Integration Sites Representing >30% of the Total Integration Sites (2 Years)
|
0 number of participants
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: 24 monthsThe infections of interest in this study were severe infections or opportunistic infectious episodes, defined as infections requiring hospitalization or prolonging hospitalization and/or documented infections by opportunistic pathogens. Infections that took place in the first 3 months of follow-up post treatment were excluded from calculations to avoid possible bias introduced in the data by the effects of conditioning.
Outcome measures
| Measure |
OTL-101 Gene Therapy
n=20 Participants
The primary efficacy population consists of all subjects treated with OTL-101 at University of California, Los Angeles/National Institutes of Health (UCLA/NIH)
|
HSCT Controls Without MRD
n=13 Participants
The primary comparator population from the HSCT historical control group consists of ADA-SCID patients without a medically eligible human leukocyte antigen (HLA)-identical sibling or family donor and treated with HSCT at either GOSH or Duke University between 2000 and 2016.
|
HSCT Controls With MRD
n=12 Participants
ADA-SCID patients with an Matched Related Donor (MRD) treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
All HSCT Control Group
n=25 Participants
The complete HSCT historical control group, which consists of ADA-SCID patients with any type of donor treated with HSCT at either GOSH or Duke University from 2000 to 2016
|
|---|---|---|---|---|
|
Severe Infection Rate Excluding the First Three Months After Treatment
|
0.20 Infection rate per person per year
|
0.56 Infection rate per person per year
|
0.15 Infection rate per person per year
|
0.36 Infection rate per person per year
|
Adverse Events
OTL-101 Gene Therapy
Serious events: 9 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
OTL-101 Gene Therapy
n=20 participants at risk
The safety population consists of all subjects treated with OTL-101 at UCLA/NIH
|
|---|---|
|
Infections and infestations
Upper respiratory tract infection
|
10.0%
2/20 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Pneumonia
|
5.0%
1/20 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Bronchiolitis
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Clostridium difficile colitis
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Device related infection
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Influenza
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Otitis media
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Rhinovirus infection
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Sepsis
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Staphylococcal bacteraemia
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Gastrointestinal disorders
Vomiting
|
15.0%
3/20 • Number of events 4 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Gastrointestinal disorders
Diarrhoea
|
15.0%
3/20 • Number of events 3 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
General disorders
Fatigue
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
General disorders
Pyrexia
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Immune system disorders
Immune reconstitution inflammatory syndrome
|
10.0%
2/20 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Investigations
Alanine aminotransferase increased
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Investigations
Weight decreased
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Blood and lymphatic system disorders
Neutropenia
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Nervous system disorders
Ataxia
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
Other adverse events
| Measure |
OTL-101 Gene Therapy
n=20 participants at risk
The safety population consists of all subjects treated with OTL-101 at UCLA/NIH
|
|---|---|
|
Blood and lymphatic system disorders
Neutropenia
|
85.0%
17/20 • Number of events 31 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Blood and lymphatic system disorders
Anemia
|
65.0%
13/20 • Number of events 18 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
15.0%
3/20 • Number of events 3 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
10.0%
2/20 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Blood and lymphatic system disorders
Leukopenia
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Upper respiratory tract infection
|
25.0%
5/20 • Number of events 8 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Rhinovirus infection
|
20.0%
4/20 • Number of events 4 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Otitis media
|
20.0%
4/20 • Number of events 8 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Influenza
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Nasopharyngitis
|
10.0%
2/20 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Parainfluenzae virus infection
|
10.0%
2/20 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Rhinitis
|
10.0%
2/20 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Ear infection
|
5.0%
1/20 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Aspergillus infection
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Clostridium difficile infection
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Conjunctivitis
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Fungal infection
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Gastroenteritis viral
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Molluscum contagiosum
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Periorbital cellulitis
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Respiratory tract infection viral
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Sinusitis
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Tinea infection
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Infections and infestations
Vulvovaginal mycotic infection
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Investigations
Alanine aminotransferase increased
|
45.0%
9/20 • Number of events 14 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Investigations
Aspartate aminotransferase increased
|
45.0%
9/20 • Number of events 11 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Investigations
Transaminases increased
|
10.0%
2/20 • Number of events 3 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Investigations
Hepatic enzyme increased
|
5.0%
1/20 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Investigations
Body temperature decreased
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Investigations
Epstein-Barr virus test positive
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Investigations
Influenza B virus test
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Gastrointestinal disorders
Diarrhoea
|
30.0%
6/20 • Number of events 8 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Gastrointestinal disorders
Vomiting
|
35.0%
7/20 • Number of events 14 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Gastrointestinal disorders
Constipation
|
15.0%
3/20 • Number of events 3 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Gastrointestinal disorders
Nausea
|
15.0%
3/20 • Number of events 3 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Gastrointestinal disorders
Aphthous ulcer
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Gastrointestinal disorders
Colitis
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Gastrointestinal disorders
Flatulence
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
30.0%
6/20 • Number of events 9 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Skin and subcutaneous tissue disorders
Rash
|
25.0%
5/20 • Number of events 8 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
20.0%
4/20 • Number of events 5 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
15.0%
3/20 • Number of events 3 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
15.0%
3/20 • Number of events 3 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Skin and subcutaneous tissue disorders
Blister
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Skin and subcutaneous tissue disorders
Seborrhoeic dermatitis
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
35.0%
7/20 • Number of events 13 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
35.0%
7/20 • Number of events 11 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
20.0%
4/20 • Number of events 5 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
General disorders
Pyrexia
|
35.0%
7/20 • Number of events 9 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Injury, poisoning and procedural complications
Animal bite
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Injury, poisoning and procedural complications
Ear injury
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Injury, poisoning and procedural complications
Fall
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Injury, poisoning and procedural complications
Head injury
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Eye disorders
Blepharospasm
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
|
Psychiatric disorders
Sleep disorder
|
5.0%
1/20 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101
Adverse events were reported for subjects treated with OTL-101. Adverse event reporting is not applicable for the historical control arms (without MRD, with MRD, and all control patients) as this data was not collected due to the historical nature of the population.
|
Additional Information
Orchard Medical Information
Orchard Therapeutics (Europe) Ltd
Phone: +44 (0) 203 808 8286
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place